People Always, Patients Sometimes

On this episode of People Always, Patients Sometimes we welcome Michelle Shogren back to the podcast. Hi, I'm Tom Rhoads, CEO of Spencer Health Solutions. We recently spoke to Michelle Shogren, a familiar face in the clinical trials industry, most recently at Bayer Pharmaceuticals. She recently left Bayer to form her own consulting firm called Innovate In What You Do! Michelle is parlaying her 25 years of experience fostering teams and developing an innovation mindset into her consultancy. Michelle and our host, Janet Kennedy, have a lively conversation about innovation, which I know you'll enjoy on this episode of People Always, Patients Sometimes.   Janet Kennedy: (00:47) Hi, I'm Janet Kennedy and I'm a member of the Spencer Health Solutions team. It's always a pleasure to talk to Michelle Shogren on People Always, Patients Sometimes. We spoke to Michelle recently on an episode about innovation in clinical trials and the pharmaceutical industry. Today, we're gonna be talking about Michelle's new company Innovate In What You Do! Welcome back to the podcast, Michelle.   Michelle Shogren: (01:11) Hi, Janet. Thanks for having me back again.   Janet Kennedy: (01:13) I am so excited about your new company, but for those folks who might have jumped into this episode and hadn't heard the previous one, do you mind setting the stage a little bit and telling us a little bit about your background?   Michelle Shogren: (01:25) Sure, happy to do that. So I've been in the clinical trial space now for about 25 years in different roles of patient caregiver study coordinator, site, director, marketing director on that side of the fence. Then I jumped over into the CRO world as a monitor and on to pharma to be a monitor, country lead monitor, study lead monitor, and eventually working in process excellence, and then starting an innovation function. The innovation function I created in 2015 with a team of six people who were working on something called fostering innovation. And we were trying to prove innovation made sense in R&D other than coming up with just the molecule. We were able to successfully launch that and grow it over the seven year period to a point where they said, "You know what, you're doing so much now, and it's so impactful for clinical development and operations; let's move you up to strategy portfolio and operations, and you can support all of pharma R&D," which included 11 functions now, instead of just one. So obviously we were doing something right along the way, and now I've had a chance to leave Bayer and focus on some family and some health needs. And I decided what a better time to start my own company to try to help even more people and stay close to the patients.   Janet Kennedy: (02:44) All right. So I guess we're gonna start with a challenge. It's very comfortable, sometimes working for a very big organization with a lot of structure and benefits and support, and you've gone 180 degrees and you are now a self-employed entrepreneur. I understand that through work in innovation, you have to think like an entrepreneur in a big company. Do you think that that's laid the groundwork for your decision to start your company?   Michelle Shogren: (03:12) I think it definitely made it easier for me. It's also something that I've been considering and thinking about for a few years and had planned as a later in life activity anyways. So when the catalyst of change came, I thought, "You know what? We can just go ahead and advance it and make it faster." But you're right. It's a little scary   Janet Kennedy: (03:31) Well, there are a lot of folks out there to support you. That's the one thing that I would share with you; I did come from an entrepreneurial background, was very involved in startup weekends and innovation programs. And it is the most collegial group I have ever run into. Everybody wants you to succeed and they're all there to support. So I think even though you are pretty fresh on the entrepreneurial track, you will find that the network is there to support you. So there's a little bit of encouragement for you going forward.   Michelle Shogren: (04:03) Thanks, Janet. I needed that today.   Janet Kennedy: (04:06) Well, tell me about the company itself, Innovate In What You Do! Why call it that and what is it all about?   Michelle Shogren: (04:13) Well, you know, it's actually, it was a phrase that our corporate innovation had thrown out one time in a presentation and had used it a couple different times. And I said, "You know what? I love this phrase because of the fact, it doesn't ask you to do innovation." It says, "You know what? You can innovate in the things that you do on a regular basis." And that is my philosophy going forward. Why innovate in some other space? You wanna innovate in what you do. And it was all around the fact that how can we be better at our day to day activities, and how can we make a difference for people out there? I have a near and dear passion of patients. So of course, if I can use these powers for good of helping patients in particular, that would be great, but I really just have a purpose within me to help others. And I think innovating what you do can do that.   Janet Kennedy: (05:04) Now. I sense that this is not a quick decision that you made and that this is something you actually have experience doing. So tell me how the actual services that you would consider providing in your new business relate to the work that you did at Bayer.   Michelle Shogren: (05:21) So I have been consulting in some form or fashion for years and years and years, usually just pro bono, trying to help people out and give them encouragement or some direction when they were misguided. Either startup companies, tech companies, different solution providers would come to us and I would say, "Hey, what do you think about this or that," in order to help them figure out, how do they fit into this world of pharma? How do they communicate better? And how do they think about their users at all the steps of the way? A lot of times when we think end users, we think patients, but there's study teams, there's sites. There's so many different pieces that all have to come together to make it work. So one portion of in innovating, what you do is around innovation consulting and trying to help those same people going forward.   Michelle Shogren: (06:06) So maybe it's a pharma company trying to create an innovation function like I did, or maybe it is somebody who has an innovation function and they're struggling with taking those ideas that they have, and actually getting them to incubate and moving them on to implementation. But it could also be other sectors in the same environment or ecosystem that we sit in. So other solution providers, tech companies, startup companies, things of that nature, but it can also be innovating in your leadership style because that's another big piece that people need help with. And that goes back into innovating what you do.   Janet Kennedy: (06:42) All right. So let's go break this down a little bit and I'm gonna get myself some free consulting here. So here's our scenario A - it is a company that really never had anybody with a title of innovation; they were just a traditional company doing the traditional things, but they recognized that maybe their process was a little stale or that they were doing business as usual so much that business was maybe even declining or productivity was declining. That's a pretty big task to come in as an outsider and try to shift that. So how would you go about it?   Michelle Shogren: (07:15) It depends on what their main problem or what they think their problem is. Sometimes they don't even know. And that's where we have the first stage of Innovate In What You Do! So I call it my 'two eyes wide' approach. Cause if you look at the abbreviation for Innovate In What You Do!, it's I.I. - two eyes - and then W Y D - which also sounds like wide. So with this approach, we always have to make sure we know the problem itself and there's different innovation techniques that we can do to ensure that we are thinking about this all the way around. And we really do understand what the problem is. If they have that figured out, then we look to see, okay, well, what are your ideas that you have to fix it? Sometimes they have ideas and they just don't know what to do with it. And I can help them with that other times, they're like, you know what?   Michelle Shogren: (08:00) We just know the problem. And we've been kind of trying to come up with ideas for a long time and not have a much success. So in those cases, I can help with innovation workshops, design sprints, things like that, using my knowledge of seven different innovation methods, as well as facilitation methods that allow them to focus on the content and not how to get to a solution or a resolution. If they have the ideas and they need to figure out how do I incubate them for success? A lot of people approach things like regular projects, just typical project management. This doesn't work for innovation. You actually need to know how to do innovation project management, which is an advanced form of project management. It allows you to do iterations and pivots where a standard project management doesn't even know what to do in those situations.   Michelle Shogren: (08:46) And it also has a huge focus on the user. And how do you bring their voice in early? And how are you testing along the way to make sure you're on the right path and set up for success and building champions within the people that you're gonna have to end up selling this to later on. And if they have an idea of how to do that, and they've gone through everything, but now they're struggling to actually implement it. They've proven in a proof of concept. It makes sense. It totally has the benefits that are there and it's doable, but they're just failing at how to get that implemented either at their company, or maybe they're tryin

Hi, I'm Tom Rhoads, CEO of Spencer Health Solutions. We are proud of the amazing work that pharmacies do to support patient health and wellbeing. The need for innovation in the clinical trial ecosystem has challenged traditional pharmacies to rethink their strategy since they are the ones with the greatest access to a diverse and underserved population. On this episode of People Always, Patients Sometimes, we hear from a member of the clinical trials team at Walgreens that recently announced a corporate launch into providing clinical trial services at neighborhood store locations. Adam Sampson, head of clinical delivery operations for Walgreens is one of the team members tasked with managing the plan at clinical trial services to brick and mortar locations by the end of the year. I hope you enjoy this conversation on People Always, Patients Sometimes.   Janet Kennedy: (00:53) Hi, I'm Janet Kennedy and a member of the Spencer Health Solutions team. I'm looking forward to speaking with today's podcast guest, Adam Samson on People Always, Patients Sometimes. Adam is the Head of Clinical Delivery Operations for Walgreens, and he has a really big project in front of him. Welcome to the podcast, Adam!   Adam Samson: (01:12) Thanks so much, Janet. I really appreciate you having me on the show.   Janet Kennedy: (01:15) Well, you know, we've heard a little bit about you from my CEO, Tom Rhoads, but I'd really like to figure out how you got where you are today, especially from your startup background. So do you mind giving us a little bit of a catch up with your career and how you ended up where you are today?   Adam Samson: (01:31) Yeah, I'd be glad to. So I started my career in clinical trials as many people do, you know the cliches - to say that we kind of fell into the industry. I was a registered dietician out of undergrad and very quickly found my way into interacting directly with patients as part of clinical trials and worked as a clinical research coordinator for a few years and transitioned from there about 10 years ago into the more business side of our industry. So I worked at a couple mid-size CROs, took a very standard path. I was a regional monitor, worked in project management in line management. And then about seven years ago, my wife and I moved out here to North Carolina and I worked at Duke Clinical Research Institute for a few years. While at Duke, I did a lot of work in government funded trials in the pediatric space and completed a master's degree through George Washington in clinical trials.   Adam Samson: (02:28) And then I moved to a large pharma company and was conducting global, late phase studies. And then the pandemic hit, like many of us, I was, scrambling to keep my study going. You know, it's a hundred sites in eight countries. And how do we, possibly kind of keep operations, running lots of challenges. It was an eye opening experience around how in this industry, we really are under utilizing technology. That's no surprise to anyone we've gotten a bit better, but especially at that time, the start of the pandemic, getting even things like e-consent and other things approved was very challenging. So I decided to kind of take a leap of faith and join a very small time startup that was in the decentralized clinical trial space and had a great software product, but wanted to really build out their services and operations.   Adam Samson: (03:22) I joined the team when there were about 15 people and was with the team until series B and about 130 people. It was a really exciting couple years and enjoyed working in that space of how do we bring trials into patients homes and give them additional options to participate in trials? The one thing that remains a pervasive challenge when we're trying to do decentralized trials is that technology of course, is not enough. Sometimes patients there's a lack of trust. If things come through via email or they see them on the internet, not everybody wants somebody in their home. Sometimes people want to, go to a trusted healthcare provider, that's local to them. So when I saw this opportunity open up at Walgreens recently, where Walgreens was getting into the clinical trials business kind of light bulb went off, I'd been watching what's happening just overall in the retail pharmacy space.   Adam Samson: (04:16) And, I saw this as just an incredible opportunity to be able to take what I had learned across my career and kind of running trials in the traditional site based model, as well as, more recently working in the decentralized trials model and saying, "What if, we can take the best of both?" And we've got the technology and Walgreens has 9,000 approximately stores in the US. So 78% of the, the us population is within five miles of the Walgreens. What if we can take and plug clinical trials into that, right? There's the existing trust within the Walgreens brand and with the pharmacists that these patients are seeing every day. And we can really build a model on a really strong foundation that Walgreens has started to build in the healthcare space. So I joined the team as Head of Clinical Delivery Operations, as you said. And my role here is really to operationalize our stores, our physical footprint. How do we make sure that we have the right people process and technologies to be able to start seeing clinical trial patients within some of Walgreen's locations?   Janet Kennedy: (05:26) All right. So let's go back a little bit to joining Walgreens in clinical trials. This is very new for them and you are in a very new group at Walgreens, are you not?   Adam Samson: (05:37) Absolutely. Yeah, you're correct. So about seven months ago, actually I might even take a step farther back than that and say spring of last year, we had our new - no longer new, I guess - CEO, Roz Brewer, joined. And Roz, very quickly - I believe it was by, by fall of last year - announced that we were moving into the healthcare space with the offering of Walgreen's Health. As part of that vision that she had, right? Overall moving to a more interoperable health type framework to ensure for our patients, our main constituents here at Walgreens, that we were not decoupling them from their healthcare providers, but really helping to bridge that care continuum. Part of that offering, they realized clinical trials could be part of this, right? So about seven months ago, my boss, Ramita Tandon, our Chief Clinical Trials Officer, joined the team, joined Walgreens Health, and she began to lay the framework for what this clinical trial business would look like. What services we might offer to begin with, what are the key roles that we needed leadership to get going?   Adam Samson: (06:50) And then Ramita, a couple months ago started bringing on other folks like myself. So I've been with Walgreens - I believe this is the end of week six for me - we've had some folks on a couple weeks longer, or a couple weeks less than me, but we're coming from different backgrounds. We have folks on the product side, we have folks who are really accustomed on digital optimization and how do we engage with patients in a meaningful way, making sure that we leverage our footprint to engage with folks in diverse communities. so really exciting to be part of this new and growing team.   Janet Kennedy: (07:29) I am both thrilled and awestruck for a huge company like that to make such a big move is amazing. And I keep thinking, you're trying to move a tank when the industry needs to be on motorcycles. That might be a horrible analogy, but it's a huge company you said over what, 7,000 locations within five miles of individuals. So how do you do that? Are you cherry picking certain markets or certain states where you already have some of those type of services in play or is this literally you're starting from a blackboard and you're just starting from the very beginning.   Adam Samson: (08:09) Yeah, it's a really good question. And honestly kind of going back to when I first saw the opportunity open up here for a position with Walgreens, my first thought similar to years I think was how could this possibly happen as a company so big, right? how could you possibly get something this big done, and after a call with Ramita, it was quite clear to me that Walgreens was very much invested in this, but also that they had the leadership team Ramita included to really make this happen. I've been blown away since I've joined at not just our small core group that is starting, but outside of that, in the Walgreens ecosystem, just a lot of excitement around Walgreen's health and clinical trials in particular. So obviously we're part of this larger company, but we are somewhat entrepreneurs, right?   Adam Samson: (08:54) We are being given the opportunity to start something new within a very large business and allowing some flexibility around how we might be able to make sure that we can do that in a way that is both effective and compliant, but also nimble. Now 9,000 stores - obviously we're not gonna try and open them all at once and, and activate them as clinical trial sites. The biggest thing on my mind right now is I'm traveling to a number of different locations. We have other folks on our team who are as well meeting with stores, understanding capabilities. We have identified already a number of stores that really are the ideal kind of first step, if you will, to be clinical trial sites. So we have a few investments, key investments that we've made, and some companies that I'll talk about that will really support that.   Adam Samson: (09:47) And also, I will say that Walgreens has, we've started building out what we call health corners. So this has already been happening for a year. Plus health corners are a place where there are a small physical footprint within a Walgreen store. That's staffed with a registered nurse or a registered pharmacist. They have private health rooms and they have places to interact with technology

Hi, I'm Tom Rhoads, CEO of Spencer Health Solutions. Today we have the first of two interviews with Michelle Shogren. Michelle is a familiar face on conference stages as a 25 year veteran of the clinical trials industry. Most recently as Senior Director of Business Excellence and Innovation for Pharma R&D at Bayer Pharmaceuticals. She recently left Bayer to form her own consulting firm, but more on that in a later episode. For now, I know you enjoy this discussion on innovation in the pharma industry with Michelle Shogren and Janet Kennedy, on this episode of People Always, Patients Sometimes.   Janet Kennedy: (00:40) Hi, I'm Janet Kennedy and I'm a member of the Spencer Health Solutions team. It is such a pleasure to have Michelle Shogren as our guest on People Always, Patients Sometimes. We've known Michelle for a few years and have followed her leadership posts on social media even longer. Welcome to the podcast, Michelle!   Michelle Shogren: (00:58) Thank you so much, Janet, for having me. I'm really excited to be here today.   Janet Kennedy: (01:02) You know, you've heard a little bit about Michelle from our CEO, Tom Rhoads, but I'd really like to dig in and learn a lot more about you and your background. So can you kinda gimme the short version of how you got where you are today?   Michelle Shogren: (01:16) I think like many of us, I kind of fell into clinical research. I started out as a nurse and someone upstairs was doing clinical trials and got to go to cool places for investigator meetings. And I said, I wanna do that too. And I convinced the doctor that maybe this was also a good way to show our patients. We were forward thinking and we were looking at additional options and have another revenue stream to come into the office. And we kicked it off from there fast forward, 25 years later. And here we sit today, having had a chance to be a patient in trials, a caregiver for my family, as well as be on the site side of things as a site director and marketing director, as well as a study coordinator. And then on the CRO and pharma side, as a monitor and someone leading the studies at different levels, as well as having an opportunity to start an innovation function at Bayer.   Janet Kennedy: (02:07) Well, that really was a short introduction and I have like 10 questions just from hearing that. You know, it's interesting - you really have walked the walk. So it isn't just an esoteric type of job description; you have participated in and are living the life that many of our patients and our colleagues just talk about.   Michelle Shogren: (02:31) Yeah. I actually have personally experienced the value that can come from clinical trials as someone, as an individual who needed help and have offered this kind of help to my friends and family by helping them search for trials and finding ones that might meet their needs because it's an often missed opportunity for patients out there.   Janet Kennedy: (02:53) So when you joined a pharmaceutical company, was it a big shock? Was it very different from what you were expecting or did it feel like a natural evolution?   Michelle Shogren: (03:03) I think one of the most surprising things for me was I had always heard that big pharma was only after your money and they couldn't trust them. And when I came to pharma and I started meeting all the people who are in research and development, I found that so many had stories like me, so many were connected to someone who had some condition and they were trying to find hope for them. And so many were dedicating their lives to really make people better or bring new opportunities there. And I was blown away by the size of the hearts from these people.   Janet Kennedy: (03:33) But from a size perspective, many pharmaceutical companies, and certainly say top 20, are huge organizations. So somehow I have a hard time getting the word 'innovation' and big, giant, slow moving tank of a company in the same place at the same time.   Michelle Shogren: (03:53) Absolutely. So when we started the innovation function at Bayer, it was funny because at the time back in, I guess it was 2015, we were doing an initiative called fostering innovation cause nobody thought we needed innovation in R&D other than finding the new molecules themselves, which is very innovative - I give it that, but there's so much more involved. And we had to do an eight month long research project to find out exactly, does this make sense or not? And then we had to pitch it and say, Hey, we really think this does make sense. And we can make a big difference for Bayer for our patients out there who need it and for the rest of the world, by having this. And it was a little bit of a tough sell. They're like, "Oh, I don't know. You know, we're really regulated here. I don't know how much we can do that's different." But we convinced them to do it. And it was a little struggle to get it going. And the culture change around. It was also hard. People were used to doing everything the same way for such a long time, but it was amazing to see the changes.   Janet Kennedy: (04:50) So when you joined the innovation group and I'm looking here for officially your role there, so you moved from business process manager, process excellence, to head of innovation. That seems a very dramatic switch.   Michelle Shogren: (05:06) Yeah. So I guess my whole career, I was always innovating in what I did anyways. I was always finding things that I said, there's gotta be a better way and then posing solutions and in process excellence, a lot of that is part of what they were doing. They were looking for. How do you bring the excellence into our ways of working? So once we did this initiative, which I was a team member with six people, we needed to have a head for this new function that we had created and I applied and was lucky enough to get that role. And so that's where I made the jump, but it felt very natural for me, especially having been part of innovation for so long in different ways.   Janet Kennedy: (05:45) So I'm interested in the definition of innovation from a pharmaceutical company's perspective. I'm feeling like early on innovation was all in the lab, but now it's really every department ought to be thinking about how they can innovate.   Michelle Shogren: (06:01) Yeah Janet, you're exactly right. It was always about bringing those new molecules to life or new products on a shelf. And people missed the opportunity to innovate in how they do things and their ways to gain efficiencies, to decrease time to market, to improve their business models of their own working groups, even. So this shift that we had, a mindset of, how do you apply innovation to your day to day activities was really monumental going forward. And we had a corporate innovation function that was starting at the same time as our innovation function, which formed an umbrella of services, which was absolutely wonderful. I don't think we would've been able to really get off the ground as easily as we did without having that group there. But it became very evident that we could do a lot here. And especially when we started really focusing more on our users and their involvement earlier on in our processes, that was a big change that came about with bringing in innovation.   Janet Kennedy: (07:02) Well, I definitely wanna talk about patients in a little bit, but let's stay focused for just a second on this idea of innovation. Now we know that telehealth has been around for over 20 years, and in some cases, people are thinking like digital health just grew up in the last two years or the last three years. But when you think about the kind of things you were recommending innovating way back when in the dark ages of 10 years ago, what kind of things were you actually focusing on?   Michelle Shogren: (07:31) I remember one of the first things that we were really bringing in at the time was eConsent and we had looked out into the industry and we saw this coming and there was a lot of pushback for it and not really a sure belief that it made sense or how could we even be sure that the actual patient was the one consenting and is this really adding value or just complexity? So that was one of the early on ones. And then now we have it as optional tool for all of our studies. So a lot has changed since then. And we were able to show that, just like children learn differently - some of them need to have something that is written, others wanna hear it, others have to interact with it to really understand things - especially our informed consents needed to have the same type of opportunity for our patients.   Janet Kennedy: (08:19) Oh, that's fascinating. So then things really start to take off in the startup health space, which doesn't necessarily mean that they were funded by or driven by pharma, but it's all happening outside. So how did you go about as a company evaluating some of these new, very green ideas?   Michelle Shogren: (08:40) That's a funny thing. So while technology involves so quickly in leaps and bounds, unfortunately, big pharma companies move much slower. We're used to having several years to get a drug to market. And these tech companies are launching new products left and right. So for us just to stay up on top of what's out there and what's being offered and being able to see how does this even fit into our regulated environment? What are the risks associated? Where are the real benefits at was a daunting task? And I think most pharma companies also felt that when you add in our archaic procurement type of process, which worked well for many, many years, for many different reasons, all of a sudden it didn't work well for this new style of work, this technology business situation, we would vet companies based on how long they've been around and you know, their financial ability to stay afloat. When you think about needing the data from these tech companies for many, many years later, you're sometimes just h

