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 https://everylifefoundation.org/ 

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

In this episode, P4A speaks to a special guest - Alliance of Regenerative Medicine's chief executive officer Janet Lambert on the ARM's key initiatives such as the Foundation of Cell and Gene Therapy medicines as well as the 2019 report on ATMPs (Advanced Therapy Medicinal Products).

Presenter: Aparna Krishnan

Contributor: Janet Lambert, CEO of Alliance of Regenerative Medicine

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