New Alzheimer's drug is a problem for FDA's pass-fail approach

From the The Washington Post (Opinion)

By Jerry Avorn, MD

SUMMARY: In this Washington Post opinion piece, Dr. Avorn argues that the FDA's binary approve-or-reject framework is structurally ill-suited to drugs like Leqembi (lecanemab), which occupy a gray zone of modest benefit and meaningful risk. Leqembi offers only modest benefit for Alzheimer's disease, could pose worrisome safety risks, and stands to cost the nation $2 billion to $5 billion per year. Avorn contends that even when a drug's clinical value is marginal or its risk-benefit balance uncertain, the FDA's decision nearly always comes down to full approval or rejection — with no intermediate pathway that might allow conditional or restricted use for specific patient populations. The piece calls for structural reform of FDA approval processes to allow for nuanced, graduated decisions that better reflect clinical reality for patients and prescribers.

BACKGROUND: The FDA was approaching a full approval decision for Leqembi, a new Alzheimer's disease drug that had received accelerated approval on the basis of a surrogate endpoint.

KEY FINDINGS: Leqembi offers only modest benefit, could pose worrisome safety risks, and stands to cost the nation $2 billion to $5 billion per year. The FDA's approval framework is effectively pass-fail, providing no intermediate pathway for drugs with ambiguous risk-benefit profiles.

IMPLICATIONS: The FDA needs structural alternatives to its current all-or-nothing approval approach, particularly for drugs where benefit is modest and risk or cost is substantial.