Health Affairs November 13, 2023
On the face of it, the Food and Drug Administration’s (FDA’s) regulatory mandate is straightforward: Approve drugs that are safe and effective for their intended use. However, as demonstrated by several recent decisions regarding drugs to treat conditions such as Alzheimer’s disease, Duchenne muscular dystrophy, and amyotrophic lateral sclerosis (ALS), the reality is often far more complex, necessitating a balance between speedy patient access and reasonable certainty about drug benefit. When should the FDA demand more evidence, even if it will mean that a promising medicine might take longer to reach patients? And when should it exercise “regulatory flexibility” based on the risk tolerance often exhibited by those facing terrible diseases without good treatment options?
In answering these questions, it’s...
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