The rare disease community has found strength in small numbers by banding together to share their stories. On this rare disease day, we would like to take a moment to recognize and celebrate the patient advocates and organizations that keep the focus on these underserved communities. Hi, I'm Tom Rhoads, CEO of Spencer Health Solutions. I'm proud to announce that today's podcast will be the first in a series of conversations with leaders in the rare disease community. Our host, Janet Kennedy, welcomes Eden Lord to the People Always, Patients Sometimes podcast. Eden is the founder of the Dash Alliance and 24 Hours of Rare: a Global Celebration of Rare Disease. I hope you enjoy their conversation as much as I did.   Janet Kennedy: (00:49) As we celebrate Rare Disease Day, I have the honor of bringing somebody to the podcast that, in my mind, overwhelms me with her initiatives and dedication to helping create community and conversation in the rare disease community. Eden Lord is the founder of multiple rare disease initiatives, including the Dash Alliance, the Rare Fare, 24 Hours of Rare, and Rare 72! So much happening to make it easier for folks who are in the rare disease community to come together for conversation. So on this special day, Eden, welcome to People Always, Patient Sometimes.   Eden Lord: (01:34) Thank you so much for having me.   Janet Kennedy: (01:36) You know, I actually had a chance to meet Eden a few years ago when I was invited - and I'm very proud to say - to sit on a panel during one of her virtual events. And it was a real eye opener. And, you know, we talk about imposter syndrome; I'm very fortunate in that I am a generally very healthy person. And when you are surrounded by people who are feeling the struggle of their health, every second they breathe, it sure does put things in perspective. And I was honored to be part of that program, but also humbled, by what a challenge life can be in living with a rare disease.   Eden Lord: (02:18) It definitely is, but there are so many blessings that come with the path that you walk in the disease community and so many people - like yourself - that you get to meet who really have an understanding, or want to have a better understanding, of what it's like to be a patient or raising a patient and what that path looks like and how people can help. It's the attitude that we look for when we're building our communities. And it's definitely a hard road, but it is definitely a blessed road as well.   Janet Kennedy: (02:46) Tell me a little bit about your background and how you became such an advocate for rare disease.   Eden Lord: (02:54) Well, I, myself am a third generation rare disease patient. So it started with my grandfather, passed my dad, myself, and now we are raising four kiddos who all have a chromosome microdeletion, and it has manifested in multiple rare disease diagnoses for two out of our four kids, likely going to be three out of our four kids. So been hoeing this row for a while now.   Janet Kennedy: (03:18) So from a rare disease perspective, I think the first challenge might be even figuring out that you have a rare disease?   Eden Lord: (03:26) Definitely has been a struggle to walk that path towards diagnosis. And, you know, everyone's story is different. For us, the story that stands out is our daughter who was not caught on newborn screening panels with her condition. And it was four months before we reached a diagnosis and it was a significant medical crisis that precipitated her diagnosis. So that's definitely something that we're cognizant of is the path that it takes. I think, on average, it's seven years before you actually have that diagnosis in hand. And that's if you're one of the lucky ones; a lot of the times you won't have an answer and you will go undiagnosed. And then you're at a point where you're just treating symptoms.   Janet Kennedy: (04:02) Now to be qualified - and I'm doing air quotes here - as a rare disease patient, that means you're one in what? Is it one in a million, one in 10 million? How do you qualify as a rare disease patient?   Eden Lord: (04:16) Well, it's gonna depend on a couple of things. One, it's going to depend on what country you live in and what the prevalence is. And that changes frequently, because again, we don't have everybody diagnosed that needs to be diagnosed. But here in the US, we would say it's one in every 10 or about 10%. So if you put population somewhere around 300 million, we're looking at 30 million Americans who are living with a diagnosis right now.   Janet Kennedy: (04:41) Well, that's actually quite a significant number.   Eden Lord: (04:44) It's not small. Factor in who's still waiting for a diagnosis; you know, I suspect that number climbs significantly, and those are the people that we really want to also reach as a community and support, even though they don't have a name to put with what's going on in their lives.   Janet Kennedy: (05:02) Now I have to say that I think the rare disease community is very fortunate in that you and your husband have a unique set of skills that have enabled you to say, well, not only are we gonna manage and deal with our family's health issues, but we're gonna do something to help other people. So tell me a little bit about how what you're going through as a family ended up actually becoming the profession that you've committed to.   Eden Lord: (05:28) Well, for us, it was kind of a frustration post-diagnosis for our daughter because we literally left the hospital and didn't even have a sheet of paper with her diagnosis written on it. In this instance, she suffers from something called panhypopituitarism, and the only way I could remember what that diagnosis was, was Peter Pan, and thinking, I need to go home and research the heck out of this and figure out what's what, because this was prior to smartphones and I didn't have a laptop with me, so I was kind of on my own as far as research because the hospital didn't provide that information. So for us, it's really been an opportunity and a challenge to take our backgrounds in tech and education and try to build resources and tools that make things like, you know, diagnosis support, or different medical devices, medical treatments, more accessible to people and give them a place where they feel like they're connected and engaged, even if they can't travel to a conference. Even if they're struggling to figure out where to start, we just wanna try to be that touchstone and point people towards the best resources available for the diagnosis and the situation that they're living in.   Janet Kennedy: (06:37) And what does that mean from the standpoint, if I were to go to the web, how would I find that information? How would I interact with you and your husband, Jeff?   Eden Lord: (06:45) So we have a couple of different channels right now. We have the Dash Alliance, which is kind of our main umbrella organization. And under that, we work with several different organizations to help them become more patient-centric, to amplify the patient voice. We kind of worked across the spectrum with different stakeholders in the rare disease community there. It absolutely also means that patients and caregivers in other industries, stakeholders, representatives have access to our virtual events, which for us is a goal to try to bring as many people together in the same room and kind of do away with the hindrances of travel, the cost, the, just physical challenges of managing oxygen or a wheelchair or whatever that may be, and give people a space to gather that is safe, where they know that their voices are being heard, and that they can network with other experts in the field or other patients who are just living the same or a similar story that they are are.   Janet Kennedy: (07:39) You know, it's interesting you mentioned the travel; COVID aside, it is essential that patients participate in national conferences and be face to face with the pharmaceutical medical communities. However, even before COVID, that was not an easy trick. Aside from the financial impact, it's not easy for a lot of folks who need to be seen and heard to travel, get on a plane, go to these places, and actively participate. So it doesn't matter that COVID showed up; you really needed to have a virtual event in order to support these patients.   Eden Lord: (08:18) Absolutely. And that's kind of why we launched this in the first place with the virtual platform and virtual events is because I was doing quite a bit of travel and I was attending conferences. And it was fantastic because you build really solid, good relationships with people at the events. And then you see them a few months later, but the problem was you saw the same faces in every room, no matter what side of the country you were on. It's very frustrating when you go in and you say, okay, this person would benefit, this person in my network would benefit, but I know they couldn't be here because Delta's broken their wheelchair twice, or this has happened, or this has occurred with their oxygen, or they just can't travel because it's an expense, or who's going to watch their kids if they come to advocate for their loved ones in DC. There's so many barriers that should not necessarily be there, and the virtual event platform was our way of telling everyone you're welcome here. So our first year, our theme was "welcome to the table, we saved you a seat." Because there is a place for you, and there is a place for your voice, and we will do our very best to amplify it and connect you with the people who need to hear it the most.   Janet Kennedy: (09:26) So what's the virtual event that's tied around Rare Disease Day?   Eden Lord: (09:31) So that's something that we started last year that was so much fun. We had an absolute blast. What we do is for 24 hours solid, we skip around the globe and we get updates from people who are actually in the field doing the work. So nonprofit

The last two years have been driving many discussions of the need for change in the pharmaceutical industry, particularly in the clinical trial arena. Discussions have not always led to actions, however. Hi, I'm Tom Rhoads, CEO of Spencer Health Solutions. As we look towards 2022, I believe we will record this as a year of make it so. The time for moving on from debate is here, as patients are demanding action and forward momentum on today's episode of People Always, Patients Sometimes, our host Janet Kennedy has a conversation with the new executive director of the Clinical Trials Transformation Initiative, Sally Okun. Sally brings a unique and essential perspective to CTTI, as a former nurse and VP of Patients Like Me. They have a detailed discussion of the Transforming Trials 2030 initiative, one that Spencer Health Solutions is 100% behind. I hope you enjoy this episode of People Always, Patients Sometimes.   Janet Kennedy: (00:59) It's very exciting for me to have today's guest. Sally Okun is now the executive director of the Clinical Trials Transformation Initiative. The last time I spoke with her was on the Get Social Health podcast, where she was the vice president of Patients Like Me. So interesting that we're going to have another conversation, but in a totally different role, and yet, still very patient centric. So please join me in welcoming Sally Okun to the People Always, Patients Sometimes podcast. Sally, I'm so glad to have you here. I know it's been very, very odd over the past few years in working and collaborating and life transitions, but we had an opportunity to talk to your predecessor, Pamela Tenaerts, who was the executive director of the Clinical Trials Transformation Initiative. We spoke to her pre-COVID. So now we're in the middle and maybe hopefully heading towards the end. And there are a lot of exciting things happening at CTTI. So welcome to the podcast, and I'd love to hear a little bit about how you ended up joining CTTI.   Sally Okun: (02:09) Janet, it's so nice to be here, and I really appreciate the opportunity to talk more about the Clinical Trials Transformation Initiative, and certainly the shoes that Pam left are large, and I'm still trying to find ways of filling them, and I think that's gonna take some time. How did I get to my current role? Well, interestingly enough, I had left Patients Like Me in 2020 and I was working on a variety of interesting projects at the time across the health spectrum. You know, I was consulting on a variety of different things and also enjoying some newfound freedom of semi-retirement, which was an interesting experience for the first time in my career. You know, by about mid 2021, maybe in light of the time that we were all spending at home as opposed to spending out enjoying each other's company socially or professionally, I started thinking more and more about the next chapter of my professional career.   Sally Okun: (02:57) So it wasn't long after that, that I was approached about the position of executive director at CTTI. And frankly, initially I didn't see the fit for me. You know, I've not spent my career in clinical trial work. I've spent a lot of my career in research and clinical research with patients and others, but hadn't really focused specifically on clinical trials and the clinical trial enterprise. So at first I really wasn't exactly sure it would be a good fit for me. However, after talking with some trusted colleagues and others, including the chairs of CTTI's executive committee, I was truly intrigued by the organization's impressive portfolio of work. I really hadn't been aware of just how much they have done over the last 15 years, 14 years, in terms of contributing to the improvement overall of the clinical trial experience, not just for participants, but for investigators as well as sponsors and others.   Sally Okun: (03:48) So, you know, I then looked a little more closely at the transforming trials vision, and I thought, you know what, here's an organization that is setting goals as a priority to be seeing clinical trials as patient centric and easily accessible as its number one pillar. And I thought, well, you know what, I think maybe this fit might be just right. So I spent a bit more time thinking about it and then made the leap in October of just a couple months ago - just hit my two month anniversary.   Janet Kennedy: (04:13) You mentioned something just now about the Transforming Trials 2030 program. What is that?   Sally Okun: (04:20) Well, you know, a couple of years ago, again, under Pam's leadership, they discussed the need to set some milestones to really stop sort of talking about improving clinical trials, but actually putting some stakes in the ground or pillars up to suggest that we need to have a goal for that transformation; that have to have things that we're working towards as a community in order to be able to achieve a better experience for everyone involved in clinical trials.   Sally Okun: (04:49) So what came out of that initial discussion was a set of five pillars that actually articulate quite specifically what we believe clinical trials should be like by 2030. And each one of them has a host of different objectives beneath the goal, that actually really describe how we might ultimately get to the point where we can say, "Yes, indeed, we have clinical trials that are now patient centered and easily accessible," for example, for the first pillar. But each one of them is complex. Each one of them requires a lot of understanding of what needs to get unpacked to better get at the kinds of things that need to get changed. So we are in the process right now of beginning a strategic plan for doing just that: taking each of the pillars and starting to think about how will we know that we've achieved success on this pillar? What will be the metrics we need to start thinking about for those.   Janet Kennedy: (05:42) Alright. So let's do some metrics here. What are the other four pillars?   Sally Okun: (05:46) Number one, as I said, is that clinical trials will be patient centered and easily accessible. And if we don't reach that, then some of the others are almost irrelevant. So that is actually the place to begin. But then the second one is that they're fully integrated into health processes. So that gives us an opportunity to really begin thinking about the continuously learning health system, where we embed within the care environment, the opportunity to research and learn, and then put that learning back into the care environment again, so that we have a full circle loop. The third one is that they are designed with a quality approach, and CTTI has done an incredible amount of work on something called quality by design.   Sally Okun: (06:27) It has a whole set of recommendations around how to determine how the quality of your trial based on a variety of different things - whether it's your recruitment numbers or other aspects of what the trial efficiency looks like. The fourth one is that clinical trials take full advantage; they maximally leverage all available clinical and nonclinical data, including data that's collected now with digital technologies to ensure that we max - minimize - the collection burden of getting data and actually improve the opportunity to make data more meaningful and impactful as part of the trial experience. And then lastly, although not least, is that clinical trials contribute knowledge to how to prevent diagnosis and treat disease. And that becomes something that's an objective around improving population health and the health of our people.   Janet Kennedy: (07:12) Okay. Those are very large topics, very large goals. Who is the Clinical Trials Transformation Initiative? Who's going to do this work?   Sally Okun: (07:23) We are a group of people who have come together within the organization that includes project managers who oversee our projects, and then strategic and engagement and communications team that oversees all of our communications with our steering committee members in our member organizations. So to give you an idea of what that structure looks like: we have a steering committee that includes about 80 organizations who are actually members of CTTI, Clinical Trials Transformation Initiative, and they commit to have a person assigned as a steering committee representative. So as we begin to think about tackling a new topic or a new initiative, we go to the steering committee and we make available to them the opportunity to join the project team. That project team then starts to create the infrastructure for accomplishing that project, which can include, first of all, the project plan and setting the aims and the objectives.   Sally Okun: (08:17) In many ways, it's creating a research study for the topic under consideration with that. Then they set out to consider what's the literature already say? Who are the expert people we need to be speaking to? So there's a lot of qualitative interviewing that goes on as part of the social science team from the Duke Clinical Research Institute; that is a connection for our team to be able to work with. And then the findings from those interviews begin to shape the recommendations that will come out about a particular topic area. For example, thinking about trials in healthcare setting; what do we need to know about the various barriers for those and the opportunities for those as well as the current landscape within which the clinical experience might or might not include participation in research activities? So there's an entire project management approach that is put on any topic we tackle.   Sally Okun: (09:08) The problem with it; it's an incredible opportunity for us to be able to map out these topics and really think about the issues as a group, but it's a labor intensive and time consuming. It can take up to two years to complete some of our projects because they are so intense in terms of diving deep in

