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Rare Disease, Cell & Gene Therapy Monthly RoundUp
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Rare Disease, Cell & Gene Therapy Monthly RoundUp

Author: Partners4Access

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A monthly podcast highlighting the most important news developments and its impact on the orphan drug, cell and gene therapy world. Visit
61 Episodes
In this month's episode, the P4A team take a deep dive into the impact of Brexit on the UK's market access landscape. Four months after leaving the European Union, the UK's attractiveness as a key destination for commercialisation of new drugs is being tested. In order to improve its credentials, the UK is exploring a slew of measures that could potentially spark a reform.  But will that happen? P4A's Joanna Fernandes and Jayne Watson consider two distinct initiatives - MHRA's (Medicines Healthcare Regulatory Agency's) new I-LAP (Innovative Licensing and Access Pathway) scheme and England's HTA body NICE's (National Institute for Health and Care Excellence's) method review.  Presenter and Contributor: Joanna Fernandes Other contributors: Jayne Watson Producer: Aparna Krishnan   
The world of orphan drugs is at the cusp of a transformation in the post COVID era. In this episode, the team discuss the impact of the pandemic on value demonstration of orphan drugs. In order to be successful, manufacturers of precision medicine will need to focus on carving out a niche for their drugs and support health systems to find the right patient for their treatment at the right time.  The team also explore the role of digital tools such as Artificial Intelligence (AI) and telemedicine in helping drugmakers optimise manufacturing and treatment delivery. This combined with a multi-stakeholder approach will provide the recipe for success for next generation`niche-busters'. Presenter and Contributor : Akshay Kumar Other contributors: Richard Wang, Andrea Bernardini and Erfan Akbraian Producer: Aparna Krishnan
In this episode, the P4A team discussed the impact of cost-containment measures in Germany, the European Commission's recent move to revise legislation on orphan medicinal products and potential legislative reforms in the US under the new Biden administration.  Presenter: Jens Leutloff Contributors: Chloe Sheppard, Max Rex Producer: Aparna Krishnan
In this Rare Disease Day special episode, we focus on the impact of COVID -19 on rare disease patients. Some of the key challenges for patients during this pandemic include disruption in their access to treatments, struggle to continue participation in clinical trials, lack of access to vital equipment such as PPE (Personal Protective Equipment) and also to healthcare personnel. We speak to Genetic Alliance chief executive officer Jayne Spink, patient Nicola Whitehill and Danielle Myers, mother and carer of patient 10 year old Dylan Myers. Presenter: Aparna Krishnan Contributors: Jayne Spink, Nicola Whitehill and Danielle Myers Producer: Aparna Krishnan More information on : Genetic Alliance can be found at Nicola Whitehill's blog:  Dylan Myers' story:  and
In this episode,  the P4A team review all the key events of the year 2020 in the orphan drug, cell and gene therapy world. The emergence of COVID-19 pandemic has caused disruption but also opportunities for the biotech industry. From  change in regulations, new stakeholder collaborations to impact of Brexit and US drug pricing reforms,  a full round up of 2020. So do listen in! Presenter and Contributors: Sophie Schmitz  and Akshay Kumar Producer: Aparna Krishnan
This episode discusses patient access to AveXis' gene therapy Zolgensma from Orsini Healthcare, US based specialty pharmacy's perspective.  The senior team at Orsini  provide insight on their experience of Zolgensma access including key manufacturer criteria, payer landscape along with challenges and lessons learnt.  Presenter: Aparna Krishnan Contributors: Dave Frobel, Senior Vice President, Trade & Tom Shaughnessy, Senior Vice President, Health Plans     Producer: Aparna Krishnan About Orsini Healthcare: Founded in 2009, Orsini is a leading independent specialty pharmacy focused on rare conditions and gene therapies. Orsini was one of the first specialty pharmacies to provide Zolgensma, the first gene therapy for Spinal Muscular Atrophy. Today, Orsini has access to 40 limited distribution drugs, has more than 525 direct payor contracts and is recognized as a reimbursement expert in managing both medical and pharmacy benefits.
