FDA chief acknowledges potential stumbles in approval process for Biogen’s Alzheimer’s drug

Janet Woodcock, the acting commissioner of the US Food and Drug Administration.Al Drago/Bloomberg

Janet Woodcock, the head of the US Food and Drug Administration, on Wednesday defended her agency’s decision to approve Biogen’s controversial Alzheimer’s disease therapy but acknowledged potential stumbles that may have undermined public trust in the process.

Speaking at the virtual 2021 STAT Breakthrough Science Summit, Woodcock said the approval of Aduhelm, which is conditioned on Biogen completing another clinical study to prove the drug works, was “based on very solid grounds” and the “right thing to do for patients.” Still, she’s looking into the steps her agency and Cambridge-based Biogen took during the approval process, to address outside concerns.

Last week she sent a letter to the Department of Health and Human Services’ inspector general, a watchdog that oversees the FDA, calling for an investigation into the interactions between the drugmaker and her agency’s review staff, including “some that may have occurred outside of the formal correspondence process.”

“Others have expressed concern, and we need to evaluate those concerns and put them at rest or deal with them if they are found to be valid,” she said Wednesday.

The acting commissioner said she does not take issue with the disagreement in the scientific community about whether the drug works, or what data her agency’s staff used to make its decision.

“Was the process done exactly the best that it could be? Possibly not,” she said. “It is possible the process could have been handled in a way that would have decreased the amount of controversy involved.”

The approval of the drug has drawn criticism for other reasons, beyond whether the communication between the FDA and Biogen violated any rules.

For example, when the FDA approved Aduhelm on June 7, the agency did not limit its use to a specific patient population. However, roughly a month after the approval and following concern from physicians, the FDA updated the drug’s label, indicating it should only be prescribed to patients with early symptoms of the disease, the population Biogen studied in its clinical trials. Woodcock said it was her understanding that the agency’s neuroscience group was following its “established policies for neurodegenerative diseases” when it approved the broad label.

”I think after getting feedback, they realized they should be more descriptive about who was in the trial and what was known about those not in the trial,” she said.

After the approval, three members of the FDA expert panel that advised against authorizing the new drug quit in protest, and a handful of private insurers have said they would not cover the cost of the treatment.

Following the FDA chief’s call for a federal probe, Michael Carome, a director at public health watchdog Public Citizen, wrote to the inspector general that the agency must also “examine the role played by Dr. Woodcock in this troubling matter.” During the STAT interview Wednesday, Woodcock did not acknowledge whether she knew about the “off-the-books” meeting between an FDA regulator and Biogen, first reported by STAT, which led to the resurrection of Aduhelm after the drug’s prospects appeared to be dead.