Undoubtedly, the week’s biggest news was the FDA’s approval of Biogen’s Aduhelm for the treatment of Alzheimer’s disease. ICER has already issued a statement sharing our reaction, but here’s a quick rundown of the facts:
- While Aduhelm’s potential benefits are uncertain, its side effects are both common and potentially serious.
- The FDA granted Aduhelm an accelerated approval — a pathway intended to expedite drugs to market with surrogate evidence in the rare situations where the preferred method of measuring actual clinical endpoints would simply take too long — even though Biogen’s two pivotal trials already measured the actual clinical endpoints.
- Despite the FDA’s earlier insistence that “we’re not using the amyloid as a surrogate for efficacy,” the Agency ultimately did approve Aduhelm based on its ability to reduce amyloid, a surrogate endpoint achieved by many previous investigational therapies that neither improved patients’ cognition nor achieved FDA approval.
- The FDA’s decision to ignore its own Advisory Committee’s recommendation against approval has already caused
one twothree committee members to resign in protest.
- While Aduhelm’s label covers more than 6 million Americans, Biogen has suggested a more realistic potential patient population of 1-2 million, which at the treatment’s price could feasibly deliver annual revenues to the drug maker in excess of $50 billion per year, making Aduhelm the most lucrative therapy in the US market.
- With Medicare Part B’s 20% coinsurance and no max, many Medicare beneficiaries will be required to pay more than $10,000 out-of-pocket each year for Aduhelm.
- While Aduhelm will drive immediate profits for Biogen, the FDA is allowing the company until 2030 to confirm whether or not Aduhelm actually improves patients’ lives.
- Even many voices within biopharma disagree with Aduhelm’s approval and price.
While those are the known facts, many important questions remain: How will patients be diagnosed? How will insurers cover Aduhelm? How will patients be screened for possible side effects? How will Biogen and the FDA design the confirmatory trial? And how can Aduhelm be introduced into practice in a way that addresses the systemic disparities across our health care system? At the ICER public meeting on Aduhelm on July 15, we’ll tackle these important issues with all stakeholders at the table. And we’ll also address the question of fair pricing for a drug that now seems likely to become one of the top selling therapies in the history of the United States.