This week, the Alzheimer’s Association sponsored, and the Hill hosted, a discussion on accelerated approval with policymakers, experts and patient advocates. Through the Food and Drug Administration’s (FDA) accelerated approval pathway, people living with an unmet medical need — like Alzheimer’s and other dementia — can gain earlier access to new and innovative treatments.
Alzheimer’s is a fatal disease with no survivors. New and better treatments — especially that use novel mechanisms of action — are urgently needed for Alzheimer’s and other dementia.
Thanks to the accelerated approval pathway we’ve seen tremendous progress in our nation’s treatment of diseases. This has meant so much to so many individuals facing a tough or fatal diagnosis.
In the discussion moderated by the Hill’s Bob Cusack, Dr. Mark McClellan, a former FDA Commissioner, a former CMS Administrator and the Founding Director of the Duke-Margolis Center for Health Policy, Duke University, spoke on the importance of the accelerated approval pathway. “The evidence base is going to change a lot in the space of Alzheimer’s disease monoclonal antibody drugs over the next year. There are three other drugs that are in pivotal FDA clinical trials now that are expected to have results in the coming weeks and months, not just on the biomarker, but within the next year on the actual clinical endpoints that the FDA approved demonstrating benefit in terms of slowing the progression of loss of cognitive function in Alzheimer’s disease.”
On the relationship between FDA and CMS, Neil Thakur with the ALS Association noted, the expectation is “once FDA approves a drug, people are going to be able to access it. I think a very disturbing trend, not just within CMS but outside CMS, is we’re trying to control the price of drugs by limiting access to those drugs. That’s not a great approach. If we have a drug and we think it has benefit, then a doctor and a patient should be able to work those things out together and those drugs should be affordable and accessible, especially when we’ve identified systemic bias all throughout our health systems.”
The event also featured remarks by:
- Rep. Neal Dunn, MD (R-FL), Member, Energy & Commerce Committee & Doctors Caucus
- Rep. Brian Higgins (D-NY), Sponsor, Comprehensive Care for Alzheimer’s Act
- Rep. Brad Wenstrup, MD (R-OH), Co-Chair, Doctors Caucus; Member, Ways and Means Subcommittee on Health
- Cynthia Rice, Chief Mission Strategy Officer, JDRF
- Jamie Sullivan, Senior Director for Policy, EveryLife Foundation for Rare Diseases
During the event, Joe Montminy, a person living with Alzheimer’s disease, shared his thoughts on accelerated approval. “I don't have the luxury of time. Accelerated approval gives people like me hope — hope that I might retain my cognitive skills a bit longer, hope that it will be awhile until I need assistance getting dressed or eating, hope that I can live at home with my family as long as possible.”
Learn more and watch the event here.