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P4A Let's Talk Rare: The Life Science Podcast
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P4A Let's Talk Rare: The Life Science Podcast

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Welcome to P4A Let’s Talk Rare, a monthly podcast highlighting the most important developments in the world of rare diseases orphan drug, cell and gene therapy, hosted by Georgie Rack and Owen Bryant of Partners For Access. To find out more about Partners For Access and our commitment to sustainable orphan drug access for patients with high unmet need, visit partners4access.com
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Hello and welcome to this special episode of Let’s Talk Rare: The Life Science Podcast as we celebrate 5 years of bringing you the podcast that is now the number one life science podcast across all platforms. We at Partners 4 Access want to thank every single one of our 35,000 subscribers and all the guests who have graced the podcast from the bottom of our hearts, we would not have gotten here without you.  A special episode calls for special guests, and joining us today are our in-house expert and Managing Partner at P4A, Sophie Schmitz, our host from 2018-2021, Aparna Krishnan, and Prasan Subedi, Access Strategy Team Lead at Pfizer. Together, we walk down memory lane and relive the high points and challenges of the past five years, and share insights on rare diseases and cell and gene therapies. Aparna Krishnan Bio: Aparna has over 10 years of experience in the pharmaceutical and healthcare industry. As part of the senior leadership team, her primary focus is on strategic corporate engagement, legal, financial operations, and human resources. She has previously worked as Manager of Life Sciences at IHS Markit and as Consultant at Parexel’s Commercialization unit, among others specializing in market access, pricing, and reimbursement. Sophie Schmitz Bio: Sophie is the managing partner at Partners 4 Access, a company that believes in a sustainable rare disease world, which means helping our clients unlock the true opportunity of orphan drugs and ATMPs. Her 20+ year career spans various disease areas and medical devices, successfully supporting the strategy development and operationalization for orphan drugs. She brings a wealth of commercial and access experience across orphan and cell and gene therapies to the benefit of clients. She plays an active role in advising clients on price and market access strategies, with the ultimate goal of optimizing fair access for all stakeholders in rare diseases.
In this podcast, Georgie, RJ, and Owen discuss revolutionizing patient outcomes and the power of digital health solutions. RJ, a healthcare industry expert, shares insights on the role of technology in improving patient care and the future of digital health. They also touch on RJ's involvement in triathlons and his passion for running. With a friendly and engaging tone, this podcast offers valuable insights into healthcare and the potential of digital solutions. RJ Kedziora Bio: Mr. Kedziora is the co-founder of Estenda Solutions, a leading company specializing in custom software and data analysis for healthcare and medical companies. With a remarkable journey spanning over 30 years, he possesses a deep understanding of designing, developing, and deploying successful software projects. His extensive experience enables him to provide valuable guidance and innovative insights, resulting in cost-effective solutions that improve patient outcomes. Mr. Kedziora received his M.B.A. from West Chester University and a Bachelor of Science in Computer Science from Duquesne University where he received the Excellence in Computer Science Award. He has spoken at numerous technical and healthcare conferences on a variety of topics and written or co-authored multiple articles focused on healthcare information technology, several published in peer-reviewed scientific journals. Social Media Links: FACEBOOK (BUSINESS): https://www.facebook.com/EstendaSolutions LINKEDIN: https://www.linkedin.com/in/rjkedziora/ INSTAGRAM: https://www.instagram.com/estendasolutions/ TWITTER: https://twitter.com/Estenda YOUTUBE: https://www.youtube.com/channel/UCuAfCbIFW0DmeI5PDS1PXcg
