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

Author: Partners4Access

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A weekly podcast highlighting the most important news developments and its impact on the orphan drug, cell and gene therapy world. Visit
52 Episodes
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 RawsonContributor: Sophie SchmitzProducer: 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 AllenContributor: Joanna FernandesProducer: 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 KumarContributor: Joanna FernandesProducer: 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 KrishnanContributor: Max RexProducer: 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 KrishnanContributor: Max RexProducer: 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 KrishnanContributor: Joanna FernandesProducer: 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 KrishnanContributors: Sophie Schmitz, Ciaran CassidyProducer: 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 KrishnanContributor: Jack RawsonProducer: 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 FernandesContributor: Christina Poschen, Aparna KrishnanProducer: 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 KrishnanContributors: Joanna Fernandes, Christina PoschenProducer: Aparna Krishnan
Weekly Roundup: May 18, 2019

Weekly Roundup: May 18, 2019


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 KrishnanContributor: Max RexProducer: Aparna Krishnan
This week, the P4A team discuss a new experimental therapy - Phage therapy or genetically engineered phages in order to treat bacterial infections and the acquisition of Theracon by Big Pharma firm Pfizer for upto $810 million.Presenter: Aparna KrishnanContributor: Ciaran CassidyProducer: Aparna Krishnan 
Weekly Roundup: May 3, 2019

Weekly Roundup: May 3, 2019


This week, we feature a special guest podcast speaker - Scott  Dorfman, chief executive officer of non-profit gene therapy developer Odylia Therapeutics on his personal journey and the future in gene therapy development. If you would like to know more about Odylia Therapeutics, please visit Presenter: Aparna KrishnanContributor: Scott Dorfman, CEO, Odylia TherapeuticsProducer: Aparna Krishnan
This week, we look at the European Federation of Pharmaceutical Industries and Associations or EFPIA’s patient wait survey and its key findings as well as news developments in the spinal muscular atrophy world relating to gene therapy Novartis’ Zolgensma and Biogen’s Spinraza.Presenter: Aparna KrishnanContributors: Sophie Schmitz, Jack RawsonProducer: Aparna Krishnan
 As part of the continuing World Orphan Drug Congress (WODC) special, this episode discusses the cGMP facilities and biosafety solutions from Germfree’s Carol Houts, P4A’s Sophie Schmitz looks back at the 2019 conference and Terrapinn’s Andre Singer talks about what to expect from WODC 2020. Presenter and Producer: Aparna KrishnanContributors: Carol Houts, Director of Regulatory and Quality, Germfree; Sophie Schmitz, Managing Partner, Partners4Access and Andre Singer,  General Manager for World Orphan Drug Congress USA, Terrapinn
This week, the P4A team are podcasting from the World Orphan Drug Congress in Washington D.C. We speak to André Choulika, Chairman and CEO of Cellectis on the challenges of commercializing a CAR-T cell product and ethical concerns surrounding it, and Anna Bucsics from the Mechanism of Coordinated Access to Orphan Medicinal Products (MoCA) on their work supporting new biotech entrepreneurs.Presenter: Joanna FernandesContributors: André Choulika, Chairman and CEO of Cellectis; Anna Bucsics, Project Advisor to MoCA and Sophie Schmitz, Managing Partner, P4AProducer: Aparna Krishnan
The team discuss the creation of a new health economics advisory committee in Spain expected to influence pricing and reimbursement decisions for drugs; and Japan’s approval of its first gene therapy and CAR-T therapy  - AnGes' HGF Plasmid and Novartis' Kymriah respectively. Presenter: Joanna FernandesContributor: Max RexProducer: Aparna Krishnan
This week, we are looking at The Institute for Clinical and Economic Review or ICER’s white paper on alternative options for the US rebate system, and the establishment of a national agency to evaluate drug effectiveness and negotiate prices in Canada. Presenter: Max RexContributor and Producer: Aparna Krishnan
This week, we look at Ireland's access to orphan drugs as compared to rest of western Europe and the first outcomes deal for CAR-T cell therapies in Germany. Presenter: Joanna FernandesContributors: Nader Murad, Ciaran CassidyProducer: Aparna Krishnan
This week, the P4A team discuss the spate of mergers and acquisitions involving Big Pharma companies in the gene therapy space. Particularly, we looked at the deals behind Roche's acquisition of Spark Therapeutics and Biogen's buyout of Nightstar. Also, Vertex's continued struggle to get its cystic fibrosis drug Orkambi reimbursed in the UK. Presenter: Joanna FernandesContributor and Producer: Aparna Krishnan
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