Is It Time for More Non-profits to Lead Reimbursement and Commercialization of Gene Therapies to Promote Affordable and Sustainable Access?

ISSUE:

Licensed gene therapies have been withdrawn from the European market by for-profit (pharmaceutical or biotechnology) manufacturers for ‘commercial reasons’, including challenging reimbursement pricing, expensive post-launch data requirements, high manufacturing costs and small patient populations. Conversely, the non-profit, Telethon Foundation, recently took over the commercialisation of Strimvelis in Europe after it was discontinued by its for-profit manufacturer. Furthermore, the Access to Gene Therapies for Rare Diseases (AGORA) initiative was recently established to support non-profits with sustainable access to gene therapies, demonstrating the growing appetite for non-profit commercialisation.

It will be important to understand whether non-profits have the resources and solutions to overcome the challenges of gene therapy commercialisation. Could the anticipated challenges of the upcoming Joint Clinical Assessment (JCA), including differing requirements for assessing orphan treatments across member states, be tougher for non-profit manufacturers than for-profit manufacturers? With patient communities most affected by the availability of gene therapies, what is their take on the issue?

OVERVIEW:

Kate Hanman will summarise the commercial challenges leading to gene therapy withdrawals (10 minutes). During the ensuing discussion (35 minutes), Annabel Griffiths will share a patient community’s experience of a withdrawn gene therapy and discuss the risks and benefits from the patient perspective around for- and non-profit-led commercialisation. Mohit Jain will discuss how for-profit manufacturers have the resources needed for sustainable commercialisation of gene therapy, including post-launch data requirements. Arjan Lankester will discuss how non-profits can support licensing, delivery and affordable access to treatments which are otherwise commercially unsustainable. Finally, Kate will ask the panellists how non-profit and for-profit manufacturers will navigate the anticipated challenges of JCA. The audience will vote on whether more non-profits should commercialise gene therapies at the start and end of the session, to see if the debate changes their opinion. Time for the audience’s questions will be allocated (15 minutes).