Welcome to this month's episode of the Let’s Talk Rare podcast brought to you by Partners4Acess. Georgie and Owen are joined by Chloe Sheppard and Akshay Kumar to discuss the new EU HTA regulation set to be implemented in 2025. They explore the implications for drug developers of ATMPs, the attractiveness of the EU market, and the importance of proactive preparation for the upcoming changes.  The topics covered include: key milestones of the EU HTA regulation; the importance of drug developers of ATMPs and oncology medicines to not adopt a wait and watch strategy; the risks of ignoring this change and the potential impact on clinical development plans, commercialization strategies, and patient access; the opportunities the EU market presents; and the need to start planning and adapting internal processes to successfully navigate the new regulatory landscape. Chloe Sheppard Bio: Chloe serves as a Senior Consultant at Partners4Access, working closely with clients to craft custom solutions for their pricing and market access obstacles. Her motivation lies in transforming the immense potential of advanced medicines into tangible therapeutic benefits for patients. Akshay Kumar Bio: Akshay, a Partner at Partners4Access, brings over 13 years of experience assisting pharmaceutical companies in devising market access and pricing strategies that align with their broader commercial and organizational contexts. His expertise spans not only product-level solutions but also process improvements and capability enhancement. In addition to market access, Akshay is well-versed in various other aspects of drug commercialization, including opportunity assessments, product positioning, forecasting, customer insights, and organizational design. Episode Resources: Chloe’s LinkedIn Akshay’s LinkedIn Partners 4 Access Website
P4A are joined by Xortx Therapeutics' CEO, Dr Allen Davidoff, as he discusses the journey from early development to potential launch for their novel therapy XRX-008 to treat patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD). Allen discusses the company vision for developing novel therapies for rare progressive kidney disorders, and tells us about the launch journey for their main therapeutic candidate for AKPKD. How they managed to complete enrolment for late phase study in record time by engaging with patients and advocacy groups early, to really understand the patient journey and how the disease affects their quality of life. This helped to shape the clinical trial design by truly putting the patients at the forefront of all decisions and to ensure they were involved at all stages of the process. Bio: Dr. Allen W. Davidoff, Ph.D. (15 years drug development experience ), Currently President and CEO of XORTX Therapeutics Inc (2013- ), Formerly, Chief Scientific Officer, VP Product Development and co-founder of Stem Cell Therapeutics Corp (Trillium Therapeutics- TRIL: NASDAQ) (7 yrs). Prior to Stem Cell Therapeutics Corp., Senior Scientist and Head of Pharmacology at Cardiome Pharma Corp. Dr. Davidoff has 12 + years of therapeutic drug development experience with a focus on clinical and regulatory developmental affairs, in the US, Canada, Germany, Denmark, and India. Senior management and leadership experience includes pharmaceutical non-clinical and clinical R&D including 2 investigational new drug (“IND”) applications or supplemental IND’s, 2 phase I studies (4 multi-country), 7 phase II studies, and 1 NDA. Dr Allen Davidoff: https://www.linkedin.com/in/allen-davidoff-9a80968/ XORTX Therapeutics XORTX Therapeutics, Inc. is a drug based biotechnology company primarily focused on orphan disease indications which have aberrant purine metabolism and frequently high serum uric acid imbalance. Our focus on developing kidney therapies also includes therapeutics programs for large markets such as diabetic nephropathy and acute kidney injury associated with respiratory virus infections such as SARS-CoV-2. We possess patents and patent applications that may include U.S. and international rights regarding the development of uric acid-lowering agents for the treatment of hypertension, insulin resistance, diabetes, metabolic syndrome, and kidney injury. XORTX Therapeutics: https://www.xortx.com/