Spencer Health Solutions was founded knowing that pharmacies are an integral part of the patient's health journey. Hi, I'm Tom Rhoads, CEO of Spencer Health Solutions. Pharmacy partners are key to supporting Spencer in patient homes for clinical trials and commercial programs, especially specialty pharmacy partners. To dig deeper in the specialty pharmacy and learn about some of the challenges and changes coming to the industry, we invited Sheila Arquette, CEO of the National Association of Specialty Pharmacy to come onto the podcast. I hope you enjoy this conversation with Sheila and our host, Janet Kennedy, on People Always, Patients Sometimes.   Janet Kennedy: (00:43) We are honored to have as our guest, Sheila Arquette. She is the president and CEO of the National Association of Specialty Pharmacy. She joined NASP in 2017 and has been focused on increasing the value of the NASP membership. Some of her initiatives under her tenure have resulted in a substantial increase in their membership, also in member benefits and value, and she's expanded the annual meeting at expo that's held every year in the fall. Specialty pharmacy has a critical role to play in helping patients live their best lives, and I'm thrilled to have her on the People Always, Patients Sometimes podcast. Welcome Sheila!   Sheila Arquette: (01:24) Thank you so much, Janet. It's a pleasure to be here with you today.   Janet Kennedy: (01:27) Now I gave a very quick high level look at your background, and I'd love for you to fill it in a little bit more for us. Your relationship with NASP didn't start in 2017, did it?   Sheila Arquette: (01:37) No, it didn't. I started in a volunteer capacity with NASP way back in 2013, in 2014, and I started off volunteering to serve on the membership committee. And then I was asked to co-chair our government affairs committee and just really loved the mission of this organization and the incredible group of people that were so passionate about specialty pharmacy and patient care. And so I started to get more and more involved and then helped with our first ever specialty pharmacy law conference. And one of our board members, Rebecca Shanahan, it was her vision to establish the Specialty Pharmacy Law Conference and provide continuing legal education credits for lawyers and specialty pharmacy, because it was really challenging to try to find those credits. And then she was also incoming president of the NASP board. And so she knew at the time that she was gonna need help running the organization. And so that's when they reached out to me and asked if I would consider coming on board, full-time.   Janet Kennedy: (02:38) You know, Spencer joined in the not-too-distant past, and we're now members of NASP, and one of the first and frequent emails I get is about what's happening in Congress. And that is amazing, all the work that you're doing there. So what are the big topics that you're covering in the political affairs and government committee?   Sheila Arquette: (02:58) We're very, very focused on ensuring that we have a competitive landscape in a fair imbalanced marketplace. That specialty pharmacy patients have access to the medications that they need and appropriate access, meaning that it's the right drug for that patient at the right time during their clinical treatment. But we also want it to be from the pharmacy of their choosing, right? So we wanna make sure that our pharmacies have access to networks. That reimbursement is fair, and it's not being used to keep them from participating in different payer networks. And we wanna make sure that patients have access and choice. And we're very focused on pharmacy DIR fee reform, right? And ensuring that pharmacies are compensated or paid for their performance and that, you know, that we're all driving to the same end goal, right? We're looking at enhanced clinical outcomes. And we also wanna decrease total cost of care because it's a huge issue, right? We don't have an infinite amount of money or resources.   Janet Kennedy: (04:01) And do you feel like the pharmacy section of healthcare has got a voice that they need to have, or is this something you're trying to build and sustain to a higher level?   Sheila Arquette: (04:13) So with respect to specialty pharmacy, it is something that, you know, we had to start from ground zero, right? When we would meet with members of Congress or, you know, different offices of the administration, or even CMS, when we would talk about specialty pharmacy, there really was a huge learning curve and a lot of education that we had to do because fortunately not very many folks still to this day are treated with specialty medications. It's only still around two or two and a half percent of the total number of prescriptions that are dispensed. So when you would talk about pharmacy, the image that comes to everybody's mind is that corner drug store, right? Your community pharmacy in the middle of town, or that you've always frequented. Then when you started talking to them about, you know, specialty pharmacy and the nuances and the distinctions, there was this kind of scratching of their head. Like, no, not sure what they're talking about. So we really had to start and lay the foundation about, you know, this is what specialty pharmacy is. This is why it's different. And pretty much, this is why you should care about it.   Janet Kennedy: (05:11) Is that NASP's primary goal, or do you have multiple goals for the organization?   Sheila Arquette: (05:17) We have multiple goals, Janet. We have four foundational pillars. One of which is advocacy, but then we also are focused on education, certification, and then also on the membership. And what I mean by the membership is providing all of the resources, the tools, fostering the connections and the networking that leads to enhanced patient care, right? It takes a village, right, to make a specialty pharmacy patient. So we wanna make sure that our members have at their fingertips, everything that they need to better care for their patients.   Janet Kennedy: (05:51) So I am a layperson in the conversation. I don't come with a pharmacy background. I've more of a marketing background. So I'm gonna ask a couple of questions because I know there are a few other folks out there like me who need to ask this deeper level of question. So a 'specialty drug' is something that I couldn't walk into my corner drug store and get?   Sheila Arquette: (06:10) Well, and this is a challenge because we don't yet, still we don't have an established definition of specialty drug. And typically what happens is it's the payers, right? It's the health insurance companies, it's the PBMs. They determine what medications are in their specialty drug program. So back in 2016, members of Congress approached NASP and asked for us to put forth some definitions that they could use as a reference. So with respect to specialty drug, what we focused on was complexity. Specialty medications are complex. It's either in the way that they're administered, dosed, their side effect profile; it could be in the insurance coverage process, that's required to gain approval for patients to receive these medications. There can also be financial barriers that have to be overcome. So typically specialty medications are complex. They may have to be stored or handled in a particular way. Oftentimes you can't get what we consider a specialty drug at your local community pharmacy. However, there are always exceptions to the rule, and as the payers continue to redefine what medications they include in their specialty drug program, sometimes then all of a sudden, you know, these medications are available at your community pharmacy. But typically no, they're restricted to these pharmacies that, you know, manufacturers have selected to work with because of the capabilities, the inherent capabilities that specialty pharmacies have.   Janet Kennedy: (07:38) So how does it work out numbers wise? Is there a specialty pharmacy in everyone's community?   Sheila Arquette: (07:44) No, there's not. And typically most of these pharmacies are closed door. And what I mean by that is patients don't walk in and out, right? There's not a transaction at a pharmacy counter. Most of these patients are managed remotely. You may have a specialty pharmacy with a central fill location where all of the prescriptions are dispensed. And then they have an enormous patient service center type area where all of the patient management services are carried out. So we have folks that will take care of the insurance verification and the benefits investigation, and helping you to understand as a specialty patient, what is your copay for this medication? And if you can't afford it, what are the available options? Then we also will have folks reach out to you to schedule your delivery of the medication and help you with refill reminders. Pharmacists and nurses will also counsel patients, right?   Sheila Arquette: (08:33) Review the medication, take a detailed history, talk about your disease process, make sure that that patient understands what it is they're being treated for and why it's so important to take this medication as prescribed. And what do you do if you have side effects and what are the side effects to look out for? And then specialty pharmacies will also help to coordinate all of this information and interact with other members of the patient's healthcare delivery team. Also, caregivers are really important too. And specialty pharmacist and pharmacy personnel will also interact with those caregivers. So oftentimes no, there isn't going to be a specialty pharmacy in your community, but you will have access to the medications that you need and the support services that you need.   Janet Kennedy: (09:13) Here I was gonna ask my next question about "Gee, are specialty pharmacies patient centric?" and that actually sounds like the very definition of a specialty pharmacy.   S

Pharmacy companies are an important part for Spencer Health Solutions, and pharmacists are an integral part of fulfilling our mission to serve patients in their homes. Hi, I'm Tom Rhodes, CEO, Spencer Health Solutions. We have had the opportunity to bring patient pharmaceutical and digital health thought leaders to previous podcasts. However, today we're speaking with our first pharmacist, digital health advocate and analyst, Timothy Aungst, also known as the digital apothecary. He joins our host Janet Kennedy for an insightful conversation and a call to action to the pharmacy industry on the People Always, Patients Sometimes podcast.   Janet Kennedy: (00:45) Welcome to People Always, Patients Sometimes. I'm very excited that today's guest is Timothy Aungst, the digital apothecary. He is an associate professor of pharmacy practice and also a clinical pharmacist. Timothy, welcome to the podcast.   Timothy Aungst: (01:03) Thank you, Janet, I'm really excited to be here today to talk about, you know, all this stuff that's been going on.   Janet Kennedy: (01:08) It's been a crazy couple of years and that's one of the things that I wanted to talk to you about. I found you because I read your primer, "Digital Health Primer for Pharmacists." You published it in February of 2019. At that time you wrote, "after being involved in the digital health space for almost a decade, I can say with complete sincerity, that the topic is still relatively out of the realm of the general pharmacy profession grasp." Okay. You put a challenge out there. 10 years you've been involved in digital health and you're saying pharmacy is still not up to speed. Would you say that that's true two years later?   Timothy Aungst: (01:52) Yes. I still would stand by that. I would argue that we have seen entrepreneurship within the pharmacy profession gravitate towards digital health at large, but I would also conversely say that as a profession for pharmacists, we have not really actually actively engaged in this area. We still lack a large number of educational roles and trying to get people aware of the space. There is a lack of discussion around it. Most of our public organizations that provide guidance on what our next steps for the profession don't really think about it in, I think it's for that reason, I still would say the gap is there. Now that is changing. I would say that there has been a semi call to action amongst several pharmacy organizations, whether it's say PHA, ACP and several others who are now trying to get the profession up to speed on what digital health is.   Timothy Aungst: (02:44) And that's been a big focus of mine serving as so-called subject matter expert or key opinion leader to help get information out there regarding the topic. But very, at this time it's quite topical just because we are not actively highly engaged with it. I would probably say we're maybe like two or three years behind other healthcare professions, such as the medical community. The American medical association has an active digital health component that they've been pushing, I would say probably for about two or three years at this point, have reports coming out, organizations associated with it. And I think pharmacists have to play catch up to that. And depending on key stakeholders at this current time that may or may not go fast or may go slow. And that's one thing I'm actively keeping my eye on.   Janet Kennedy: (03:27) Well, I'm curious about whether pharmacists or the pharmacy is even included in some of this digital health development.   Timothy Aungst: (03:36) I'm always a person who would've actually separate the two. The pharmacist and the pharmacy, I think are no longer synonymous organizations. You don't need a pharmacy to have a pharmacist. I think it's going to be what we see in the 2020s or 2030s at this point. I think those two things will actually diverge and that will just come down to logistics and also some legal parameters I think people are pushing right now. Technicians will be empowered to take on most of the stuff on site and pharmacists will probably feel remote. That being the case then to accomplish that, and also to get pharmacies up to speed where healthcare is going, I think the pharmacy businesses will engage in digital health activities. For good or for bad. The big news right now, right, is that Elizabeth Holmes is in court with Theranos. And if we look at Thernos, who was one of the biggest backers? That was Walgreens. And I always looked at what happened with Walgreens being burned there is a reason why they actually had a huge number of digital health initiatives back in the 2010s. And I think they pulled back because they got burned so heavily.   Timothy Aungst: (04:31) In the meantime, we see, let's say a business like CVS Health going down a whole vertical pipeline. Now we got Aetna involved, we got long-term care stuff, we got them pushing to go into kidney disease with dialysis treatments. They are partnering up with digital health companies all over the place. One of the biggest ones, for example, was Sleepio for a digital therapeutic that they've been piloting out there, which has actually initial positive results I'm actually reading through right now. So some of these companies are more involved than others. And I think it's going to come down to, you know, what assets they have to really push that. Whether they see the market going a certain way and they want to meet consumer demands because they are also aware of that, with the changing dynamics within healthcare, pharma is going down the digital health path. Payers are looking at this. Employers are looking at this and even the big tech companies are pushing this stuff out there. So are they going to let other people dictate to them the incumbents of what to do, or are they going to be the drivers themselves? And I think that's going to be something that's going to be interesting to watch.   Janet Kennedy: (05:34) Well, so you mentioned the need to separate the pharmacist from pharmacy. So speaking from the individual's perspective, do you hear pharmacists talking about wanting more and better tools to be able to help their patients or are they just basically the Amazon employee at the warehouse where they're just cranking out the work?   Timothy Aungst: (05:57) See, I used to think we're still on the razor's edge between two possibilities: one was pharmacists finding some kind of clinical services they pay for under some, say, value-based care agreements. And by providing clinical services would receive renumeration that would allow them to be so-called clinicians in health care. Or the other one was being fully remote and being consigned to these activities, to these virtual workshops where they basically just review medications that have been turned out by Amazon or other companies like true pill, et cetera. There might be a third path, which is a mix between the two. And that's kind of where I'm more leaning towards right now from a pharmacist perspective. Yes, there is a huge interest in digital pathway as a means to basically call back clinical services and kind of like be able to provide services that could then actually have objective feedback in terms of what they did, that they could then bill for services and, you know, make money from it.   Timothy Aungst: (06:51) But I don't know if that's going to be enough at this current point to actually say, "oh, well, you're now a remote clinical pharmacist that overviews these data's on people's adherence or their information on disease states, and you get this much," because one of the issues is actually even though they're like remote patient monitoring services that we're seeing CPT codes being pushed up by CMS and such, they're in the physician still. So the pharmacist almost has to still be partnered with someone else in order to receive the renumeration. And then at that point in time, you know, it was kind of like, what slice of the pie are you going to get? And I think that's really what concerns me is that from a healthcare perspective, the pharmacist is still really trapped in their other engagements, which has traditionally held us back. And I think this is because we again have very little stakes in digital health and we were coming to the table kind of late. The other people have already kind of figured this out and have already been making inroads. Making propositions to other organizations to get themselves ahead. And we're kind of almost, I don't want to say asking for crumbs, but we're going to have to really do our best claw back some of this stuff for ourselves. And that's going to delay, I think those aspects.   Janet Kennedy: (07:56) And do you see the trade associations being the ones that should be leading this charge?   Timothy Aungst: (08:01) I think the trade associations unfortunately all have different stances out why they would want to engage in this stuff. The trade associations value pharmacists to different extents. And within a pharmacy community, we know with the alphabet soup of different pharmacy organizations, they don't all have one central voice. They don't all have one central take on what is the role of pharmacists. I've heard some people say we have a pharmacist practice at the top of their license. I don't know what that means. To be quite honest, whenever I hear that I kind of have to roll my eyes, because what is the top of their license? I think in their eyes probably is just, you know, sending off for prescriptions. So that's great. That's what they see as the business that they have to focus on. I could see definitely some trade associations making an argument to empower technicians, to move back at scale back the role of the pharmacist as a, you know, as a rate limiting step in terms of evaluating prescriptions and getting them out the door.   Timothy Aungst: (08:53) And also because there are high cost margin right there, and that would reduce the

What happens to a person who finds out they have a rare disease? When you're diagnosed with a condition that affects one in a thousand or one in ten thousand, where do you turn to find information, help, and support? Recognizing the need for that resource, a team of dedicated individuals on Rare Revolution, a magazine dedicated to spotlighting the rare disease community. Their goal is to bring about a dramatic and wide reaching change in conditions and attitudes for the rare disease community. We are honored to bring Rebecca Stewart, CEO of Rare Revolution Magazine, to the podcast. Her conversation with our host, Janet Kennedy, truly reflects our philosophy - People Always, Patients Sometimes.   Janet Kennedy: (00:50) Welcome to People Always, Patients Sometimes. Today we're really taking a patient perspective and we're taking a unique look at patients who really need some attention right now, and those are the patients that reflect the rare disease community. With me is the CEO and founder of Rare Revolution Magazine, Rebecca Stewart, and we're going to be talking about the thousands of patients who may not be getting the attention they deserve and need - those with rare disease. Rebecca, welcome to the podcast.   Rebecca Stewart: (01:24) Thank you very much for having me Janet. It's a pleasure to be here.   Janet Kennedy: (01:27) I would like to think that we really are having a Rare Revolution, but is yours a call to action or a reflection of what's actually happening in the rare disease community?   Rebecca Stewart: (01:39) So to give you some context, there are over 7,000 known rare diseases, which affect globally 350 million people. So we're the biggest, rare community that there is. It's a huge number of patients, but of course, individually, each individual disease affects a small number. But globally it's a huge health crisis.   Janet Kennedy: (02:05) How about how the community is being served? Is this something where individual patients are truly having to learn how to advocate for themselves, or are they growing together as a community to speak and support each other?   Rebecca Stewart: (02:20) I think there's both. So, as a community, the rare disease community have both really good individual disease communities, mostly. Of course, there are those rare diseases that are so rare that there aren't support groups, there aren't patient communities. And so those people find themselves really quite isolated. And then collectively the rare disease community comes together to really help influence policy, to help influence healthcare, to help push for things like interest in orphan diseases, from pharmaceutical companies and research, and to support each other because individually, the individual nuances of each rare disease, whilst those might be very different, collectively people with rare diseases tend to have very similar problems; that's access to expertise, access to healthcare, education, meaningful employment, social services. These are all shared problems. And so they can really support each other across disease. So, you know, really disease, agnostic support as well as the absolutely amazing job that individual support charities and organizations provide on a specific disease level.   Janet Kennedy: (03:34) So what is the Rare Revolution Magazine role in all of this? What is your mission and how do you become involved in this community?   Rebecca Stewart: (03:43) In 2012, my sister, who's also my co-founder, and I actually began a disease-specific support group called Teddington Trust, which is to support families affected by the ultra rare disease Xeroderma Pigmentosum. And through that, what we found was a real gap in our ability to raise meaningful awareness for the work that we did and for the condition and that it was done in a way that we felt was sensitively dealt with and valuable to us as an organization and a community. And we thought, well, we can't be the only people that struggle with this. And of course we discovered we weren't. So Rare Revelation Magazine was founded out of a real gap in rare disease education and awareness materials. And what we wanted to do was to become a platform where it didn't matter whether you were a patient, a caregiver, an advocacy leader, someone from industry, someone from research, or a healthcare professional: right across the ecosystem, you could come to us and both be heard, talk about the work that you're doing, but also gain value and find information and education in what you read. But it was really important to us that the tone, the design, the curation was something that added value and was sensitive to people's lives. And that's really, our mission is to be a source of awareness, education, signposting, so that people can find each other and really be a platform to elevate the voices of our communities. So we don't speak for our community. We're just here to elevate the voices of the community that exists and already do a great job.   Janet Kennedy: (05:24) Well, you know when I look at the magazine, which is digital only, is that correct?   Rebecca Stewart: (05:28) It is. We do occasionally print them if we're going to conferences in pre-COVID times, but you know, it's a digital e-reader and then a website as well. So web based content, and then the magazine itself has produced as a digital e-reader.   Janet Kennedy: (05:42) Well, kudos to you. It is a fabulous looking magazine. It is so professionally produced. The photography's incredible. Your design is great. This is not a newsletter typed out in your backyard. This is an amazingly beautiful magazine.   Rebecca Stewart: (05:59) Thank you. And you know that was actually really important to us that, I think when you have a rare disease or a chronic illness, you spend a lot of your time online carrying out research. And it was really important to us that when we produce this magazine, it was something that told a story, but it was really, it was, we spent enough time on our screens. We wanted something that was really lovely to look at that it was an enjoyable process. Because to us, that means people spend longer in the publication, looking at things and reading articles that they might not normally read and that we have found to be true. Certainly our average time that people spend on the magazine is much longer than you might expect people to spend in digital content. And I think that is down to the amazing job that our two designers and also to our editor and chief creative director, Nicola Miller, who curates and has a hand in overseeing all of the publications that we do.   Janet Kennedy: (06:58) Tell me a little bit about the process of putting the magazine together. You've mentioned over 7,000 rare diseases; well you'd think it would end up being something as big as the Encyclopedia Britannica. So how do you curate and where do you get your content and how is it put together?   Rebecca Stewart: (07:14) Well, we do between three and four core additions of the magazine each year. And then on top of that, we do a number of spotlights additions, which tend to be smaller and more focused. And we will do three of those perhaps in a year. And then we have our online content as well. And when it comes to what themes we're going to tackle, we keep quite a close ear in the community. We see where people are talking, what they're talking about, what the current challenges are. But also people will reach out to us and say, you know, we have this condition, have you ever thought about covering it? And so we'll put together an editorial calendar over the year that are topical things that are going on in the community or areas where we feel we can really reach out to the community and add value.   Rebecca Stewart: (08:03) For example, we have just done an edition on inclusion, equity, and diversity. Prior to that, we have looked at rare bone diseases or rare cancers, rare kidney diseases. You know, what we're trying to do with those core additions is have a very broad topic that we can encompass many rare diseases in under that. Obviously we're never, you know, Nicola and I will not live long enough to cover every rare disease at 7,000 plus, but we're giving it a good shot and we're doing it as best we can with these topics. And then with our spotlight additions, those really allow us to get very, very detailed on very specific subjects. And generally those additions are brought to us by sponsors who have got a particular need to talk in an area and they want to really shine a light on something. And so we will work with those sponsors to produce those spotlights.   Janet Kennedy: (08:56) And how do you vet out in essence, the difference between say paid and editorial content?   Rebecca Stewart: (09:02) In our core additions, we have an editorial sponsor, but that editorial sponsor only has any control over the articles that they give us for that particular edition. Everything else is the copyright of the contributor and of Rare Revolution. So they don't have any editorial control there, which really allows us to make sure that the conversations that we're having are genuine and are not influenced by a sponsor. And even when we do our spotlight additions, which tend to be solely sponsored still in that situation, the sponsor does not have editorial control. And what we generally do in that situation is we will carry out all of the patient patient group and healthcare professional interviews; which again, allows us to make sure that we're asking appropriate questions, that we're keeping all of that content compliant and valuable. And we understand what our readers are looking for. So, you know, we very much have an input in any brief that's being laid out for the spotlight edition. But in our core additions, over 90% of the content will be from individuals and charities. There's very little paid for content actually makes its way into those additions.   Janet Kennedy: (10:16) Which makes it even

