In order to approve a new Alzheimer’s treatment, the Food and Drug Administration is rewriting its rulebook, an enormously risky move that could accelerate the public’s access to medicines but upend the future of drug regulation, forcing the 114-year-old agency to do the equivalent of redesigning a fighter plane in mid-flight.
With its conditional clearance of Biogen’s Aduhelm, the FDA is creating a framework that could require less certain evidence of safety and efficacy for medicines beyond Alzheimer’s treatments. In doing so, it is choosing to alter many of its well-established processes and standards, seemingly on the fly.
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This situation is yet another argument to come up with a statutory framework for conditional approval subject to strict evidence-creating pathways and milestones (coverage with evidence-development), rather than an on-off switch. Accelerated approval is FDA’s way of kind-of approving with contingencies. The open question will be whether we will get the evidence-building once this hits the streets. Will we capture the information needed to make a cool-headed decision several years hence or not?
Absolutely. The FDA’s primary job is to protect Americans from unsafe drugs and treatments. As far as pre-market efficacy requirements, they should be lower for cases of unmet need or life-threatening / devastating / terminal illnesses and much higher for things like acne treatment or birth control. It makes sense in terms of expected utility – there is an ethical imperative to provide drugs faster and also provide drugs that have a lower probability of working because if they do work the benefit is much higher.
There is evidence that FDA is overly cautious and that type I errors (failure to approve useful drugs) occur at an unacceptably high in many areas (see https://fivethirtyeight.com/features/how-the-fda-could-change-the-way-it-approves-drugs/).
Some people like the idea of FDA having a really high bar for efficacy before drugs can go to market. But a high bar discourages investment in risky ideas, slows time to market, and pushes up costs. One option is to implement some form of adaptive licensing or tiered approvals (see my brief description here: https://moreisdifferent.substack.com/p/a-laundry-list-of-possible-fda-reforms).