Did you miss the World Orphan Drug Congress Europe 2022, held in Sitges, Barcelona in November? If you did, don't worry P4A have you covered! This month's special  podcast was part recorded live at the conference and part recorded back in London with members of the team that attended! We will be speaking to a few presenters & sponsors to discuss their presentations, key learnings and their personal highlights from the conference. Also the P4A team will be talking about our favourite sessions and the key takeaways from the conference. Special thanks to our guests for their insights and learnings throughout the podcast. Presenters: Georgie Rack & Owen Bryant Guests: Alexander Natz, Secretary General at EUCOPE - https://www.eucope.org/ Leon van Wouwe, Clinical Innovation Director at VOLV Global https://www.volv.global/ Wing-yun Cheung, General Manager at Terrapinn & organiser of WODC EU - https://www.terrapinn.com/conference/world-orphan-drug-congress/index.stm Chloe Sheppard, Consultant at P4A Joanna Fernandes, Senior Consultant at P4A
 Winds of change are howling in Germany, with the draft healthcare bill now approved to stabilise SHI fund finances. What will manufacturers, with innovative orphan drugs and cell and gene therapies, launch strategies be? With the latest decision from Janssen to avoid the German market altogether for x2 Rare Oncology innovative drugs, will this be a trend we are likely to see continue?  Join Stefan Walzer & Fisentzos Stylianou discuss the new bill, in regards to the biggest changes that impact orphan drug (OD) manufacturers. Will OD manufacturers still see Germany as the first go to market within Europe and what does this means for rare disease patients?  Will there be delays to new treatments or will manufacturers decide not to launch in Germany at all to protect the price of their new drug?  We will be discussing this and so much more! If you are a drug manufacturer planning your launch strategy, this podcast is for you!  Presenters: Georgie Rack & Owen Bryant Guests Stefan Walzer, CEO President & Founder of MArS Market Access & Pricing Strategy GmbH Fisentzos Stylianou, senior analyst and P4A's German country expert Dr Stefan Walzer, CEO President & Founder at MArS Market Access &Pricing Strategy GmbH https://marketaccess-pricingstrategy.de/en/ Bio & Fun Facts  1) Economist, PhD in health economics, diploma in clinical trials 2) Experience in MA, reimbursement, HE and pricing since 2004 in consultancy and industry 3) Founder and CEO of MArS - THE D-A-CH market access consultancy. Linked to that also co-founder of SMS2DACH including the full spectrum for D-A-CH support (distribution, management, launch, etc.): www.sms2dach.com 4) Founder of P&N (pricing-and-negotiations.ca) with a focus on negotiations across the world including being the co-founder of www.thenegotiationlab.com 5) Member of a European network Tesseract (https://www.tesseracteurope.com/) which can serve companies moving from outside Europe into Europe including not only reimbursement services but also logistics, customs, etc. 6) Teaching at various German universities 7) Love spending time with my family, being a soccer coach of under 14 years-old and being a supporter of Borussia Dortmund Fisentzos Stylianou, Senior Analyst & German Country Expert Fisentzos role at Partners4Access includes conducting primary and secondary research to support the development of market access and reimbursement strategies for clients in the pharmaceutical and biotechnology industries. With a passion for innovative treatments, he closely follows the cell and gene therapy field as it expands to treat more patients with rare diseases.  Prior to joining Partners4Access, Fisentzos worked as a Research Associate at Imperial College London, where he also earned his Ph.D. in Structural Biology in 2020. During this time, he conducted research as part of a multidisciplinary team to advance his understanding of the structure and function of biofilm-forming proteins, paving the way for the design of novel antimicrobial therapeutics. Fisentzos also holds an M.Sc. in Biomedical and Molecular Sciences Research and a B.Sc. in Biomedical Science, both from King’s College London. 