Decentralized trials are here to stay, says Dr. Isaac Rodriguez-Chavez, our podcast guests today. Hi, I'm Tom Rhoads, CEO of Spencer Health Solutions. We are delighted to have Isaac join the podcast to discuss decentralized clinical trials and why pharma companies should embrace digital technology for the benefits of patients. A former FDA officer who was instrumental in the review and approval of digital health technology in clinical trials, Dr. Rodriguez-Chavez is now the Senior Vice President for Scientific and Clinical Affairs and Head of Strategy for Decentralized Clinical Trials for Icon. I hope you enjoy this conversation with our team member, Janet Kennedy, on the People Always, Patient Sometimes podcast.   Janet Kennedy: (00:46) I can't tell you how thrilled I am to have finally landed Isaac Rodriguez-Chavez as a guest on the People Always, Patient Sometimes podcast. He is an industry leader that is someone that everyone listens to, and it's really exciting to have him here today. He is formerly the FDA officer focusing on clinical research methodology, regulatory compliance, and medical policy development, and recently joined the private sector as the Senior Vice President of Scientific and Clinical Affairs, Head of Global Center of Excellence, DCT Strategy for PRA Health Sciences. So he's got a foot in both camps and it's going to be a very exciting conversation. Isaac, welcome to the podcast!   Isaac Rodriguez-Chavez: (01:30) Thank you, Janet for having me and all the listeners, I am delighted to be here with all of you, sharing some thoughts on information that is really, I hope, pertinent for all of us.   Janet Kennedy: (01:43) You have been very active as an FDA member and as a member of the PRA Health Sciences in promoting and discussing decentralized clinical trials, and it's time is now. However, for the one listener who might not realize who you are, I'd love to explore a little bit of your background, what work you did with the FDA, and then what made you decide to come over to the private sector. So can you tell me a little bit about your background and how you came to be where you are today?   Isaac Rodriguez-Chavez: (02:14) Absolutely. I'd be happy to do so. The FDA, I was a senior officer for clinical developing medical policy and working internally. Also leading a program on decentralized clinical trials and supporting the modernization of clinical trials in general, including the implementation of digital health technologies in modern clinical investigations. The FDA is an amazing place. And my colleagues there are really wonderful, friends more than colleagues, because the work that we do really matters and impacts the entire nation. Essentially, I was a professional consulting internally and externally on decentralized clinical trials implementation of decentralized clinical trials. I believe in the modernization of clinical research. So I am a basic scientist by training with specialties in immunology and biology, but I became a clinical scientist also by training and experience over 20 plus years ago, conducting clinical trials, doing clinical research, being a sponsor, being the auditor, being essentially in multiple capacities, engage in investigations.   Isaac Rodriguez-Chavez: (03:32) So for me, my passion is really what I discover is the direct impact on the patients and their lives, and clinical research, along with the infectious diseases, which is at the end of the generic or the bigger umbrella where my expertise lands, are the things that I like and I enjoy doing. Direct impact to patients and their lives in clinical investigations, with novel products and in infectious diseases and even in vaccines, which are also a love of my life. And that's what I did at the FDA. And then I transitioned to PRA Health Sciences to lead the strategy of a newly created global center of excellence for decentralized trials. The idea really for me was about to transition from the regulatory world and diversify into the real world in how the trials are conducted and essentially lead the strategy and implementation. We are in a transformative time in clinical research because of all of these modernization efforts that are happening building decentralized trials.   Isaac Rodriguez-Chavez: (04:43) So I ended up believer in doing things better, and for the benefit of the patients, and PRA offered me essentially the venue to do so. So I am truly excited to be here with a group of professionals that are outstanding and they believe as strongly as I do in providing essentially end to end solutions and creating partnerships with multiple vendors, so that together and in unison, we can deliver the best in class and in the field in regarding decentralized clinical trials enabled by digital health technologies. And for the benefit of the patients and with efficiencies on the trials and the steps that can represent benefits for the sponsors, for the science - benefits for everyone engaged in the clinical investigation. This is the place, right now, as you said - now is the time, now is the place - in which everyone benefits. It's positive. There are multiple advantages for everyone, so, excited to be here. And my journey has been the one of love and passion for what I do and believing in doing things better for our people everywhere.   Janet Kennedy: (05:55) I'm very interested in the three-legged stool of the organizations that are involved in clinical trials. You have, of course the FDA, you have the pharmaceutical company, and then you have the groups like PRA, the clinical research organizations, that really are much more involved in the implementation of the clinical trials. What I'm curious about is, is that stool steady? In other words is one leg, a lot shorter than the others? Are all three of these organizations moving forward at the same speed? Or do you find that maybe the CROs might be ahead of the game and trying to get pharma to catch up a little bit with decentralized clinical trials? Or are you finding that partnerships with pharmaceutical companies - that everybody is about at the same place?   Isaac Rodriguez-Chavez: (06:42) I could say I see these more as a round table with multiple legs and multiple parties being engaged. One thing that is happening right now that is historically never seen is a different type of collaborations and the level of collaborations and the frequency of the collaborations between multiple entities is unprecedented. The COVID-19 pandemic really has put everyone on the discussion table to find ways to continue the traditional clinical investigations that were happening when we were hit by the pandemic. So the way to do it was the implementation of elements of decentralization. And with that, the creation of hybrid decentralized clinical trials in which some elements of decentralization are happening, and others are still happening in the brick and mortar site - the traditional clinical research site. Now days. And what we're seeing is essentially the business beyond the pandemic. The transformation that is happening really is permanent, is driven by multiple factors, and the engagement is at all levels.   Isaac Rodriguez-Chavez: (07:58) So the industry, the regulatory agencies, the clinical research organizations, such as PRA health sciences, are really making huge efforts to work together, to drive this transformation. Of course we are in a learning curve and a learning process. All of these happened suddenly. And though we are embracing decentralized clinical trials and technologies to modernize clinical research, we're still in the transition time, we're still in the changing of how to do things. Everyone is not at the same level. Everyone's not at the same speed. There is a spectrum in terms of the uptake and implementation and racing, decentralized clinical trials and technologies; that kind of goal. Everybody's thinking about how to do it in the pharmaceutical industry, in the biopharmaceutical industry - small, mid-size, large size pharmaceutical industry - but the implementation is different. So there is a whole spectrum that can go anywhere between 20% to up to closer to 80%.   Isaac Rodriguez-Chavez: (09:08) And the surveys show that information. But everyone is having conversations and the projections are very strong, that decentralized clinical trials really will become the mainstream of clincal research within the next two to five years. So this is happening, it is irreversible, it is transformative for everyone engaged in the clinical investigation. And the conversations are happening and the collaborative work is happening between multiple parties. So I not only see the regulatory agencies and the pharmaceutical industry and the CROs; I see the vendors, I see multiple vendors engage. I see the science engaged. I see the patient advocacy groups engaged. It's really multiple entities engaged in the conduct of these kinds of investigations. And it's for the benefit of everyone, as I said.   Janet Kennedy: (10:06) So you're saying you really don't think we're going to go backwards to the way it always used to be done - that people have learned, not just that patients want a new way of participating in clinical trials, but this is actually a better way?   Isaac Rodriguez-Chavez: (10:21) Pretty much. Though decentralized clinical trials are not the universal solution for every single medical product that is going to be tested, it is very common to see decentralized clinical trials in multiple therapeutic areas, and in all phases of the clinical investigation. There are multiple reasons and benefits for doing this. Essentially, the potential for enhancing convenience to the participants is a main driver for the implementation of decentralized clinical trials, the opportunity to improve recruitment and retention of participants, participants in the ecosystems where they live - meaning their homes, their towns, their cities, their countries - and not h

Understanding the patient journey is an important part of clinical trial development. Hi, I'm Tom Rhoads, CEO of Spencer Health Solutions. On today's podcast, we welcome Shazia Ahmad, Senior Director of Patient and Physician Services at UBC. She is a thought leader in the space of patient communities and supporting the patient throughout the clinical trial process. I hope you enjoy this conversation on People Always, Patient Sometimes.   Janet Kennedy: (00:31) Hi I'm Janet Kennedy and I am your host for People Always, Patient Sometimes, a production of Spencer Health Solutions. I am really looking forward to a conversation today with thought-leader Shazia Ahmad. She is the Senior Director of Patient and Physician Services at UBC. Welcome to People Always, Patient Sometimes, Shazia!   Shazia Ahmad: (00:52) Thank you Janet! Thanks so much for having me today.   Janet Kennedy: (00:54) I've been trying to track you down for a conversation for actually a number of years, and I'm so glad I finally captured you today.   Shazia Ahmad: (01:03) Yes, absolutely! So excited to be on.   Janet Kennedy: (01:06) Well, I want to be talking to you a lot about the work that you do specifically as a director of patient and physician services; but first I think we need to set the stage. Can you tell me a little bit about UBC?   Shazia Ahmad: (01:18) UBC was founded in 2003 by industry experts with a passion for innovation and a commitment to working with pharmaceutical and biotech organizations in proving the safety, efficacy, and value of pharmaceutical medicine products. UBC is a leading provider of pharmaceutical support services, and we partner with life science companies to make medicine and medical products safer and more accessible.   Janet Kennedy: (01:46) Now, would you consider U B C to be a C R O?   Shazia Ahmad: (01:51) I would say we're CRO, but we really are proud to kind of stand out as a service provider, I would say.   Janet Kennedy: (01:59) Okay. Well tell me a little bit about what that means to be directing patient and physician services.   Shazia Ahmad: (02:05) At UBC, I have the honor of leading a team called patient and physician services. And what that is, is we're involved in supporting biotech and pharmaceutical sponsors in different services around patient recruitment, engagement, compliance, retention. We offer different services around making sure that we engage patients early on in research to ensure that the sponsor captures everything they need to, to really understand the patient journey and the development of whether it's the protocol design, developing the right strategies for recruitment retention. We're also able to integrate capabilities and services around awareness campaigns, where we use social media, digital outreach, and traditional advertising. A lot of programs that we do work on now at UBC involve the rare disease space. And I think Janet, that's where you and I really connected most over the last few years; a lot of the rare disease work that I lead. So we're able to also integrate different capabilities to really work with patient advocacy groups and really involve them early on in the launch of our programs to ensure that we're not just getting the patient voice by making sure that we work with the patient advocacy group as a partner, and really we work as a bridge to the sponsor with the patient advocacy group to ensure that we have the right strategies in place to support recruitment and retention for our program.   Janet Kennedy: (03:41) So are you actually bringing in these advocacy groups so early that they're participating in evaluating or contributing to the protocol design?   Shazia Ahmad: (03:49) Exactly. Exactly.   Janet Kennedy: (03:51) Well, that's kind of exciting. I'm curious - what sort of feedback have you seen coming from these groups that actually made the sponsors rethink how they had written a protocol?   Shazia Ahmad: (04:03) It's been interesting because there's been cases where we're able to learn something. And this could be in a rare disease indication where something was not thought about before, on what mattered to the patient, even the caregiver or care partner around different end points that were not considered. For example, they've not thought about maybe walking up the stairs would be something important to think about in the protocol design or certain visits to come to the study site and learning those things early on have really helped us integrate solutions to really make it easy for the patient caregiver care partner to participate in the study. And also we're able to integrate the solutions that really make sense for that protocol. For example, we have a clinical trial nursing service offering that we provide to different sponsors, where it makes sense to integrate home nursing for a clinical trial. The other thing that we're starting to do now based on our learnings, working with advocacy groups is integrate more decentralized solutions for clinical trials, which really has been going on a lot pre pandemic. But I think the pandemic has now really invigorated that even more and making sure that we can really bring in innovative solutions.   Janet Kennedy: (05:25) Certainly if you're dealing with a rare disease community that probably has multiple comorbidities - maybe a suppressed immune system - they're definitely gonna want to stay at home. So I'm very curious about the decentralized part. Are you finding that what you ended up developing was just more opportunities to gather data from the home, or were you working around fewer visits to a clinical office, but you tried to get more done at the time you were there? How that actually impact the work that you did in 2020?   Shazia Ahmad: (06:00) Actually it's a combination of all those things where we've been able to do more home visits and be able to really capture real world evidence and really real end points that couldn't be captured before; even things in between visits. So that's been very valuable, especially with the home nursing. And then also with the decentralized trials. One avenue we're actually looking to go to now is we've often had challenges in getting referrals from referring providers to really open up the pool of participants in areas where they want to participate, but are unable to, because they're not near a study active study site. So that's another way that we're able to connect participants patients to trials in their communities and really going to where patients are at.   Janet Kennedy: (06:53) Well, that's interesting. So in other words, in the past, we tended to have our principal investigators in larger metropolitan areas near maybe hospital systems with robust research, but the patients aren't necessarily there. They could be out in the boondocks somewhere. And what you're saying is now with this emphasis on decentralized solutions, you've been able to incorporate more patients - maybe even a broader range of patients from a diversity perspective - because these solutions made that possible.   Shazia Ahmad: (07:26) Exactly, Exactly.   Janet Kennedy: (07:29) So when you talk a little bit about real-world evidence and real-world outcomes, what kind of information are you gathering?   Shazia Ahmad: (07:35) We're looking at, and this could be any therapeutic area, but specifically in the rare disease space, we look at different - and this is more a question, I think, clinical operations, we work with the clin-ops team and developing the outcomes that matter - but what we do is we work with the patient advocacy groups to really hone in on a protocol and make sure the questions that are being asked, whether it's the patient reported outcomes, those things are really elements that matter to the patient. And it could be just the normal six minute walk tests. That's part of usually a pulmonary hypertension study or whether a cystic fibrosis patient had to take a day off from school. So it could be any of those things.   Janet Kennedy: (08:17) Shazia, what kind of key trends are you seeing in the industry regarding patient engagement in clinical trials and in healthcare work?   Shazia Ahmad: (08:27) That's a really good question, and something I'm really excited about. We're starting to see the need and the importance of integrating virtual patient communities. Something I'm really proud of that we've been able to actually start at UBC many years ago with a partner sponsor where we've developed patient communities, where patients can be part of a clinical trial and be engaged in part of a community. And they really, they feel then that they're really being, not just a contributor to the research, but then it's like a community building where they can be brought back or given information, not just about the research they participated in, but they can be informed on other trials that go on and they may be interested to participate in, in the future. So that's something I'm really excited about. I think there's more and more sponsors wanting to do that. And the other thing is the importance of giving back to the participants and making sure that they know what they contributed to participants often many, many years ago, did not receive information about the clinical trial they participated in, but now more and more sponsors are seeing the importance of needing to do that by giving back to participants, by providing lay summaries and a lay summary is really just summarizes the clinical trials, the results in a very patient friendly format.   Janet Kennedy: (09:58) Recently, there were two different Huntington's disease studies that did not come to fruition and announced in the same week, which was, I think very, very disappointing for the HD community. And that I think proves the point that not all research is going to work. But you have to acknowledge the contribution that patients have given and support them, even when things don't work out; help them understand why and explai