This week is a special episode celebrating Rare Disease Day 2020.  Our guest speaker is Annie Kennedy, head of policy and advocacy at Every Life Foundation, a US based non profit organisation that works to advance the development of treatment and diagnostic opportunities for rare disease patients through science-driven public policy. Here she speaks about the organisation's initiatives on drug access.  To know more about the foundation, visit  Presenter and Producer: Aparna Krishnan Contributor: Annie Kennedy, Chief of Policy & Advocacy, EveryLife Foundation for Rare Diseases
As 2019 winds down, P4A's Sophie Schmitz and Akshay Kumar discuss the key trends that defined this year  -  marketing approval of cell and gene therapies; pricing and reimbursement challenges in US & EU5 as well as industry merger and acquisitions.  Presenter: Sophie Schmitz Contributor: Akshay Kumar Producer: Aparna Krishnan
The team discuss the latest update on the proposed EU health technology assessment (HTA) regulation. The key objectives of the 2018 proposal was to promote convergence in HTA tools, procedures and methodologies; reduce duplication of efforts for HTA bodies and industry and improve joint use of outputs. This episode goes indepth into the one of the controversial aspects of the proposal - The joint clinical assessment - that caused a near stand still in negotiations between member states.  Presenter: Christina Poschen Contributor: Ciaran Cassidy Producer: Aparna Krishnan
The team discuss the NORD summit particularly FDA commissioner Scott Gottleib’s presentation, panel discussion on cell and gene therapy pricing as well as P4A’s lunch and learn on cell and gene therapy access – learnings from the EU. Also in this episode, Novartis’ Kymriah innovative payment agreement in Italy, the changing evaluation trends for gene therapies among HTA bodies and Alliance for Regenerative Medicines’ Q3 report. Presenter: Jack Rawson Contributor: Sophie Schmitz Producer: Aparna Krishnan
Following an autumn hiatus, the team come back to discuss Vertex's journey to commercialize Orkambi picking up the story from the latest deal struck with NHS England in October 2019.  Presenter: Nicola Allen Contributor: Joanna Fernandes Producer: Aparna Krishnan
The team takes a look at the most recent legislation in Germany known as GSAV or the law for more safety in the supply of pharmaceuticals and its implications on the access to orphan drugs. GSAV was approved by the German cabinet on January 30, 2019 and is due to come into effect in August this year.  Presenter: Akshay Kumar Contributor: Joanna Fernandes Producer: Aparna Krishnan
This week, the P4A team discuss the coverage of Novartis' Zolgensma in the US in particular how US health insurers have received the gene therapy. Also on the agenda was the impact of Zolgensma's launch on rival Biogen's Spinraza and the potential for dominance of the gene therapy in the spinal muscular atrophy landscape.  Presenter: Aparna Krishnan Contributor: Max Rex Producer: Aparna Krishnan
The debate over US drug pricing reform has reached fever pitch as the US Department of Health and Human Sciences announced its latest Medicare Part B proposal to include international reference pricing. The Partners4Access team looks at the background to this proposal; the political and patient organisation criticisms; the potential industry response and its ramifications.  Presenter: Aparna Krishnan Contributor: Max Rex Producer: Aparna Krishnan
This week, we analyze Bluebirdbio's commercialization plans for its gene therapy Zynteglo including the new payment model and a delay in launch due to manufacturing issues.  Presenter: Aparna Krishnan Contributor: Joanna Fernandes Producer: Aparna Krishnan
This week, the team discuss the emergence of 'drug buyer's clubs' in the UK involving Vertex's cystic fibrosis drug Orkambi.  Presenter: Aparna Krishnan Contributors: Sophie Schmitz, Ciaran Cassidy Producer: Aparna Krishnan
This week, the team discuss Vertex's acquisition of Exonics and expansion of its collaboration with CRISPR Therapeutics. Also, we take an indepth look at the European Medicines Agency's approval of Bluebirdbio's gene therapy Zynteglo as a treatment for beta-thalassemia .  Presenter: Aparna Krishnan Contributor: Jack Rawson Producer: Aparna Krishnan
On our anniversary episode, the P4A team discuss the USFDA approval of Novartis' Zolgensma, a one time gene therapy for paediatric spinal muscular atrophy patients and a look back at the last 12 months of our podcasting journey.  Presenter: Joanna Fernandes Contributor: Christina Poschen, Aparna Krishnan Producer: Aparna Krishnan
In this episode, we look at Novartis CEO Vas Narasimhan calling for a change in the US drug payment systems, arguing for new economic models to identify how much value a cure represents. Also, Italy's attempts at introducing a draft resolution to improve transparency in drug pricing at the World Health Assembly. Presenter: Aparna Krishnan Contributors: Joanna Fernandes, Christina Poschen Producer: Aparna Krishnan
This week, the team discuss the deal between UK's National Health Service and Biogen prompting the successful reimbursement of Biogen’s Spinraza as a treatment for spinal muscular atrophy and Solid Biosciences’ clinical update on its Duchenne Muscular Dystrophy gene therapy product SGT-001.   Presenter: Aparna Krishnan Contributor: Max Rex Producer: Aparna Krishnan
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