P4A’s 2 part -Patient Empowerment podcast series has been released. Our incredible panel of experts Laurence Woollard, Neil Bertelsen & Sophie Schmitz are back with us to carry on the discussion in part 2 on true patient empowerment and really looking at the WHY’s! Why should drug manufacturers involve & listen to and involve the patients. What are the implications, if any, for drug manufacturers who do not include the patient voice. How can we bring all stakeholders together to work completely new approaches to medicine approval and patient access. Lastly, we will look at initiatives or services our panel are offering to try to bridge the gaps and barriers for sustainable patient access. P4A’s’’Let’s talk rare’’ monthly podcasts are available wherever you listen to your podcasts. If you are a drug manufacturer and you haven’t yet listened, this needs to be at the top of your to do list! If you missed out on part 1 - listen here: https://spotifyanchor-web.app.link/e/hOBI3UqkPtb Hosts: Georgie Rack (G-Rack)  & Owen Bryant (OB1) Produced by: Operations Team Links: P4A’s PCAC: https://partners4access.com/services/pc-council/ Part One: Patient Empowerment: https://spotifyanchor-web.app.link/e/hOBI3UqkPtb ABPI article in recognition of Rare Disease Day 2022 - “Moving      beyond box-ticking and lip-service - why patient involvement matters in a      new era of ATMPs for rare diseases” James Lind Alliance Priority Setting Partnership - “Setting       priorities for bleeding disorders - Final report” (2018) “More than a Top 10: How James Lind Alliance Priority Setting Partnerships transform research, people and organisations” (2019)
This month we have a special 2-part series focused on Patient Empowerment, with special guests Laurence Woollard, On the Pulse, Neil Bertelsen, Independent Consultant & Sophie Schmitz, Managing Partner at P4A. This podcast is a MUST to listen too if you are a company looking to successfully develop and commercialize an orphan drug or ATMP The first part will focus on how do we ensure there is genuine patient involvement at all stages of drug development? What are the challenges and barriers for patients living with a rare disease vs drug developers within the rare/ultra-rare sphere? The second part will solely focus on the WHY! Why should drug manufacturers, payors, regulators and HTA bodies involve and listen to the patients? What do they bring to the table? We will also dive into the implications of not including the patient voice and use examples of companies that have successfully managed to do this and the benefits of doing so. Laurence Woollard, Owner On The Pulse Laurence Woollard is founder and director of On The Pulse – an independent, strategic consultancy providing specialist insight to global healthcare providers and multi-agencies on the development and rollout of patient activation campaigns in haemophilia and rare disease. Laurence has extensive patient advocacy experience, having advised on and implemented a broad scope of community awareness, research and educational initiatives for commercial partners and third-sector organisations.  Neil Bertelsen, Independent Consultant Neil Bertelsen is an independent consultant and patient advocate with over 25 years of experience bringing the patient voice to health care decision makers and communicating the science of health care to patients in a way that truly informs their own personal health choices. Neil is passionate about bringing the patient experience and perspectives to decision-makers including industry and health technology assessment (HTA) bodies. Neil is the past chair of HTAi (the global scientific society of HTA) Patient and Citizen Involvement Interest Group. This is an international multi-stakeholder group whose remit is to work alongside HTA organizations and patient organizations to bring patient involvement processes and patient insights and experiences into access decision making processes. Sophie Schmitz, Managing Partner P4A Sophie is managing partner at Partners4Access(P4A),an award winning global consultancy focused on access for orphan drugs & ATMPS. P4A support the biotechnology & pharma industry along their launch journey to help secure successful price, reimbursement and access for orphan drugs & ATMPs. The company has solid partnerships with clients supporting their strategy & operations to effectively launch success. 