Huntington's disease is a rare genetic condition that impacts over 30,000 people in the U S and 200,000 worldwide. It is a neurological disorder that impacts movement mood and the thinking process. Hi, I'm Tom Rhoads, CEO of Spencer Health Solutions. On this episode of our podcast, we ask patient advocate BJ Viau, co-founder of the Huntington's Disease Youth Organization, to join us in a conversation about patient advocacy on People Always, Patients Sometimes.   Janet Kennedy: (00:35) Hi, my name is Janet Kennedy, and I am your host for People Always, Patients Sometimes, a production of Spencer Health Solutions. I'm really looking forward to a conversation today with BJ Viau. BJ is passionate about positively changing the lives of people impacted by rare conditions. He has a history of working to support patient advocacy and rare disease non-profits by holding grassroots fundraisers. BJ also helped create an international nonprofit for Huntington's disease, the HD Youth Organization, or HDO. He has an MBA and has been working in the pharmaceutical industry for over a decade. Currently he's the Director of Patient Advocacy at Horizon Therapeutics. Welcome to People Always, Patients Sometimes BJ.   BJ Viau: (01:20) Hey Janet, thanks for having me. Excited to be here, look forward to a conversation.   Janet Kennedy: (01:24) Absolutely. Well, I think one of the things I'm most interested in learning about right now is what is Huntington's disease and how does it impact young people?   BJ Viau: (01:35) Yeah, I mean, it could be a 20 hour answer, but essentially broken down, Huntington's is a rare genetic disorder impacting about 40,000 individuals across the United States. It's a neurological disorder that unfortunately takes over one's ability to do anything and everything in life. Most commonly described the combination between more common conditions like ALS, Alzheimer's, and Parkinson's all wrapped into one. How it affects young people is typically symptom onset starts for individuals later in life, probably in their thirties or forties, although it can vary. Although that's not technically a young person, where it starts to affect the young person is it's their parents. So you start to see decline in your parents, they lose their jobs, they lose their ability to function. Young people end up having to be caregivers for their parents. And what some might say the worst part about Huntington's is its autosomal dominant genetics, so every child of a parent who has Huntington's has a 50% chance of also getting Huntington's disease later in their own lives. So it's a little bit of looking in the mirror and wondering, am I going to have this future of my parents, or am I going to have a future of no Huntington's? And that's a really emotional decision that young people have to make.   Janet Kennedy: (02:50) Now you mentioned it's genetic. So does that mean you can take a little test and find out, or do you have the sword of Damocles hanging your head your whole life?   BJ Viau: (03:00) Yeah, take a little test. It sounds very simple and easy. Essentially it is. It's a blood test or saliva test that will provide you pretty much, if you will have Huntington's or if you will not have Huntington's. But even though it's simple, it's got huge social, emotional, psychological kind of bucket that comes with such a simple task.   Janet Kennedy: (03:22) I'm curious about the fact that you're focusing on young people. Is this to help prepare them for a future of thinking through how does Huntington's going to affect them, or is this actually helping them deal with their parents?   BJ Viau: (03:38) Yeah, a little bit of both. 15 years ago, when we were kind of dreaming up the Huntington's Disease Youth Organization A few of us young people were attending conferences and all the sessions and all the meetings were really focused for our parents, and there wasn't much support or education for us young people. In fact, a lot of people said, "Well, we don't even want to talk to young people about it because they shouldn't even be in the conversation." They were afraid to bring young people into this conversation, and that's where we thought that's not the case. We need to learn. We need to get educated. We need support. Not just to understand how to deal with our parents today that are going through Huntington's, but how we can start to shape and plan for our own future, which may or may not have Huntington's in it. So how do you prepare for school? How do you prepare for a job? How do you prepare for having kids, getting married, buying a house? Those are all really tough decisions that young people in Huntington families have to make very different from kind of the the normal person.   Janet Kennedy: (04:35) Absolutely. And I think it begs the question - do young people consider that they have to make a lot of different decisions than other people because their issue is genetic in nature?   BJ Viau: (04:48) I think they do. A lot of it relies on when the parents tell the child that Huntington's is in the family. Unfortunately in some families it's still a hidden secret and parents hold that secret from their children until the last possible day. And then, you know, the kids end up finding out some improper unique way. So what HDO's model is all about, not just helping the kids, but also helping the parents have that conversation with their kid to say, "Hey, you know, mom or dad has Huntington's. And that unfortunately means you may get Huntington's one day, but it's okay. You know there's people out there who can support you. We can get education, we can do this together." And it's creating that safe environment to have safe conversations that we truly believe at HDO is really best for everybody.   Janet Kennedy: (05:36) On your website, you actually have a section for kids. Is there an age that is too young to learn about this, or does that actually take away a lot of the veiled mystery or concern by just making it part of a natural conversation at a very young age?   BJ Viau: (05:54) It's a tough question. You're going to get a lot of different answers on who you talk to. I mean, I can speak from my personal experience. My parents told my sister and I, when we were about eight, nine, ten years old. And I would say them sharing that information with us. So young helped shape conversations that happened over many years to help my sister and I have the knowledge to create power for our own lives and feel motivated and empowered to understand what we're facing. I know a lot of other young people whose parents treated it similarly, and I see them these days dealing with HD in a very positive manner. I've seen a lot of families do the opposite. They hold it till the last possible minute until their kids are out of college, have their careers in front of them, are about to have a kid, and then they drop what I would call the HD bomb on them. And I've seen it go really poorly. So I would love for there to be a true research paper on when is the right time to talk to young people and tell them about Huntington's we don't have any statistical data to show when the right time is, but it would be great to provide that kind of statistical evidence to the parents who are unfortunately gonna face this decision with their children in the coming future.   Janet Kennedy: (07:09) That sounds like something the NIH ought to fund, because it isn't just hard conversations about HD that parents need to have. There are innumerable number of conditions or diseases that are genetic, that have a serious connotation. Even things that aren't genetic - it might be considered a disease that isn't quite visible - and at what point do you share with your children a serious condition that they need?   BJ Viau: (07:33) Totally. Yeah. It always seems like probably in the parent's perspective that tomorrow is a better day than today, but looking back, yesterday was always the best day. But that's just really difficult. It's tough. We're not trained in school, to have those conversations or trained as professionals. So it's a tough situation, but yeah, that'd be a great research study to get funded by somebody.   Janet Kennedy: (07:55) Alright. Well, you've planted a seed there. We'll see where we go with this. Well, let me ask you a personal question, If you don't mind. I know that you are a new dad. How did that conversation go with your then fiance?   BJ Viau: (08:08) Well, I'm pretty open and honest about my involvement with Huntington's. I am one of the few. There's actually a statistic that does show that about 90% of the people who are at risk for Huntington's choose not to get tested because there's, unfortunately, there's not much you can do about it as far as medication treatment wise today. So there's only about 10% who say, "Hey, I'm ready to know, even though I can't necessarily do something about it medically." But I, when I got tested, I learned that I would not get Huntington's in my future, which would eliminate the ability for my wife and I to pass Huntington's on to any of our children in the future. So it was a little bit of an easier conversation to have with my wife versus many decisions or many conversations that a lot other HD impacted family members have to have with a spouse. But anyway, her understanding of my involvement was very positive. So she got HD. She understood it. And I do think even if I had HD, we would have made a decision to have a kid, same way we did.   Janet Kennedy: (09:10) Okay. I'm a little bit shocked here. And I, I want you to reiterate - you said that nine out of ten people do not take a test to confirm whether they're going to actually experience Huntington's or not?   BJ Viau: (09:21) Yeah. Before they have symptoms. I mean, it's a tough question. I think if you ask the general public, would you want to know if you're going to get Huntington's disease or not? I think the general public you'd probably get the opposi

It is always a great pleasure to engage with clinical trial innovator and thought leader Craig Lipset. We asked Craig to join the People Always, Patients Sometimes podcast to share more information about DTRA, which stands for Decentralized Trial and Research Association. As an industry, we have embraced more fully the idea of serving patients better with a decentralized clinical trial. As we bring the corona virus under control, it's important that we don't become complacent and return to business as usual. Give a listen to the podcast and I hope you'll join us in declaring there is no going back.   Janet Kennedy: (00:43) It is always a pleasure to welcome Craig Lipset to the podcast. He is a man that really needs no introduction, but I'm going to give him one anyway. As the former head of clinical innovation and venture partner at Pfizer and on the founding operations committee for TransCelerate Biopharma, Craig is recognized as a leader at the forefront of innovation in clinical research and medicine development. He is a frequent speaker at healthcare and pharmaceutical conferences, and he has also led the call to innovate and recognize that there is #nogoingback to improve clinical trials. Today though, we're talking about DTRA, Decentralized Trials and Research Alliance. Welcome to People Always, Patients Sometimes Craig.   Craig Lipset: (01:29) It is such a pleasure to be back with you, Janet, thank you for everything that you're doing to give so many voices the opportunity to share.   Janet Kennedy: (01:38) Oh my great pleasure. This is always a learning opportunity for me. I am not a journalist, but I do play one on this podcast and I get to ask all the air quotes, dumb questions, that I'm sure a lot of people want to know about, but sometimes they get caught up, that something's already rolling ahead. And they're like, did I miss it? Did I have a fear of missing out what's going on?   Craig Lipset: (02:00) Absolutely. You know, there's so many different ways for people to stay current and stay connected. And you know, sometimes we have to hit a lot of different channels to make sure that everybody gets that opportunity to, to connect and to stay current with what's going on out there.   Janet Kennedy: (02:18) So on the connection aspect, we obviously have been dealing with the pandemic for gosh, a year now, and this time last year we were preparing for the last in-person event, which might've been SCOPE, and then suddenly things spun out of control and we were all sent home. I think things have changed a lot for you as well. So I'm curious, being able to look back a year over how things happened in 2020, what do you think has really impacted clinical trials with folks being sent to their rooms?   Craig Lipset: (02:56) You know, I think a lot of people will expect me to say things telemedicine and remote monitoring and risk-based monitoring. I would say that the thing that's really impacted us is willingness to adopt. And what I mean by that is so many of the things that have been so important for business continuity this year, the things that have kept our trials running weren't solutions that had to be cooked up in a lab in the year 2020. Most of these were solutions that already existed. They were just struggling for adoption, usually struggling for adoption because we operate in a very risk averse environment. But when the risk changed in the environment and all of a sudden, some of these things that might've been viewed as risky, suddenly became risk mitigation, they became the way to maintain business continuity. 2020 became a story about adoption, about so many great solutions that had been at our feet that had been at the doorstep suddenly seeing their moment in the sun when study teams and organizations were able to pick them up and bring them into their studies and bring them into their portfolios. And that's an exciting way for us to now get 2021 going, because now our organizations have seen that we can bring these different things to life, whether it's risk-based monitoring decentralized trials or whatever other solution your listeners were able to bring into their organizations to keep their work going.   Janet Kennedy: (04:23) Well I think that's an interesting point and you've been fortunate in that you've been able to sit a little bit in the cat bird seat because you've had an opportunity to participate in a lot of virtual conferences. You advise a number of companies. So you've been able to get feedback from a lot of different sources, as opposed to, if you were solely ensconced at Pfizer, you might not have access to the kind of information I'm thinking of. So here's my question. Do you believe that all companies, saw that opportunity to innovate and to accept and to accelerate? Or do you see that there's been a, sort of a separation of the wheat from the chaff and there are the companies that already had a innovation growth mindset, and then there are the folks that are just struggling to keep up?   Craig Lipset: (05:11) I think that in that April, May, June timeframe, a lot of organizations were all in the same boat. They were all trying to scramble to keep their portfolio going. They were rushing to introduce whatever countermeasures they could to keep patients being monitored, keep drug supply flowing, and keep capturing data. So that studies didn't wind up futile. But, Janet, I do think that we start to see some separation when we look to the latter half of 2020, because in that latter half of 2020, then we start to see that there are some sponsors out there that are starting to commit to some of these changes they're putting in place, new resources, they're relooking at some of their SOP and processes. They're expanding their vendor and partner lists some really starting to rethink how they write protocols and introduce some new training. These are the companies that are really starting to show commitment. They're really looking at the things that were adopted earlier in the year and being thoughtful about what's needed now to instantiate these and make them a part of our organization going forward. And that's different from some others that just maybe haven't been able to step forward to make those kinds of organizational commitments yet because they're hard.   Janet Kennedy: (06:35) Well, not only, they're hard; you may not even have the environment within your company that can embrace that kind of change and thought process. You really do need leadership that is willing to invest in that kind of information and that kind of structure, those kinds of individuals.   Craig Lipset: (06:53) Absolutely right, Janet. If there's one thing we saw in 2020, it's that these new approaches, well, they don't need special regulatory permission. Yes. The FDA produced some really important guidance for the industry about running clinical trials during the pandemic. But I encourage people to take a close look at that guidance. They didn't lower the bar on what technologies or approaches to introduce. They just said, use them and use them thoughtfully engage with your regulatory reviewers. And so from a regulatory perspective, these solutions were available; from a technical perspective, they were available. What does that leave? Exactly where you're pointing - culture, and our organizations ready in terms of their own internal culture and receptivity and willingness to change, or is it the culture that will ultimately stand in the way of those organizations being able to commit to and adopt these new approaches that were introduced last year?   Janet Kennedy: (07:51) I imagine that's a big part of what you do as a consultant is helping companies revision how they look at the growth of their company and expanding their capabilities through innovation.   Craig Lipset: (08:04) You know, Janet, I can only help organizations that are ready to be helped, but when they call, I'm certainly happy to be there. And mostly what I do professionally, whether with pharma companies, tech companies, or with others, is to help them make their strategies fabulous. And to help them make their implementations resilient. I can't fix the culture on my own. I can't show people solutions that they're not ready for. And so, you know, I can really only come in if leaders are ready to commit to these areas.   Janet Kennedy: (08:39) So you had a lot of time on your hands where you might've been traveling on airplanes, going places, and you obviously found a nice little hobby on the side where you decided to get together with some other thought leaders and launch a new initiative, the DTRA, the Decentralized Trials and Research Alliance. So I'd love to know the backstory. How did that come about?   Craig Lipset: (09:04) With all of that free time? Really Janet. So, it's interesting. DTRA is planning actually preceded the pandemic. It dates back to just prior to the pandemic when a friend and colleague Amir Kalali, who is well known in the industry from his time at Quintiles leading the neurosciences, and his time as the leader for the CNS summit Amir reached out and that he was exploring some different convening opportunities around decentralized. And we both appreciated that the world didn't need another conference on this topic. But when we have talked about decentralized in different meetings and events, there was always something left on the table. There was something that would come up at every one of those conversations about an opportunity to make the field better together. That was then left behind when that conference ended. And so we started to explore together what could be really sustaining and help to change this field.   Craig Lipset: (10:02) And then, the pandemic emerged. Which, in many ways, amplified everyone's attention around decentralized, but also was pretty distracting in terms of people needing to be heads down and keeping their portfolio going. And so while there was a lot of interest from a lot

One of the foremost conferences in the field of clinical trials is SCOPE, which stands for the Summit of Clinical Ops Executives. While held virtually this year, the conference was packed with interesting and innovative presentations. Spencer Health Solutions served as a premier sponsor of the event and co presented at the conference with a client and collaboration partner, Otsuka Pharmaceuticals. Our content was presented in the track, focusing on accessing and generating real-world data. The presentation was titled Implementing a Transformative Medtech Device Program to Gather Real World Data. Our co presenters were Tom Rhoads, CEO of Spencer Health Solutions and Kelly Roland, Associate Director, Otsuka. In planning this presentation, we decided to show a little bit behind the curtain of how a large pharmaceutical company evaluates digital health technology before it is written into a patient protocol. Let me set the stage. My name is Janet Kennedy and I'm the host of the People Always, Patients Sometimes podcast. Coming up is a conversation between Tom and Kelly walking through Otsuka's reasons for creating an internal focus group to evaluate the Spencer SmartHub. As part of the evaluation, Spencer health look forward to having Otsuka learn about and experience our deeper data and a more comprehensive look at how patient real-world evidence can be used to support the patient and improve outcomes. I hope you enjoy this candid conversation between Tom Rhoads and Kelly Roland on People Always, Patients Sometimes.   Tom Rhoads: (01:42) Hi, I'm Tom Rhodes, CEO of Spencer Health Solutions. Spencer is and FDA class one medical device for use with medication management, patient engagement and data collection. The Spencer SmartHub is being used in care management for about three years now. And in 2019, we added the clinical trial and commercial pharma division and launched Spencer SmartHub into both the clin trials, as well as post approved commercial farm applications. We're really pleased to be presenting at SCOPE this year, and we're very proud to be joined in the discussion today by our partner in an innovative internal focus group program. With me is Kelly Roland, Associate Director of Otsuka Pharmaceuticals. Hello Kelly, how are you doing today?   Kelly Roland: (02:23) Hi Tom. Thanks so much for inviting me to join you today. I'm excited to be here.   Tom Rhoads: (02:28) Well, that makes two of us. Kelly, our presentation in the SCOPE track is focused on accessing in generating real-world data, which is having a greater impact on the design and clinical trials and patient programs more than ever before. But before we jump into a discussion of our focus group, could you share any insights on why real-world data is a priority for Otsuka Pharma?   Kelly Roland: (02:49) Happy to. So as you know Tom, real-world data, real-world evidence - it's really information that creates action. So using this information, we're able to not only look at the improved design of clinical trials, but also conduct those clinical trials in new and different ways. Better data really lends itself to potentially faster analysis and better drug development overall, with really the aim advancing towards understanding both patients and drugs sooner. So from an operational standpoint, real-world data real-world evidence can enable more efficient, effective clinical trials and hopefully remove any friction for patients, investigators, and sponsors.   Tom Rhoads: (03:37) Well, it's interesting cause you know, as we were designing Spencer, the ability of patients to provide data back to the SmartHub was always central to our design. In fact, whether we're capturing biometric data passively through a Bluetooth connection, or from the patient's direct response for survey questions. We always wanted to be able to provide multiple layers of health data beyond the moment of medication dispensing, and our program today is really to share a rare insight into how a large pharmaceutical company can bring new technology to their patients and ensure that the internal team has buy-in on the new program. So Kelly, with digital health apps have been around for quite a while; why is the team at Otsuka interested in designing an internal evaluation before introducing Spencer to your patients?   Kelly Roland: (04:21) Otsuka is a company that's really dedicated to serving those with unmet medical needs, and we really strive to innovate and defy limitations. When we first evaluated Spencer, we felt it could really help us put the patient at the center of the trial first and foremost. And at the same time, we also knew it would break those current processes and operational logistics that you have for more traditional ways of running a clinical trial. So by getting out of our comfort zone and kind of embracing this new way, this new technology, a new way of doing things, we decided to pilot our own internal focus group, Otsuka only. We could have easily outsourced this to another group to research for us. However, we thought that this internal approach would allow Otsuka functional area champions really, or subject matter experts with that really in-depth personal experience with the device. They could experience firsthand what the patient would experience and help to develop new processes and new solutions based on those insights.   Tom Rhoads: (05:29) You know, you remind me, as we went to develop Spencer, we developed it from the patient out. So to see pharma taking the same position of understanding firsthand what the patient would experience is truly innovative, and we were obviously thrilled to learn you'd be interested in having your team experience Spencer in person. So maybe let's break down a few reason why that's a good idea.   Kelly Roland: (05:52) Holding that internal focus group served a few key purposes for us. First, as I mentioned, gather those insights very quickly and you get that firsthand knowledge that you wouldn't have otherwise. We wanted our teams to look through the lens of the patient, the site, and actually their own functional area when evaluating Spencer. So in the case of Spencer, it's a new technology, it requires a bit more organization, a little more prep work on the part of the team. And we thought this could be an exciting new approach for us. So our goal was to really set up a focus group so we could better evaluate everything end to end from the program set up, data integrations, training materials, and overall how Spencer worked.   Tom Rhoads: (06:38) So when you think about really the protocol that you're outlining, why was it important for Otsuka to kind of evaluate Spencer in each of the different roles?   Kelly Roland: (06:47) So when we're planning protocols or planning our programs, we really want to anticipate as much as possible where there could be difficulties where there could be challenges. One such area are those new roles and skill sets, quite honestly, that need to emerge when you layer technology and roll real-world data into the mix. In the past, we've relied heavily on, I'd say more manual processes together feedback from patients and sites. But with new digital platforms like Spencer, we can be much more proactive in assessing how a patient's doing between their clinic visits, especially as decentralized trials become much more common here in the future. But that said, we can better respond as a company if we've lived the experience in those key roles. It makes our team more intuitive in designing a protocol, and also a little more creative in the support materials that they bring along as well. So a focus group like this creates a lot of change in the company, but it also creates change agents, I'll call them, because now they've lived the experience, they can spread the word to their colleagues and other functions about the tool. They can be that subject matter expert in their function and help others to understand either the value and or challenges of this particular tool. So I think a focus group is really interesting and great way to start that change management activity.   Tom Rhoads: (08:15) That's a great point. So as we kind of break down the roles, maybe we can take a deeper dive into the patient role for a moment. What were you interested in evaluating as a patient using Spencer in a clinical trial?   Kelly Roland: (08:27) I said it before. I'll say it again - I really think this is going to give us invaluable experience from that patient perspective for our greater team. You know, we know we're working with a tool that has really interesting data around adherence and engagement, and we really wanted to explore that further. You know, we're asking ourselves questions like, "could Spencer cause annoyance or frustration for a particular indication?" "Are there other indications that may lend themselves more readily to a Spencer device versus another?" Would the collection of biometric data in the home be more desirable to a patient or caregiver versus having to go to the doctor's office?" I think if COVID has taught us anything that does seem to be a much more desirable functionality that people are looking at now. All this to say, it's very different reading about a tool and assessing capabilities versus actually experiencing them and walking in the patient's shoes.   Tom Rhoads: (09:22) So when we look at some of the aspects of the platform, how does the ability to gather patients' answers, to post medications, dispense survey questions, factor into your plan for utilizing real-world evidence? For instance, we found some eliminating data on how different individuals - in fact, two different individuals, both were basically 98% adherent - can reveal very different situations for those patients. Just curious, how do your plans factor in for that real-world evidence?   Kelly Roland: (09:50) So I think, as you indicated, medication adherence is only part of the story. So the questions we ask patient

