Democratic leaders of two House committees are stepping up a probe of the Food and Drug Administration's (FDA) controversial approval of the Alzheimer's drug Aduhelm, pressing the agency for information on "anomalies" in the approval process.
In a letter to FDA sent Wednesday, House Energy and Commerce Committee Chairman Frank Pallone Jr.Frank Joseph PalloneHouse Democrats announce bill to rein in tech algorithms House Democrats ramp up probe of FDA approval of Alzheimer's drug Intercept bureau chief: Democrats dropping support of Medicare for All could threaten bill's momentum MORE (D-N.J.) and Oversight and Reform Chairwoman Carolyn MaloneyCarolyn MaloneyHouse Oversight Democrats ask NFL for information from investigation into Washington Football Team New York City helicopter complaints skyrocket Trump company in late-stage talks to sell DC hotel: report MORE (D-N.Y.) said "significant questions" remain even after FDA staff briefed their panels.
The lawmakers launched the investigation in July, and requested a slew of documents from the pharmaceutical company Biogen over its Alzheimer's treatment.
FDA granted accelerated approval for the drug on June 7. Aducanumab, sold under the name Aduhelm, was the first new Alzheimer's drug approved in nearly 20 years, but the approval came over the near-unanimous objections of the FDA's expert advisory committee.
Three members subsequently resigned.
Additionally, the approval came after two large-scale trials to gauge the drug’s efficacy were halted early because they were deemed unlikely to work.
According to the lawmakers, documents made public after the drug’s approval show that experts in FDA’s Office of Biostatistics raised concerns about the “inconsistency” of the medications supporting clinical data.
In one instance highlighted in the letter, a member of the Medical Policy and Program Review Council, an internal group that helps set agency policy, raised concerns that approval of the drug could “result in millions of patients taking aducanumab without any indication of actually receiving any benefit, or worse, cause harm” from side effects.
Among the information now sought by Pallone and Maloney is details on how FDA makes an approval determination when there is internal disagreement between agency staff as well as disagreement with the relevant advisory committee.
The lawmakers also questioned the agency’s interactions with Biogen during the review process, something that is also being reviewed by an independent government watchdog.
Aduhelm is not a cure for Alzheimer's, and the FDA made its decision without clear evidence that the drug can even slow the progression of the disease. Instead, the agency based its approval on how well it reduces the amount of plaque in the brains of Alzheimer's patients. Brain plaques are thought to contribute to Alzheimer's disease, but it has not been proven.
Biogen has also drawn criticism for Aduhelm's pricing. The drug is priced at about $56,000 a year per patient, and experts have raised concerns about the effect on Medicare spending.
Since the drug's approval, the lawmakers noted that major medical centers such as the Cleveland Clinic and Mount Sinai have decided not to administer Aduhelm to their patients, large health insurers have declined to cover the drug and the Department of Veterans Affairs has declined to add the drug to the VA National Formulary.