This month we have put together a slightly different episode with a twist. Georgie & Owen will take you behind the scenes at P4A and discuss 'What makes P4A an award-winning global market access consultancy?' Why we do what we do every day, and what it means to each and every member of staff. Pride, Passion & Partnership are the core values at P4A, and we take a closer look into each one. Later in the show we have a very special announcement from our Managing Partner, Sophie Schmitz. Host: Georgie Rack, Communication Executive & Owen Bryant, Creative Director Produced By: Operations Awards P4A have won in 2022 (so far!) - https://partners4access.com/about/awards/ 1. BOBI Awards 2022 - Analyst Team of the Year Link: https://www.bhbia.org.uk/bobi-awards/award-winners/bobi-2022 2. Benelux Enterprise Awards 2022- Best Orphan Drug & Gene Therapy Experts Link: https://www.eubusinessnews.com/awards/benelux-business-awards/#ourwinners 3. International Life Science Awards 2022 - Best Cell & Gene Therapy Global Market Access Consultancy Link: https://www.ghp-news.com/awards/international-life-sciences-awards/ 4. Healthcare & Pharmaceutical Awards 2022 - Leading Experts in Orphan Drug & Gene Therapy Access Link: https://www.ghp-news.com/awards/healthcare-pharmaceuticals-awards/
In this episode Chloe & Alexander will be starting the discussion with an overview of EU HTA regulation, looking at the harmonization debate and how this came about. We then discuss JCA evaluation methodology (e.g. comparator selection, acceptability of clinical evidence), the potential new role of individual country agencies and how the joint HTA process will link to national processes. EUnetHTA21 and how the methodology is evolving with G-BA & IQWIG increasingly involved, will this be a major influence in the development of final methodology? Finally, we look at how Pharma companies should be best preparing for the implementation of the regulation. Host: Chloe Sheppard Guest Speaker: Alexander Natz Produced by: Operations team Helpful links: #EUHTAPriorities – EUCOPE’s Five Priorities for a Successful EU HTA Procedure Sounds of Science Podcast – Episode 1: A Future-Proof HTA system in Europe? https://partners4access.com/whitepapers/pan-european-joint-hta-what-does-the-future-hold-for-innovative-therapies/ About Alex: Alexander Natz is the Secretary General of the European Confederation of Pharmaceutical Entrepreneurs (EUCOPE) in Brussels and advises innovative pharmaceutical and biotech companies, including start-ups, in regulatory and pricing & reimbursement matters from the EU law and German law perspective. From 2008 to 2013, he was Head of the Brussels Office of Bundesverband der Pharmazeutischen Industrie e.V. (BPI). Before, he worked as a lawyer at Sträter Law Firm in Germany with a special focus on managed entry agreements and licensing of pharmaceuticals. Dr. Natz has also worked in the field of competition law with the European Commission and in the pharmaceutical industry. As a research assistant at Duke University (USA) he has dealt with international pharmaceutical law. His doctorate was supervised by the former judge at the European Court of Justice, Prof. Dr. Dr. Ulrich Everling Email: natz@eucope.org LinkedIn: https://www.linkedin.com/in/alexander-natz-63467433/ About EUCOPE: For more than a decade, the European Confederation of Pharmaceutical Entrepreneurs (EUCOPE) has been the voice of small to mid-sized health technology companies in Europe. As a trade association, we advocate for sound public policy that supports innovation, while fostering a community built on a shared purpose: improving and saving the lives of European patients through innovative therapies and medical technology. Website: https://www.eucope.org/
In 2020 China’s pharmaceutical market passed an important milestone, a Deloitte report cited that China was expected to grow its pharmaceutical market volume by 2020 to $220 billion (USD) becoming the second largest market behind the US. The commercial potential is huge, so what do we know about the orphan drug (OD) potential in China? Fisentzos Stylianou discusses China’s healthcare system, key challenges for OD manufacturers, Spinraza’s commercialisation journey in China, and incentives available to OD manufacturers in China. Reference: https://www2.deloitte.com/cn/en/pages/life-sciences-and-healthcare/solutions/life-sciences.html Presenter: Aparna Krishnan, Partner – Global Operations Contributor: Fisentzos Stylianou, Analyst Producer: Operations team
Over the last few years, the Middle East has piqued the interest of orphan drug manufacturers, and some have prioritised launching in this region before European markets. Akshay Kumar and Lavni Varyani discuss what is driving this behaviour, the orphan drug infrastructure within the region, and opportunities, barriers, and key considerations for manufacturers aiming to launch in the Middle East. Presenter: Akshay Kumar, Partner, P4A Contributor: Lavni Varyani, Founder, Pharma Business Partners Producer: Operations team