Welcome to People Always, Patients Sometimes, a podcast production of Spencer Health Solutions. I'm Tom Rhoads, CEO of Spencer. This year, we are pleased to be presenting a series of interviews with patient advocates, inspiring leaders, and influential patient organizations to ensure that we always live our motto of people always, patients sometimes. On the podcast today, is Brian Lowe, the founder and CEO of Inspire. Brian created Inspire 15 years ago with a goal of accelerating clinical trial recruitment, using safe, trusted online social networks, organized by medical condition for patients and their caregivers. Today Inspire has 110 health association partners and over 2 million members. I hope you'll enjoy our conversation with Brian on People Always, Patients Sometimes.   Janet Kennedy: (00:56) There are over 200 million people living with one or more of over 7,000 identified rare diseases around the world. Rare diseases have a wide diversity of disorders and symptoms that can vary not only from disease to disease, but also from patient to patient suffering from the same disease. Hi, I'm Janet Kennedy with Spencer Health Solutions. I am so pleased to welcome a special guest from the rare disease community to the People Always, Patients Sometimes podcast. With me today is Brian Loew, who is the founder and CEO of Inspire, a patient community unlike any other. Brian, welcome to the podcast!   Brian Loew: (01:39) Thank you. It's really great to be here.   Janet Kennedy: (01:41) You really have been quite instrumental in pulling together patients who have rare diseases and providing them an online community. And that is an amazing thing and an important aspect of caring for patients in this crazy world that we're in right now. I'd love to know a little bit more about how you came to found Inspire, and then let's talk about what you're up to.   Brian Loew: (02:08) Sure. When we started Inspire about 15 years ago, it was out of our observation that there was a real need for patients and caregivers to connect safely online. And that there weren't great spaces for them to do so way back then. And it seems like a long time ago, what you found at that time was online message boards, discussion groups. Many of them were kind of Yahoo message boards. A lot of them are unmoderated, and yet you saw the need was enormous. Patients and caregivers were trying to connect around their health. And we saw that there was a real opportunity, I think, to provide this safely and with scale, a reliable platform or patients and caregivers could join and talk about their medical conditions.   Janet Kennedy: (02:49) You brought something interesting to the table in that your background is in technology. You are not a medical professional perse.   Brian Loew: (02:57) That's right. I had a lot of interest in health and so did my co-founders, but I didn't come at this from the perspective of a doctor or a scientific researcher. It was more of a technology background. That's right.   Janet Kennedy: (03:07) So that probably gave you a great leg up in creating something that was going to work. And you're much more aware of the issues involved, but how did you handle the medical side and even the legal and HIPAA related side?   Brian Loew: (03:21) Sure. One of the things we did from day one was we partnered with experts in this case, national patient advocacy organizations, which is, you know, there are about 2000 of these in the United States and they're organized often by specific disease. A very large number of these non-profit organizations are in the area of rare disorders. And in fact, our very first partner was the Children's Inn at the NIH, which is this extraordinary place at the National Institutes of Health to help support the families of children who are undergoing treatment, usually for rare disease or cancer at the NIH. This expanded to other national patient advocacy organizations like women, heart and the ovarian cancer research foundation and osteoporosis foundation. And over time, we now have 110 of these partners and about half of them are in rare disease.   Janet Kennedy: (04:13) So for instance, something like American Heart Association or American Cancer Society, those are very, very large organizations. But on the other hand, I imagine there's obviously more than one kind of cancer or one kind of heart issue. So do they have subgroups underneath an umbrella of their organization?   Brian Loew: (04:33) You're absolutely right. That as you said, if you look at the 7,000 or some estimates are now that there are as many as 9,000 rare disorders, there was enormous overlap with rare types of cancer. For example, where people have rare conditions that might be, you know, historically called a kind of cancer and now recognized as a rare subtype. And then there are other things that have always been thought of is rare disorders, things like Wilson disease or Von Hippel-Lindau syndrome or scleroderma or sarcoidosis or Ehlers Danlos syndrome. And we have partners in all of those areas. And I think what we're coming to realize is that kind of, as you pointed out, rare diseases are quite prevalent, even though they're officially defined as conditions for which there are less than 200,000 people in the United States with a disorder. It's estimated that one in 10 humans have one or more rare disorders. And so, you know, we're talking in the United States of over 30 million people who have a rare disease.   Janet Kennedy: (05:26) I'm going to ask a lay person's question here, and I know you are not a medical professional, but I'm going to take a great leap. It's not that there are now nine or 10,000 rare diseases that are new it's that our science is getting better and better, And we're able to much more specifically identify what these issues are?   Brian Loew: (05:45) Exactly Right. Exactly Right. And a lot of this is due to incredible advances in genetics over the past 20 years, the identification of things, which before weren't known to be conditions, syndromes, or disorders as such. One of the things that we see in Inspire all the time is what's come to be referred to as the diagnostic odyssey. Patients will have a collection of symptoms. It's clear that something is going on and what the patients are wondering is, do I have something that's recognized as a disease for which hopefully there's treatment? Or am I alone? Am I an 'N' of one? There's some things that are often observed only in a single person. And this is actually kind of an incredible aspect of rare disease, which is that many of the patients who find Inspired do so because they're searching online, searching for their own symptoms and what they discover is conversations going on in Inspire among other people who have the same rare disease that they do. And so there are many people on Inspire who have written to us and said, I discovered that I have this certain condition by connecting with others in your communities, and then going to my doctor and then getting genetically sequenced and discovering that I had a certain condition and that's really gratifying.   Janet Kennedy: (06:55) Well, and while it's nice to finally put a name to the thing that you're feeling, it must be incredibly frustrating if there isn't actually a treatment or a cure for that. So how is Inspire really helping to change the game?   Brian Loew: (07:11) Right. As your listeners may know, only about 5% of rare diseases, 5% of those, you know, seven or eight or 9,000 actually have a treatment. And so the vast majority are known to be real conditions often with genetic markers, but there are not good treatments for them. So there's a real effort by scientists and industry to help find treatments for as many of these disorders as possible. One of the ways we're trying to help is we developed something called the Inspire Research Accelerator, and you can check it out at Inspire.com/accelerator where we've offered to academics, to scientists around the world. If they're working on a particular disease and they need help finding patients to participate in the research, we will invite our patients to participate in their research. There's no charge for this. Our goal here is really to help advance the science. It's something that the patients are happy about as well, because by and large, if you ask members of Inspire what they want most it's to be as healthy as they can possibly be. And so they are very motivated to help support scientific discovery.   Janet Kennedy: (08:13) How long does scientific discovery really take? If you are approached by a pharmaceutical organization or even one of your partner organizations to say, "let's try and get a solution here." I mean, it's years and years?   Brian Loew: (08:28) Years and years. In some cases, decades, right? To sort of understand the condition and the underlying mechanism, and then to begin to explore what treatments are possible. And then as you know, once a drug candidate is identified on average, it takes 12 or 13 years to bring that new treatment from concept to market. And there are many failures along the way. So it's a marathon for sure. It takes a long time to figure these things out. Most of the time, by the way, one of the biggest bottlenecks historically has been that a researcher who has great ideas, isn't able to find enough patients to participate in research. And then in rare disease, sometimes the number of patients required could be dozens that it's fairly small, absolute number, but the conditions are so rare that it can be difficult to find 10 or 20 or 30 people for the rarest of conditions.   Janet Kennedy: (09:19) Have you found that you are building a community where the patients are predominantly coming to you or do you actually have to actively recruit patients with a certain disease or condition?   Brian Loew: (09:29) It's mostly the former. So the way that a lot of patients discover Inspire because they're searching onlin

Hi, I'm Tom Rhoads, CEO of Spencer Health Solutions. Today we have invited Jake LaPorte co-founder and global head of The BIOME by Novartis to return for a second conversation on the People Always, Patients Sometimes podcast. On the previous episode, Jake spoke about The BIOME by Novartis and digital health innovation in clinical trials today, Jake shares his thoughts with our host Janet Kennedy on COVID-19 innovation, digital health, and more. I hope you enjoy their conversation on People Always, Patient Sometimes. Janet Kennedy (00:36): Hi, my name's Janet Kennedy and I am your host for People Always, Patients Sometimes, a production of Spencer Health Solutions. Today we have invited Jake LaPorte to join us again for part two of a podcast episode, where we are discussing The BIOME by Novartis as well as patient centricity in clinical trials. Jake, welcome back to the podcast. Jake LaPorte (00:57): Thank you, Janet. It's great to be back. Janet Kennedy (01:00): So for the folks that might not have caught the previous episode, can you give us your elevator pitch on what The BIOME project is? Jake LaPorte (01:08): I'll do my best. So the thesis behind The BIOME is that Novartis has made a big commitment to become a company powered by data and digital, but yet we're not digitally native. So we almost always rely on an external partner to some extent, to help us build digital solutions. And we recognize we need to get a lot better at partnering with companies in the digital and tech ecosystems. And so The BIOME was developed to break down the natural friction that exists between a large multinational pharmaceutical company and the data and tech ecosystems and help us partner with those companies more effectively to develop digital health solutions that have an impact on patient's lives. Janet Kennedy (01:54): You know, there are a lot - like a lot - of digital tools out there. How do you even sift through and figure out who is a good potential partner? Jake LaPorte (02:04): Yeah, that's a great question, Janet. And that's frankly, what our process is all about, to be honest, because what is not often acknowledged in digital health is there's a scarcity of evidence that is available to understand whether a specific digital solution is going to be scalable if that's even feasible. And that it's going to have a meaningful impact if it is scaled. And so The BIOME is really an evidence generating mechanism so that we can make better more data-driven decisions about how we allocate our resources within Novartis to make sure that we're allocating more resources to those things that are more likely to be successful and have an impact on patient's lives. So for instance, we're doing a lot of work to figure out how we can sift through this complex ecosystem more effectively and more systematically to even surface the right partners. We're doing a lot of work to think about how then we onboard those partners more quickly and how we do what I call healthy proof of concept work with them. So we're really thinking about what are the real meaningful hypotheses, the questions that we really need to answer so that we can get more comfortable, that we need to put more resource behind certain companies and solutions in order to scale them a lot of that discipline. And I think this is true of most of the industry did not exist in the digital space. And frankly, it was a paradox, right? Because we have a very healthy innovation management process when it comes to developing medicines, we certainly just don't plunk a tremendous amount of resource into medicines that haven't gone through certain stages of trials. And we certainly know about our portfolio of trials and allocating resources to those ones that have gone through certain milestones, like stage one, stage two and stage three of clinical trials. However, we weren't doing that as systematically with our digital solutions and The BIOME is really that innovation management process. That's helping us do that with external partners. Janet Kennedy (04:13): All right. So one of the pushbacks that so many young new startups get is how many clinical trials have you done? Well, you know, none because we need you to try us take a risk believe in us. Is that something that's hard to overcome internally? Jake LaPorte (04:33): There are still challenges because there are so many solutions that exist out there. It's impossible for Novartis to make a bet on every company from the very get-go and be the ones that are really sponsoring their initial work. We're trying to make as best decisions as possible. So we spread our bets. So there are some that are kind of very early on companies and we have to do our best to figure out if we believe in what they're doing without the evidence behind them. And that fits well into what we're trying to achieve. And we make a bet on them. There certainly are companies that we do that with there's others, where frankly, we might need to see a little bit more evidence behind them before we make a bigger bet. And so it's a balance that we need to strike as much as we would like to try to support everyone in the very early stages, it's just not feasible for us to do that. Janet Kennedy (05:24): Digital is a pretty broad word. So when you're talking digital, are you literally talking about apps, platforms, medical devices? What kind of things are you looking at? Jake LaPorte (05:35): So for us, we take a fairly loose definition of digital. It could be any and all of those things. Distinctively we don't play in an area where we're partnering with conventional biotechnology partners to, for instance, co-develop assets or license, an asset from them. That's a clear place that we play, but any where there is data to be generated or devices to be tinkered with. I think that's a natural area where the bio model that we developed can be very effective. Janet Kennedy (06:11): Before we jump into the bigger conversation, can you remind folks how they can find out more about The BIOME? Jake LaPorte (06:17): Sure. They could probably Google Novartis BIOME, and we have a page that will give you a little bit more information about what The BIOME is, give you links to some of the leadership team, and you can always feel free to reach out to me or others to get more information. Janet Kennedy (06:36): Alright. And we will include a link to that in the show notes. Jake LaPorte (06:39): Perfect. Janet Kennedy (06:40): So Jake, one of the things that I was very curious about is the sea change that has happened and we're golly, nine months into a COVID process. How has The BIOME changed from this time last year? Jake LaPorte (06:57): It's been a meaningful change, not so much to the operating model that we developed, because we think that's a good one for us to facilitate digital innovation and meaningful partnerships to create digital solutions that have an impact. But certainly when you factor in COVID-19, which is first and foremost, a human tragedy, it's also certainly surfaced some of the challenges of the healthcare ecosystem. And we have really focused on helping develop solutions that solve some of the challenges presented by COVID-19. So for instance, we have a big initiative as a company on helping to think about developing telemedicine solutions that improve the continuity of care for folks that now cannot necessarily set up regular doctor's appointments, right? So there's a whole second level issue occurring in healthcare, which is that people that normally should be going to the doctor and getting diagnosed with other diseases, simply aren't going to the doctor or their physician as much and are suffering from diseases that they normally wouldn't have suffered from. So how do we help patients and physicians connect more effectively? So we're working a lot on solutions like that, making them scalable and effective and providing more convenience to patients and physicians that are challenged in this new era. We're also re-diverting some of our resources to focus on specific COVID 19 challenges that have been posed by certain government. So for instance, we have a BIOME in the UK located in London and they work with the NHS and the NHS has defined some specific challenges that they wanted to try and solve. And so our entire innovation program that we have in the UK this year was diverted to helping solve some of the challenges that the NHS has presented. So I think COVID-19 has given us a new problem set if you will, to focus on. And it certainly accelerated a lot of people to really think about how to use digital solutions creatively to overcome some of these challenges. Janet Kennedy (09:20): Well, I think that's really exciting because in many cases we think about pharma as being the big engine. That's only rolling forward at a certain pace and suddenly the flywheel of COVID-19 has accelerated that. And I think it might be really exciting to be working in a company to know that this is not a solution five years down the road. We're solving something today. Jake LaPorte (09:45): Yeah, certainly. I think again, although it's first and foremost, a human tragedy, it's sad that this is the catalyst behind some of this digital transformation. I think the solutions that are being developed are solving some more fundamental challenges in healthcare that will have longer term benefits after we get to a better place with COVID-19. Janet Kennedy (10:05): I agree. Totally. And I think telemedicine, which has been in development for over 20 years is finally seeing its day. Do you see any other type of digital health solutions that we're going to be treating more as an everyday use that might not have been accelerated so quickly? Jake LaPorte (10:25): Yes, certainly the biggie out there is telemedicine, as you already alluded to Janet. But I think that frankly opens up a platform for a lot of other digital solutions to be incorporated into a telemedicine backbone. I think as telem

