House committees step up investigation into FDA approval of controversial Alzheimer’s drug

Sep 2, 2021
In The News

Two House committees are stepping up their investigation into the Food and Drug Administration’s approval of a controversial Alzheimer’s drug, requesting a raft of information on how the agency made the decision, who was involved and whether staff interactions with the drug’s manufacturer were appropriate.

The two panels — Energy and Commerce and Oversight and Reform — wrote in a letter to the agency that they were “concerned by apparent anomalies in FDA’s processes” involving its review of Aduhelm, a drug made by Massachusetts-based Biogen.

The agency’s June 7 accelerated approval of the medication, the first Alzheimer’s drug cleared in almost two decades, was welcomed by some advocates and doctors as a step forward in treating the devastating disease but strongly criticized by many experts who say the medication hasn’t been shown to be effective. The decision was especially controversial because the agency’s own outside experts had recommended against approval. Some medical centers have refused to offer the drug, which carries a list price of $56,000 a year per patient.

Many of the panels’ questions involve why the agency granted the drug accelerated approval, which is based on a “surrogate endpoint” thought to predict a clinical benefit. In Aduhelm’s case, the endpoint is the drug’s effectiveness at removing sticky clumps of a brain protein known as beta amyloid. Many scientists say removing that plaque has not been shown to slow disease. The agency and Biogen have countered that Aduhelm is much more effective at targeting plaque than prior drugs, suggesting it will provide a benefit.

The committees noted that the FDA briefed the staffs of the committees in late July and provided minutes of an internal FDA meeting in which Aduhelm’s fate was discussed. But they said significant questions remained and asked for details on other agency deliberations. They also asked how many times the FDA has granted approvals that an agency advisory committee had opposed. The letter was signed by Reps. Frank Pallone Jr. (D-N.J.), chairman of the Energy and Commerce Committee, and Carolyn B. Maloney (D-N.Y.), chairwoman of the Oversight and Reform Committee.

The FDA, in a statement, said it reviewed Aduhelm using “a science-based approach that incorporated consideration of patient perspectives in its evaluation of the benefits and risks of this therapy.” It said it will respond to the congressional requests.

The panels also are seeking information on the FDA’s contacts with Biogen, going back more than three years, and on the FDA’s assessment of those interactions. The committees want to know why the FDA is giving the manufacturer up to nine years to complete a follow-up study to show whether the drug truly helps patients.

The furor surrounding the drug prompted acting FDA commissioner Janet Woodcock in July to ask the inspector general of the Department of Health and Human Services to investigate the agency’s interactions with Biogen and other issues related to Aduhelm. In August, the inspector general’s office said it would conduct a far broader probe, including the agency’s use of the accelerated approval pathway, which is frequently used for cancer drugs.

In an interview this summer, Woodcock defended the Aduhelm approval, saying it was “very solid.” She said Congress has encouraged the agency to use its accelerated-approval authority to help patients with devastating diseases and few treatments.