For this Rare Disease Day episode, Janet and Nick discuss a range of topics associated with the rare disease community and patient advocacy. They address new-born screening, patient registries, the UK rare disease framework, the NICE methods review and much more! Presenter: Georgie Rack, Communication Executive Contributors: Janet Bloor, CEO of Duchenne Nexus Advocacy (DNA) and Nick Meade, Director of Policy and Joint Interim Chief Executive of Genetic Alliance UK Producer: Operations team
Lots of anticipated changes are expected in the orphan drug, cell and gene therapy space and 2022 is going to be an exciting year. Listen to our latest podcast on the top trends in drug development, HTA and pricing from Akshay Kumar and Andrea Bernardini to help you navigate the year ahead. Presenter: Georgie Rack Speakers: Akshay Kumar & Andrea Bernardini Produced by: Ops team
Sophie Schmitz and Joanna Fernandes discuss bluebird bio’s innovative gene therapy Zynteglo and its withdrawal from the European market. Sophie and Joanna will be looking at the fall of Zynteglo from two sides: from a company perspective and from the perspective of the EU environment. Presenter: Georgie Rack, Communication Executive Contributors: Sophie Schmitz, Managing Partner and Joanna Fernandes, Senior Consultant Producer: Operations team
On Rare Disease Day, P4A discusses the role of policy-makers in the healthcare system and specifically, the issues surrounding cross-border healthcare as part of its '6P' campaign.    This directive is a key cornerstone legislation by EU officials that enables patients to find treatment in healthcare facilities outside their home country. However, there are several challenges associated with it.  Presenter: Christina Poschen Contributor: Dr Andrzej Rys, Director - Health Systems and Products Producer: Aparna Krishnan
In another special edition episode dedicated to the '6P' campaign to mark Rare Disease Day, the Weekly RoundUp team discusses the pharmaceutical perspective on partnerships in the rare disease space.   Presenter: Max Rex Contributor: Jan-Willem Schmitz, General Manager for Nordics and Baltics, Sanofi Genzyme Producer: Aparna Krishnan
 Throughout February, Partners4Access is running a campaign to mark the annual Rare Disease Day on February 28. The '6P' campaign is aimed at creating awareness on the impact of rare diseases on key stakeholders and the need for partnership to achieve successful treatment access for patients. The 6Ps are Partnership, Patient, Policy-maker, Physician, Pharma and Payer. This week we focus on the payer who are decision-makers assessing the value of a product in the healthcare system.   Presenter and Producer: Aparna Krishnan Contributor: Einar Andreassen, senior advisor at the Norwegian Medicines Agency. Einar is a health technology assessment analyst and reimbursement decision maker for the national insurance scheme in Norway.  
Throughout February 2019, the P4A team are hosting a series of special edition podcast episodes to mark Rare Disease Day. The initiative is part of a new 6P campaign aimed at promoting awareness about rare disease challenges and its impact on people and society. The 6Ps are Partnership, Policy-maker, Payer, Pharma, Physician and Patient. Our message: 6Ps are essential to successfully serve the rare disease community and achieve access to medicines. This week, we discuss the first P – Partnership. The team reflects on the meaning of partnership in the rare disease context and what it can achieve through examples of different types of collaborations. From patient and clinical experts influencing HTA decisions; key stakeholders coming together for the Hercules Project to a collaboration between US based ICER, Canada's CADTH and UK's NICE. Presenter: Aparna Krishnan Contributors: Sophie Schmitz, Christina Poschen, Max Rex, Joanna Fernandes, Nader Murad and Jack Rawson
 This week we are looking at the USFDA’s proposed plans to address the expected rise in cell and gene therapy product applications and a significant development in a new controversial procedure called gene drive. Also,  P4A is starting a new campaign that will run throughout February to mark Rare Disease Day.  The '6P'  campaign is aimed at creating awareness on the impact of rare diseases on key stakeholders and the need for partnership to achieve successful treatment access for patients. The 6Ps are Partnership, Patient, Policy-maker, Physician, Pharma and Payer. P4A will roll out a series of podcast episodes featuring thought leaders representing these stakeholders. So make sure you listen in! Presenter: Joanna Fernandes Contributors: Nader Murad and Sophie Schmitz Producer: Aparna Krishnan
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