Hi, I'm Tom Rhoads, CEO of Spencer Health Solutions. Our podcast People Always, Patients Sometimes was created to feature innovators, disruptors, and patients driving new ways of doing things in clinical trials. Little do we know that the coronavirus would escalate the need for transformation and demonstrate which organizations were prepared to move ahead more quickly. Today we've invited Jake LaPorte, co-founder and global head of The BIOME by Novartis. Jake has spoken on the topic of digital innovation and health tech startups, and impacting patient's lives. We enjoyed our conversation with Jake so much that we have a second episode with him on the schedule. Join us for this episode of People Always, Patients Sometimes as we listen to Jake's insights on The BIOME by Novartis in digital health and clinical trial innovation. Janet Kennedy (00:52): Hi, my name is Janet Kennedy and I am your host for People Always, Patients Sometimes, a production of Spencer Health Solutions. Today we have invited Jake LaPorte to join us. He founded and leads The BIOME by Novartis, also known as Novartis BIOME, the company's first ever externally branded innovation lab. The BIOME has garnered global recognition for supporting innovative tech and digital health companies and connecting them to Novartis's vast network of expertise and resources to accelerate solutions that improve and extend patients' lives. I really love that and I can't wait to find out more about it. Jake, welcome to the podcast. Jake LaPorte (01:32): Thank you so much, Janet, for having me. I appreciate it. Janet Kennedy (01:35): You know, you've had a very interesting back from before you got to Novartis. So do you mind bringing us up to speed and telling us how you ended up where you are? Jake LaPorte (01:43): I started off my career as a scientist. I was actually an organic chemist. I did a little bit of medicinal chemistry, but early on, I sort of learned that while I loved talking about and learning about science, I didn't necessarily love the bench work. So after graduate school, in order to get a different experience and try to figure out what I wanted to do with the rest of my life, I joined a global consulting firm, McKinsey and company, but there I focus primarily on the pharmaceutical industry and even within the pharmaceutical industry, primarily on R and D. And I got really obsessed with trying to make a contribution to what was starting to be discovered at the time as the R and D productivity problem, right in pharma. And now obviously there was data suggesting that we were on an unsustainable path and I generally believe in bringing technology and science to society to improve human health. So I want to make a contribution to solve the RD productivity problem. During my time at McKinsey, I sort of learned a tremendous amount, but at some point I got tired of kind of talking about it and wanting to try to do something about it. So that led me on a journey to work at a global CRO called PPD with one of the clients that I had at McKinsey. Her name is Kristine Bigaven. She was the chief medical officer at PPD at the time. We learned a tremendous amount from her. She ultimately was hired at, into Novartis to lead a big part of their global drug development organization. And I followed her there to Novartis, to lead digital strategy and innovation for our global drug development organization. And then ultimately then created The BIOME within that. And now we're trying to grow The BIOME into an enterprise wide solution for all of Novartis. So that's, that's sort of my snapshot of my journey to where I'm at today. Janet Kennedy (03:33): Okay. So I want to talk about the good old days of 2017 and digital development. What did that mean in 2017? Jake LaPorte (03:44): Digital development meant to us, which was the name of my organization was really thinking about how to harness these digital technologies and solutions that were starting to arise and incorporate them into future clinical trial paradigms that made them that made clinical trial is a better experience for patients and the healthcare providers that were participating in our trials and frankly, as well as to make them more efficient and effective and faster so that we could more effectively develop our portfolio of medicines just to make a concrete, right. We were looking at paradigms like decentralized trials, for instance. And how do we bring those about in a scaled way into our portfolio? We were looking at things like digital end points and evidence, and how do we incorporate digital technologies to capture new information in trials to make the development of our medicines more effective. Janet Kennedy (04:41): And tell me a little bit about the sense of urgency then versus now, was it something that like this was on our five-year plan or were you really trying to focus on things that, how can we get some tests going sooner? Jake LaPorte (04:55): So I'm sort of where we're at today. Obviously we've had a huge catalyst in this area also known as COVID 19, which has obviously challenged the conventional way to do clinical trials pretty significantly. So I think the urgency to do something in this area is unparalleled now compared to where were at 2017. However, we, you know, as Novartis, we had a pretty aggressive schedule to transform the way we did trials back in 2017. And it was more about how do we do some of these digital solutions at scale versus continuing to do them in pilots. But of course the urgency, now that folks have to transform as a matched, due to this new challenge in health. Janet Kennedy (05:42): Now, when you were looking at digital platforms in 2017, 2018, even early 2019, were you looking at things that you would absorb within your ecosystem or were these more partnership opportunities? Jake LaPorte (05:58): So I think most of the time, what we were looking at is for partnership opportunities. So many of these solutions and digital, and by the way, I think this also applies to the broader part of the enterprise are not things that are naturally owned by a specific pharmaceutical company, because most of the time for digital solutions to be meaningful, they need to be adopted at scale within the healthcare sector, right? Which necessarily requires that multiple pharmaceutical companies are using these things as a standard that multiple healthcare systems are using these things as a standard. So oftentimes that almost suggests that there's a partnership that needs to happen. Janet Kennedy (06:40): So what made you decide that the work you were doing in general needed this big investment of time and effort and physical location in creating The BIOME. Jake LaPorte (06:53): What it ultimately came down to is a little bit of what I said before that that partnerships are often so critical to building meaningful digital solutions that solve complex healthcare problems. And if you really reflect on where we're at as an industry, the pharmaceutical industry is not digitally native, but obviously we need to transform. And so we almost always require a partner to some extent, to help us build a digital solution. The complication is that we have just never, as an industry, built a capability to partner with the external digital health ecosystem effectively. And so there tends to be these barriers or this friction that naturally exists between a major multinational pharmaceutical company and the digital health and tech ecosystems. And The BIOME is really a focus on how do we break down those barriers and allow our internal teams to more fluently partner. And co-create with the digital health and tech ecosystems. That's really what The BIOME is about. We knew we needed to get better at partnering and that's what The BIOME is focused on. Janet Kennedy (08:03): So I'm really envisioning here, you have the big giant pharma company, and then you've got the fly by the seat of their pants startup. Those don't seem like they would mesh very well? Jake LaPorte (08:14): Perhaps not, but in a lot of instances, if you partner with younger companies that are maturing in the right ways, they can bring about beautiful solutions that can really have an impact on healthcare, but it's the key, the devil's in the details. How do you partner with them in the right way? A lot of the expectation tends to be that that partners are going to come with a fully baked solution that can easily be plugged in to an environment and deployed at scale. In the reality, what we found is that you need to be able to be committed and make an investment in these companies to really adapt their solution and help them co-create their solution so that it can be adapted into our, the context of the pharmaceutical industry. So let me give you an example of what I mean by that, that we've come out with publicly. And we talked about right, is a BIOME project that we did in support of our global health organization. And they had already launched an initiative to increase access to medicines for sickle cell disease patients in Sub-Saharan Africa. What we did is we helped them find a technology that by the way existed in Portland, Oregon, with this company called Hemex Health, that would allow them to identify patients in Sub-Saharan Africa, more effectively, diagnose them more effectively, and therefore allow them to get medicines to these patients more effectively. But that of course required that we make an investment in this company. They weren't for instance, approved by the FDA in Ghana, which is where we were launching this initiative. So we put some regulatory resources around this company to accelerate the regulatory approval onto the Ghana market to allow them to participate in this initiative. And now they're involved in this initiative and hopefully through this partnership, we're going to be able to get more medicines to the patients that need them faster with regards to sickle cell disease. So again, when you partner a

Tom Rhoads (00:02): Welcome to "People Always, Patients Sometimes." I'm Tom Rhoads, CEO of Spencer health solutions. Our podcast is focused on many of the thought leaders and executives that are calling for change in how we design and run clinical trials. This episode is the second in a series of interviews with patient advocates, influencers, and organizations dedicated to putting the focus in healthcare where it belongs, on the patient. Our mission at Spencer health solutions is to ensure that patients are at the center of everything we do. So we are pleased to introduce Carly Flumer to the podcast. She was a young adult survivor of thyroid cancer and author and a health data scientist. I know you will enjoy her conversation with Janet Kennedy on "People Always, Patients Sometimes." Janet Kennedy (00:51): The people always patients sometimes podcast is about the change agents in healthcare who are focused on patient experience, patient engagement, or disrupting the process of clinical trials to become more patient centric. Our guest today is Carly Flumer. She's a patient advocate, a data geek, a change agent, and one of Spencer's voices of disruption. If you have not read our ebook yet, in which Carly's comments are featured, look for a link in the show note. Welcome to "People Always, Patients Sometimes", Carly! Carly Flumer (01:22): Hi, thank you so much for having me. Janet Kennedy (01:25): I have been. So looking forward to talking to you, because while you provided a great insight to our voices of disruption ebook, we really didn't have a chance to have a conversation at the time. So now I'd like to get to know more about you. Carly Flumer (01:40): Wonderful. What questions do you have? Janet Kennedy (01:43): Let's start with, we invited you here because you have a very active patient advocacy focus in your life, and it is not everything you do, but it is an important part of who you are and what motivates you. So can you tell me a little bit about how that all came about? Carly Flumer (02:01): Sure. I was diagnosed with thyroid cancer in 2017 when I was 27 years old. So I was considered an adolescent and young adults or an AYA patient. And those are very few and far between, it feels like to me, in terms of where age lies on the cancer spectrum I feel like there are a lot of younger patients and then there are, are a lot of older patients, but we really don't hear much about AYA's, which are considered between 18 and 39. And so I wanted to get my story out there and I figured out the best way to do that was to become a patient advocate and to write, which is one of my passions. And so I have been writing about my story and my perspectives in healthcare from a patient perspective, and then from a perspective of somebody who works in healthcare. So it's kind of, two-fold where I can share it from in front of the doctor while also behind the scenes. And so I think if that gives me a unique look into all of the nuances of healthcare and how it can be changed to make better out patient outcomes. Janet Kennedy (03:11): How long was your cancer journey and where are you today? Carly Flumer (03:15): So I was diagnosed the second week of January of that year. And I was in treatment until the end of November of that year. So almost a full year. I am in remission at the moment. The magical words that every cancer patients want to hear is no evidence of disease. I have not reached that status and I may never reach that status, but I was told that I was in remission and I'm checked every so often to make sure that I remain in remission and I don't relapse. Janet Kennedy (03:52): Well, I think that's one of the huge challenges with cancer is while you might beat it, that's not necessarily a forever thing. Carly Flumer (04:02): Exactly. It's something that is always on the mind of every cancer patient, because it's so different for everybody. You hear that no patient is the same, but also no cancer is the same either. Whether it's considered the good cancer, which is what thyroid cancer is considered, or whether it's a cancer, that's more serious, but it's an ongoing thing. It's something that we all think about in the back of our minds. So just something to be cognizant about always, Janet Kennedy (04:34): And is getting thyroid cancer at such a young age, pretty unusual? Carly Flumer (04:39): I would say yes, yes and no. It is on the rise in terms of how many cases are discovered. But I have found, I guess, through my research and through my advocacy work, that it is more common among females. And I would say the majority of the women who I have found in support groups, they're in a wide age range. So from the early teens up to, I would say, you know, 60's - 70's, we are all over the place in terms of age. But the majority of patients I have found are women. Janet Kennedy (05:12): Let me ask you something about your advocacy as every cancer might be different. I imagine every patient advocate would give themselves a unique job description. So what exactly does your advocacy encompass? Carly Flumer (05:27): From my own perspective, my goals are to impact health literacy, patient education, oncology research, and patient provider communication, to create a better health outcomes in the oncology space. That's what my work, that's what my writing involves around those specific topics. But then my job, I wouldn't say it's necessarily advocacy work, but it's certainly within the oncology space is where I receive protocols from cancer centers across the United States for clinical trials. And it's written in this language that no patient could understand. What I do is I translate that information into language that patients can understand. And so that information goes onto website clinicaltrials.gov, which is where patients go to find clinical trials. Should they be interested in enrolling? And so it does kind of encompass that advocacy piece, where I am making that language clearer for patients in order for them to make the best health decisions for themselves. But it's more on the scientific ground, more so than I would say most advocacy jobs are. Janet Kennedy (06:44): So this is something that's a higher level information than webMD per se, but also making that scientific language very accessible. Carly Flumer (06:53): Right. Janet Kennedy (06:54): So how did you end up writing for clinical trials.gov? Carly Flumer (06:59): Well, the way our process works is we receive the protocol and we have a program that we use for our job and we abstract certain parts of the protocol that the patient is going to be most interested in, such as what are the objectives of the trial, meaning what are the researchers looking to do? What is going to be happening? What type of treatment are you going to be receiving? Whether it be chemotherapy, immunotherapy, radiation therapy, what have you, and for how long, what are the outcomes? What are the researchers looking to gain from this experience and what are they looking for patients to experiences, outcomes, including quality of life, survival, things like that. Eligibility of course, is a huge one to make sure that you are eligible to enroll and then things such as biomarkers, which are confusing at first to understand and I still get confused by them sometimes. But those are really key and understanding how a cancer can progress or how our cancer is found. But that's another topic with genomics. So yeah, that's the type of information that I am looking for in these protocols in order to translate that for, for patients so that they can understand what these clinical trials are going to be about. And if they want to enroll this as the information that they need to know in order to participate. Janet Kennedy (08:29): And this is through your work as a scientific data analyst for the National Cancer Institute? Carly Flumer (08:35): That's correct. So I contract with a company outside of the government and we work with the NCI and then these clinical trial protocols are coming from cancer centers across the United States. Janet Kennedy (08:49): You have experience with a clinical trial, do you not? Carly Flumer (08:53): I do. I feel like thyroid cancer is very rare in terms of clinical trials. I very rarely see them in my work and I'm not sure if it's because thyroid cancer has such a great survival rate, but there are variants of thyroid cancer that are more serious, such as medullary, which are the trials mainly revolve around that type. The trial that I participated in was a quality of life trial. And those, I feel like are so important. We see a lot of trials that are focused on treatment and prevention, but then those trials that focus on quality of life and how patients really are doing, whether they're in treatment or after treatment are so important. This one focused on the quality of life of thyroid cancer patients, which I loved because thyroid cancer is considered the good cancer, you don't really consider what can happen with patients who live without a thyroid. And I didn't know what a thyroid was when I was diagnosed with this type of cancer and, and, you know, any bodily function that you can think of the, I feel like the thyroid has a part in playing. And so living without one has really impacted my quality of life in ways that I wouldn't have thought of and ways that were not really described to me when I was diagnosed. And so really focusing on the, on the quality of life of these patients with this type of cancer, I thought it was really important to open the eyes of researchers and pharma and other advocates as to what living with thyroid cancer is really like, as opposed to what the media or, or other organizations or other doctors have termed it as they've termed it as the cancer you would want to get if you were diagnosed. And that is absolutely not true. And so I really appreciated the trial that I was a part of. Janet Kennedy (10:47): I think it's insane that someb

Tom Rhoads (00:03): Welcome to "People Always, Patients Sometimes". I'm Tom Rhodes, CEO of Spencer Health Solutions. Our podcast is focused on many of the thought leaders and executives that are calling for change and how we design and run clinical trials. We have also had patient advocates on the program like Jen Horonjeff, of the Savvy Cooperative, Pam Gavin of NORD and Barby Ingle of the International Pain Foundation. Our mission at Spencer Health Solutions is to ensure that patients are at the center of everything we do. So we are pleased to kick off a series of interviews with more patient advocates, influencers, and organizations that are focused on bringing the patient experience to the forefront of the conversation in healthcare. Kicking off our series is a conversation with Lilly Stairs and leading patient advocate advisor and founder of Patient Authentic. I know you'll find her conversation with Janet Kennedy, as interesting as I did. Janet Kennedy (01:01): "People Always, Patients Sometimes" podcast is about the change agents in healthcare who are focused on patient experience, patient engagement, or disrupting the process of clinical trials to be more patient-centric. Our guest today certainly fits that bill. Lilly Stairs is a nationally recognized patient advocate, speaker, author, and innovator. She has recently founded a new company, Patient Authentic, which I look forward to hearing more about on "People Always, Patients Sometimes." Welcome to the podcast. Lilly! Lilly Stairs (01:33): Thanks so much for having me, Janet. It's always a pleasure to connect with you. Janet Kennedy (01:38): I think entrepreneurship is a big challenge and as a patient advocate where you're already facing a series of your own personal challenges, as well as challenges in the workforce. Wow. What made you think about starting your own company? Lilly Stairs (01:55): Well, Janet, I will also say that I didn't plan to start a company the year that we ended up with a worldwide pandemic, but here we are to add that challenge to the list too. But yeah, I've always had an entrepreneurial spirit. From a very young age, I was always creative and started choreographing for a kids' theater program and took the helm there. And then in high school, I started an anti-bullying program that ended up touring around our local schools in Massachusetts. When I got to college that is when I started my patient advocacy work. And because I was diagnosed with a few different auto-immune diseases I started a campaign to raise awareness about people who were living with auto-immune diseases. There are an estimated 50 million Americans who are living with auto-immune diseases. It was called the 50 cents for 50 million. I have spent most of my career in health tech startups. It just felt right to jump in and take the plunge and make a bet on myself and start Patient Authentic. Janet Kennedy (03:17): I imagine your first 30 days you were feeling pretty euphoric and then February came and you probably started to be a little nervous. So COVID, how has that impacted your life and impacted your work? Lilly Stairs (03:31): Well, I think it's really, and I don't think this is an exaggeration, it's turned my life and everyone's lives upside down. And I, as somebody who is considered high risk because I am immunocompromised due to the medication that I am on, which depresses my immune system to treat my auto-immune diseases is certainly scary and carries a lot of weight. I am fortunate that from a work perspective, all of us in healthcare, I think have been quite busy, which is important. We've stayed busy trying to do what we can to support patients. And so from a work perspective, all has been really good. It's just that we've got a lot of COVID focused projects now. And then from a personal perspective, I think that honestly, it's been hard because it's isolating and I think a lot of people are feeling that, and there's always the fear that people are not taking it seriously enough, especially as someone who is high risk. Janet Kennedy (04:30): I understand totally what your experience is. Like my two nieces are in New York City who works at Trader Joe's and unfortunately, the misapprehensions and the willful lack of understanding is really frightening for them. One of them actually did get COVID, was fortunate that it felt like the flu, and got over it, but then found out later she had definitely had it. It has changed everything in our lives. So I'm curious as a patient advocate, and I know you are talking to patients constantly, what is the general mood among the folks that you're talking to? Are they finding that this is an incredibly scary time or are they finding that even the discussion of "at-risk" is raising awareness and making people a little more sensitive or empathetic to folks who have challenges? Lilly Stairs (05:27): Well, I have to say, I think it's a mix. And I would say generally speaking patient communities across their pubic areas have certainly been vocal about concerns that people are not taking COVID seriously enough. And I think that there are some really baseline things that everybody could be doing, like wearing a mask that would put a lot of those fears at ease. I will say warranted fears because for some contracting COVID is really a death sentence when you are somebody who's high risk. I'll be honest in saying that for me personally, I live in balance. I live in breathe and balance. That's how I operate. And for me, I've been cautiously optimistic and I'm cautious in living my life. So I don't just sit at home. I will go out, I wear a mask, but I'll go do outdoor dining in Boston. We have some awesome outdoor dining and, you know, see friends at a socially distant level because I think what we're seeing with this pandemic is a mental health pandemic kicking in because so many people are struggling with the isolation and the loneliness. So I try to weigh that out. And I think that a lot of other people in the community do that too. And it's really just about finding that mix of being safe and taking care of yourself. Janet Kennedy (06:50): I agree very wise words. Now let's back up a little bit and talk about Patient Authentic. I don't know what your company is doing. So tell me a little bit about it. Lilly Stairs (07:01): Absolutely. Well, you know, Janet, I think that I'm still learning what my company is doing and I don't know if I should be saying that, but here we are. Because I think it's evolved so much since I started in January. And a lot of that is largely because of COVID, but also because I've just ended up in places I didn't expect. And so the way that I like to talk about Patient Authentic is that it is a hybrid of healthcare marketing and patient advocacy. So all of my work is grounded in developing patient-centric deliverables alongside patients. And I'll talk to you about a few projects I'm working on just to give you a flavor of what it is that I'm doing at Patient Authentic. So I am leading patient advocacy for a health tech company and that's Clara Health, which is in the clinical trials space. Lilly Stairs (07:50): And I know that Spencer does quite a bit of work there too. I am developing and running a social media campaign with oncology patient influencers to promote a nutritional supplement, which helps to aid in the healing of mouth sores that have been developed from chemo and radiation. So out of that, we've developed the hashtag #MoreThanASore campaign, which is all about how sores are so much more than just a sore and they really impact the quality of life. And so there's the marketing in me coming out through that campaign. And then, you know, I'm working with some patient advocacy groups as well. So I'm building a masterclass to help train patients from across therapeutic areas and how they can be effective advocates on a policy level. So on Capitol Hill and at FDA advisory committees. Finally, I'm also supporting the development of a COVID-19 resource hub for the transplant patient community. My projects span across therapeutic areas and they are focused, at the heart of them, they are working with patients to create something, Janet Kennedy (08:56): Oh, that's excellent. Now, is this a single consultancy? Or do you have other patient advocates that you will bring in to support some of these projects? Lilly Stairs (09:04): Primarily it's a single consultancy, but I do often bring in patients who are paid because patients should always be paid to support the work. Janet Kennedy (09:14): I totally agree. Well, let's talk a little bit about some of the work that you've done in the past and hopefully in the future related to clinical trials, have you ever participated in a clinical trial as a patient? Lilly Stairs (09:29): I participated in a study that was through my gastroenterologist for my Crohn's disease. And it happened right when I was in the office and it was quick as could be. My gastro said, "Hey, I'm working on this study. We're trying to measure the levels in the liver because the liver is often impacted by treatments." And so I agreed to do it. And it happened all in the same office visit. It was the most convenient clinical trial experience I've ever had. Did the informed consent, all there. Probably happened in 20 minutes Janet Kennedy (10:08): Now from a standpoint of your work as a patient advocate and being around the clinical trial space and helping support companies and organizations that are involved in clinical trials. I want you to put your hat on as a patient advocate. If you were to point out one or two things that are really kind of a roadblock for patients in participating successfully in a clinical trial, regardless of whether the clinical trial is a success, what do you think are the challenges that patients face that the pharma companies, CROs and physicians that are involved in research need to be thinking more about? Lilly Stairs (10:48): I'm just going to pick t

