Max Nisen: FDA error is already getting worse | Chroniclers


Review by Max Nisen Bloomberg

When the United States Food and Drug Administration approved Biogen Inc.’s controversial Alzheimer’s drug Aduhelm earlier this month without strong evidence that the drug helps patients, it raised concerns that others unproven treatments may follow. This scenario is already there.

Eli Lilly & Co. has announced plans to file for fast track approval this year for its investigational Alzheimer’s treatment, donanemab, due to its ability to remove amyloid brain plaques that some believe cause the disease. . It’s the same rationale and the same unprecedented route that the FDA used for Aduhelm. Lilly is clearly responding to the agency’s offbeat goals; in April, the company told investors it would not seek a quick approval because the regulator said there was not enough data.

But it was the old FDA. It is now possible that the agency will approve another drug based on an unproven idea. Lilly shareholders seem to think so – the company’s shares rose 8% on Thursday. And yet, while the approval would boost Lilly’s earnings prospects, it would make the mistakes of the Aduhelm decision worse and further hurt future Alzheimer’s research and the United States’ healthcare budget.

Removing the amyloid plaque has not helped patients in many previous trials, and using it as the sole basis for Biogen treatment approval has come as a surprise. The FDA released Alzheimer’s drug guidelines in 2018, rejecting these secondary measures due to scientific uncertainty. An external expert panel tasked with evaluating Biogen’s drug in November was told the agency was not considering this option. But when the group voted that conflicting evidence from Biogen’s trials did not support approval, the FDA found a new way forward. Three members of the expert panel resigned in protest.

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The FDA released a memo outlining their rationale for getting into amyloid. He cites analyzes supporting a link between reduction in plaque and slower patient decline and explaining the messy data from Biogen. But these analyzes have not been reviewed by outside experts and are worryingly biased.

Lilly’s quest for approval won’t clear things up. Donanemab may be even better than Biogen at removing amyloid. But the evidence of benefit to patients is again mixed, coming from only 257 patients. The trial appeared to slow cognitive decline, but the effect was slight and visible only on a scale that had not previously been used widely in Alzheimer’s disease trials. The drug has shown no benefit over a variety of other measures.

The consequences of another expedited approval would be dire. This would attract more R&D dollars towards amyloid drugs, which appear to have a very mild effect at best.

The FDA can right the ship. Lilly is already conducting a larger trial that may provide better evidence for its drug and the amyloid hypothesis as early as 2023. Results of other drugs targeting amyloid from Biogen and Roche Holding AG may also be available by then. By waiting a bit and doing a public assessment of the evidence for amyloid with more data, the agency may be resetting expectations and its standards instead of opening the barn door wider.

Max Nisen is a Bloomberg Opinion columnist.



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