Rare Diseases Weekly Roundup

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

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 Krishnan Contributor: Ciaran Cassidy Producer: Aparna Krishnan  

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  https://odylia.org/  Presenter: Aparna Krishnan Contributor: Scott Dorfman, CEO, Odylia Therapeutics Producer: 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 Krishnan Contributors: Sophie Schmitz, Jack Rawson Producer: 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 Krishnan Contributors: 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 Fernandes Contributors: André Choulika, Chairman and CEO of Cellectis; Anna Bucsics, Project Advisor to MoCA and Sophie Schmitz, Managing Partner, P4A Producer: 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 Fernandes Contributor: Max Rex Producer: 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 Rex Contributor 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 Fernandes Contributors: Nader Murad, Ciaran Cassidy Producer: 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 Fernandes Contributor and Producer: Aparna Krishnan

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