Welcome to this month's episode of the Let’s Talk Rare: The Life Science Podcast by Partners4Acess. Georgie and Owen are joined by Nick Meade, Head of Policy at Genetic Alliance, to discuss the challenges faced by rare patients in accessing life-saving medicines. Together they explore the EU joint HTA legislation coming into force in January 2025, and the importance of patient experience data. Nick explains that the challenges for rare patients remain the same, with diagnosis being the first hurdle. He highlights the progress being made in genetic diagnoses and screening but emphasizes the nee ..read more

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 ..read more

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 ..read more

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 https://everylifefoundation.org/  Presenter and Producer: Aparna Krishnan Contributor: Annie Kennedy, Chief of Policy & Advocacy, Ever ..read more

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 ..read more

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 ..read more

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 ..read more

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 ..read more

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 ..read more

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 ..read more