Biogen Alzheimer’s Drug Gets Spotlight in FDA User Fee Hearing

Biogen Inc.’s Alzheimer’s drug was a main focus for lawmakers during the year’s first hearing on renewing the user fee agreements that will help fund the Food and Drug Administration.

Members of the House Energy and Commerce Committee’s health panel expressed confusion over Medicare’s preliminary decision to limit coverage of Aduhelm to patients enrolled in clinical trials, despite getting FDA approval.

“I was shocked to find out that the CMS proposed national coverage determination severely restricts Medicare for a whole class of Alzheimer’s treatments,” Rep. Cathy McMorris Rodgers (R-Wash.), ranking member of the full committee, said Thursday.

She and other lawmakers pointed to a disconnect between the decisions made by the FDA and the Centers for Medicare & Medicaid Services.

Patrizia Cavazzoni, director of the FDA’s Center for Drug Evaluation and Research, responded that the two agencies make decisions “independent of each other,” and that the Medicare agency made its own decision “on the basis of their own parameters and standards.”

She added that Aduhelm met the FDA’s standard for accelerated approval, as certain biomarkers indicated the drug had a likely clinical benefit for Alzheimer’s patients.

The FDA in recent years has increasingly granted expedited approvals, which are a key part of the agency’s commitment to bring more affordable, breakthrough drugs to market. But the accelerated pathway has come under fire after the FDA cleared Biogen’s Aduhelm in June 2021, despite two clinical trials offering mixed signals about the drug’s efficacy.

Thursday’s hearing marked the first in a series of actions on must-pass legislation to reauthorize the user fee agreements between the FDA and the prescription drug, generic, and biosimilar industries. Speeding up the pace of drug and product approvals is just one of several goals of these agreements.

Congress must pass laws adopting the new deals for fiscal years 2023 through 2027, before the current agreements expire Sept. 30.

Objectivity

The Aduhelm approval has exacerbated concerns over whether the industry user fees present a conflict of interest and limit the FDA’s ability to rule objectively on product approvals.

Rep. Debbie Dingell (D-Mich.) said Thursday that FDA reviewers must “remain objective and independent” when reviewing applications. She referenced reports of unofficial Biogen meetings with FDA staff ahead of the approval decision. The reports have prompted investigations by Congress and the FDA’s inspector general.

“What actions is FDA taking to ensure that its regulators are not inappropriately influenced by industry?” Dingell asked Cavazzoni.

The CDER head responded that the FDA’s decisions “are made independently of industry’s influence” and that agency staff hold “several levels of reviews” on product applications.

“We agree that it is very important to maintain that independence,” Cavazzoni said, adding that user fees will increase FDA staffing and help the agency maintain standards of safety and efficacy during accelerated product reviews.

Rare Diseases

Over the next five years, the FDA hopes to increase the number of staff members at the Center for Biologics Evaluation and Research to review more applications for cell and gene therapies, which work by transferring genetic material into the cell of a patient to provide long-lasting and potentially curative effects.

House members at the hearing also called on the FDA to make additional commitments in the user fee agreements to bring more rare disease treatments to market.

Rep. G.K. Butterfield (D-N.C.) noted that the vast majority of the roughly 7,000 known rare diseases in the U.S. remain without FDA-approved treatments.

Cavazzoni said it can be difficult to measure a treatment’s true efficacy in a clinical trial setting because of the small number of patients impacted by individual rare diseases.

The latest user fee agreement proposals include plans for a pilot program for the agency to provide more support to rare disease drug sponsors throughout the trial process.