The National Organization of Rare Disorders has been helping patients, caregivers, organizations, researchers, and Clinicians for almost 40 years. Introduction by Tom Rhoads (00:02): There are over 300 million people living with one or more of over 6,000 identified, rare diseases around the world. A disease defined as rare in Europe affects fewer than one in 2000 people. Rare diseases are characterized by a broad diversity of disorders and symptoms that vary not only from disease to disease but also from patient to patient suffering from the same disease. Hi, I'm Tom Rhodes, CEO of Spencer Health Solutions. We are honored to have a special guest from the rare disease community on the "People Always, Patients Sometimes" podcast. Pamela Gavin is a Chief Strategy Officer for the National Organization for Rare Disorders. NORD has been educating, supporting, advocating, and building a community for the rare disease community for over 38 years. I know you'll enjoy learning more about NORD on the "People Always, Patients Sometimes" podcast. Janet Kennedy (00:56): Hi, my name is Janet Kennedy and I am your host for "People Always, Patients Sometimes," a production of Spencer Health Solutions. We have invited Pamela Gavin, Chief Strategy Officer of NORD, the National Organization for Rare Disorders to join our podcast. Patients with rare disorders often are overlooked by clinical research and drug developers, or have histories of misdiagnoses. Now that Coronavirus is impacting the globe and awareness of the fragility of persons with rare diseases is triggering teachable moments for all of us. Let's dive in! Welcome to "People Always, Patients Sometimes," Pam!   Pamela Gavin (01:33): Thank you, Janet. Great to speak with you today. Janet Kennedy (01:36): I am so fascinated by your organization. I had no idea that literally there were hundreds and hundreds of rare diseases that you all are tracking and cataloging through your organization. Pamela Gavin (01:49): Yes, it's an amazing, exciting time within the field of rare diseases. We have over 7,000 known, identified rare diseases in the world today. And so NORD as patient advocacy organization supports all of them and the work that we do. Janet Kennedy (02:08): Can you tell me a little bit more about NORD itself and how it's structured? Is this an organization that provides information to people or are you proactive in helping resolve and learn and educate people about the diseases? Pamela Gavin (02:24): So we do some of both of those things and an organization established by patients and caregivers and advocates over 37 years ago, for some of the very things that you mentioned in starting your podcast introduction. And that is to advocate for improvements in it, interest in job development, device development, medical care, and services for people with rare conditions because very little was being done in the field. It was very hard to make it work economically for companies and researchers to study rare diseases because the patient populations were so small, but people were suffering tremendously. Pamela Gavin (03:04): So the community, the patient advocates got together and pushed for legislation that ultimately led to the Orphan Drug Act being passed in 1983. And that legislation established financial incentives for those to do research and study and develop products for people with rare conditions, the majority of which even today still live with a disease for which there are no FDA approved treatments. So we advocate for change. We advocate for people living with rare conditions to have access to proper diagnosis, treatment, and care so that they can live their very best life. We advocate for an environment that promotes research and innovation and fairness and equity across the entire community. As part of that advocacy work to promote access to that which is necessary for people to live their best life. We also do a lot of education to collaborate with organizations across the rare disease spectrum. Many of which are members of NORD, other advocacy organizations to educate people, clinicians about rare diseases. We also educate advocates so that they could advocate on their own behalf. Janet Kennedy (04:23): I'm thinking back to when you were founded 37 years ago, and there's no internet there. So I can't imagine how hard it must have been for patients with a rare disease to find other patients with rare disease. It must have been like living in a cave to feel so alone and to feel like they're so unique that nobody cared about them. Pamela Gavin (04:48): They could live their whole lives without meeting somebody with the same condition. You're absolutely right. Janet, the isolation is just an incredible challenge for people to live with such a burden. I can speak personally from my own family's experience, having grown up part of my professional career without the internet, certainly the beginning of it. And now knowing not only that we have the internet, but all the amazing tools to connect people together, to communicate with one another, to bridge people across many, many boundaries across the world. So there still is isolation today, but it wasn't seemingly hard to connect to people. I can remember the first time we saw a newspaper article where there was a story about somebody with a rare genetic condition that was similar to what my nephew had. And it was as if that piece of paper that photograph trying to connect to that community. It was so impactful and so meaningful. Now we don't have to fax things over a fax machine or try to find a phone number by looking in a yellow pages or white pages or calling institutions and calling and calling and calling until you found someone who may know somebody, a family, you sit at your computer or on your cell phone and start searching. Janet Kennedy (06:10): And sometimes you're looking across States across the country, across the globe to find other people with similar conditions. Pamela Gavin (06:18): Absolutely, absolutely. And we work with organizations like ours across the globe. It's a privilege and an honor to represent the rare disease patient and caregiver community. As we try to unite ourselves across the globe. Janet Kennedy (06:35): Let me ask you a question about your organization. It is made up of other organizations that support a very specific rare disease for instance, but do you also support individuals for whom there is no support organization? Pamela Gavin (06:52): That's exactly right. NORD represents all rare diseases that are known. So we work in collaboration with organizations that exist for those diseases that are known. And then if there is no organizational representation, NORD tries its best to fill that gap and representing those communities as well. Janet Kennedy (07:12): Before we move on to discussion of patients and clinical trials, I'd love to know a little bit more about your history and how did you end up at NORD? Pamela Gavin (07:21): It certainly was not a planned professional path. I started my career working in healthcare. I at first wanted to be a clinician. I wanted to be a physician and ended up finding myself, getting involved in healthcare information systems, technology really fascinated by the opportunity to leverage technology, to improve healthcare delivery, healthcare decision making, and the experience for patients so that they had better outcomes. And I was intrigued by the impact that technology could have disruptive in a positive way to empower clinicians, to make better decisions, to have better information, better tools, equipped to do their job better. And so that's really where I started to focus. I ultimately found myself evolving into an advocacy role because of personal experiences. I had members of my family to be involved in medical errors. So I got involved in advocacy for improvements in patient safety and clinical care safety. Pamela Gavin (08:32): And then that led to getting involved in rare diseases. My brother's first son, my nephew, Trevor was born with metachromatic leukodystrophy. Although we didn't know it right away, they took some time for it to be diagnosed. And this is right when the internet was really beginning to be used predominantly for academic and for business to business purposes. So to your point about isolation, I saw that firsthand. We're a pretty close family. And so we all supported my brother and my sister in law through this journey. But one of the first organizations that they were introduced to once they finally got this devastating diagnosis, the disease for which there was no cure and was going to take my nephew over very over a period of time was the national organization for rare disorders. And I remember what it meant to be able to talk to somebody that knew something about what was taking place in our lives. Pamela Gavin (09:31): And NORD connected my brother and sister law in particular to medical information and to clinicians and researchers that were working in the field. So even though there was no treatment or cure and the outcome was going to be inevitably devastating to know that there were people working on, it provided some comfort. So that's how I was first introduced to NORD many, many years later, an opportunity to join the organization came about where I was really marrying my work in healthcare information technology and data and supporting improvements in clinical outcomes. Marrying that with my rare disease experience, I feel very privileged to have this opportunity to work in this space, representing the community and helping to make a difference for those who today live with a rare disease or love or care for someone with a rare condition in the US. Janet Kennedy (10:35): I think that's such an important thing is to have passion for the work you do. And in your case, it's well, really a job. You can never leave, but it sounds like it's a job that also sustains you. Pamela Gavin (10:49): Thank you f

Introduction by Tom Rhoads, CEO Spencer Health Solutions (00:03): This past January, I had the pleasure to speak at the open innovation and health event at the JP Morgan healthcare conference. The event was sponsored by Mount Sinai Innovation, Ogilvy Consulting and Humble Ventures. Our panel discussed the consumerization of healthcare delivery, which was led by Ritesh Patel, Chief Digital Officer of Health at Ogilvy Consulting. Ritesh is an amazing thought leader in the digital health space and has been kind enough to accept our invitation, to be a guest on our podcast. And frankly provide is timely healthcare insights who could have known then the dramatic changes we've experienced over the last six months in healthcare. Simply remarkable. I hope you enjoy this conversation between Ritesh Patel and our senior digital brand manager, Janet Kennedy on the "People Always, Patients Sometimes" podcast. Janet Kennedy (00:52): Welcome to people, always patient, sometimes a podcast production of Spencer Health Solutions. Healthcare has come to a crossroads and it's time to start listening to new ideas. That challenge are always done it that way, thinking we hope you enjoy our conversations with the disruptors, the innovators and the transformers in clinical trials in healthcare. My name is Janet Kennedy and with me today is Ritesh Patel. He's the chief digital officer for health at Ogilvy Consulting. Welcome to the podcast, Ritesh. Ritesh Patel (01:22): Thank you, Janet. Thanks for having me. I hope you're having a good morning. Janet Kennedy (01:26): Everything is going great here a little bit rainy, but uh, I'm very happy to have that. Ritesh Patel (01:31): Absolutely. Janet Kennedy (01:33): We've had a chance to run into each other. A number of times in social also engaging in the digital medicine society, which I find the Slack channel to be very, very engaging and very full of exciting people wanting to do new things. Ritesh Patel (01:49): Yeah. You know, I was funny when, when I first became a member and got invited to be a member of it, I thought, Hmm, that's an interesting way of engaging with your membership using Slack, but I'll tell you what you're absolutely right. It's a fantastic tool. And a lot of other member or societies should really look at that because the engagement level is fantastic. There's so many ideas being exchanged, people, helping each other announcements being made. I think it's a really smart move by the Dime Society to do that. So I'm very impressed. Janet Kennedy (02:23): I have used Slack for a couple of other organizations and companies that I've been in. And I constantly am surprised when people are like, Oh, just text me. You know, it's a conversation that just gets lost. It's very hard to have a group text with anybody and not drive folks crazy. And you are pushed by text, whereas Slack, you can engage with at your own pace and at your own level. So I don't know why they would prefer that, but I love the way Slack has enabled me for instance, to meet and engage with folks. And in our case to reengage after podcast interview over four years ago. Ritesh Patel (03:02): I know. We use Slack as a collaboration tool for Ogilvy Consulting for many years now. With the youngsters that we have coming in from universities, they're used to that. They're not used to us old fogies using email and mainly email. So it was interesting for me, and eye opening society would use it the way that are using it. It's very well done. Janet Kennedy (03:26): Well, I think the other interesting thing about the Digital Medicine Society is you don't join as your brand or company. You join as a person and everybody in essence is equal because it's only $50 to join. And it's an individual membership and you may or may not be speaking on behalf of your company. If you want to innovate, you can come in there and be part of the conversation. Ritesh Patel (03:47): Yeah, exactly. Exactly. I love it. Open Innovation in Health Pop Up on Sunday 12 Jan 2020 in San Francisco, CA, USA. Janet Kennedy (03:50): Well, let me ask a little bit about Ogilvy Consulting for the folks who don't mind for the folks who don't know. Do you mind giving folks a little elevator pitch on what Ogilvy Consulting is? Ritesh Patel (04:01): Certainly. About seven years ago, we noticed that the big consulting companies like Accenture and Deloitte are getting into the creative agency space and buying up agencies. I think Accenture Interactive is huge now because they've acquired so many interactive digital agencies. And so we, as Ogilvy, with our deep 75-year heritage of advertising decided to get into the consulting world. We wanted to make sure we could claim an area because of the brand Ogilvy to help our clients with. And it's really around brand customer experience and positioning in this new digital world that you're living in. So three years ago, it was all about digital transformation. And what we would do is say, well, that's all good. You're transforming your business, but how does your brand fit into that? How are customers experiencing your brand or your products or your service in that digital world, you may be transforming internally, but there is an impact that it has on the revenue that's being generated. Ritesh Patel (05:02): So that's what we focus on. We have a, quite a large group of folks around the world 400 or so that do that for big brands, as well as healthcare where I sort of read on the digital health practice is what can we do from that perspective, if you're a pharma company or a health system or a payer or a medical device company, we're doing a lot of work in that area that way. Janet Kennedy (05:26): And I understand that the work you do is also very collaborative. It isn't Ogilvy coming in to do strategy and we'll do all these other pieces for you. What you're actually doing is basically "cat herding". Ritesh Patel (05:39): You know, a lot of the times one client we're working with a global pharma company where we're creating a digital business framework, et cetera. We then work with, you know, I think they have you name it. They have McKinsey in there, Cognizant in there, Wipro in there, Accenture in there 80 in there. And then on the agency side, they have 13 different agencies they work with. And we're the Switzerland in the middle of that basically works with the business units to create all these things. And then we educate the agencies and the partners to say, this is how we need to operationalize this. So we're very much focused on what is that you're trying to achieve, how will it get achieved? And then who are the players we can help you get there. And that's not necessarily Ogilvy on the sign. Janet Kennedy (06:25): I'm curious about brand with a pharma company because some people may know a drug name better than they know the pharma company when drugs are being designed. And they're going through the naming process and the logo design process. Do they take a lead from the corporate branding or did they view each of these drugs as independent brands? Ritesh Patel (06:47): Well, I don't know if you know, it's a little secret here in the U S that there's only two companies that have the market for naming drugs, that they split between them 50/50, and it's all around the product. It's never around the parents. It's never around, it's a Pfizer products or Novartis products or a Merck product. It's all around the molecule and the medicine. And there's some taxonomy that's been created as a gentleman's agreement between the two organizations that they adhere to and the scientists adhere to. But, you know, I think Inventive owned one of those companies back when I used to work for Inventive that did that naming and they actually, they were based in Raleigh. Actually, if I'm not mistaken, I have to look them up. Now my brain is as melted around all that, but I think most cases, they look at it from the product, never the parents. Janet Kennedy (07:38): That is absolutely fascinating. And I learned my something new today. So I definitely want to find out more about that. That's awesome. Well, I know one of the things Ogilvy Consulting has been doing is some big thought leadership programs from an in-person program at the JP Morgan pre event, where my CEO, Tom Rhodes, was able to participate to a whole series of webinars that you've been doing. Tell me a little bit about how you go about selecting what you're going to be talking about and what are you getting out of these webinars, Ritesh Patel (08:12): I'll take those industries. So the first one is sort of how do we select we select based on what's happening. There's not a sort of a global annual content calendar we create. It really is around what is the interest in? And do we have enough thought leader capability or partners or people we know we can reach out to, to pull that together. And so recently you've seen an uptake on the webinar simply because we're seeing a huge amount of interest from our clients on the topics that we've covered so far. The first one we did was around the open data walls. You know, CMS announced the open data rules and interoperability rules that people like Epic were fighting for a bit and the President signed them into law not too long ago. So there was a huge amount of interest from a lot of our clients. What does that do for me? And what's the impact on my business? We thought it'd be good to bring some people together who knew about that stuff so they can get into the nitty gritty of it a little bit. So that was then, and similarly, the next one was around virtual trials, huge amounts of interest in virtual decentralized trials because clinical trials have come to a bit of a grinding halt since the pandemic. And so what can we do? I was getting frustrated because I was attending a number of these, whether it was all about the technologies, it re