“I think there’s a tremendous opportunity for long-term, outcomes-based and/or payment-over-time models to serve as that bridge and address that uncertainty between short-term available data at launch and the long-term value these therapies can provide,” bluebird bio VP-access, value and evidence strategy Shannon Baumann said during a recent panel discussion at Alliance for Regenerative Medicine’s virtual Cell & Gene Meeting on the Mesa.
Biopharma executives discussed their strategies for treating chronic diseases during the Cell and Gene Meeting on the Mesa, including R&D and regulatory considerations.
Speaking at the virtual Cell & Gene Meeting on the Mesa on 14 October, Center for Biologics Evaluation and Research director Peter Marks said the agency assesses the importance of durability of effect differently for a gene therapy that treats a disease that has no other available therapies, versus a condition for which there are multiple approved treatments.
Value-based contracting in the US is moving toward high-cost drugs for rare disease and away from lower cost chronic treatments as payers plan for the coming pipeline of cell and gene therapies. Marketed gene therapies all have some kind of risk sharing deal in place but further progress on the most innovative payment models is hampered by regulatory and operational challenges.
The Alliance for Regenerative Medicine said that once the agency feels it has enough experience, it should share its collective experience as general principles for why orphan drug designation or exclusivity is or is not granted.
A first half of the year progress report from the international advocacy group Alliance for Regenerative Medicine (ARM), finds that the regenerative medicine and advanced therapy sector is in very good shape and has performed well in terms of both clinical development and fundraising despite the challenges posed by the ongoing COVID-19 pandemic.
European companies developing advanced therapies such as gene and cell therapies secured at least €2.2B ($2.6B) in the first half of 2020. While this is encouraging in the face of COVID-19, it hasn’t been smooth sailing for all startups in the field, with many struggling to stay afloat during crippling national lockdowns.
