A Congressional Food and Drug Administration investigation into the Alzheimer’s treatment review process found that the agency “deviates” from its standard procedures to approve the drug Aduhelm based on inconsistent data.
Aduhelm is known for being the first drug in recent years to be approved to treat the underlying cause of Alzheimer’s, a disease with no known cure characterized by progressive dementia that affects more than six million Americans, according to the Alzheimer’s Association. :
An 18-month joint investigation between the House Oversight and Reform Committee and the Energy and Commerce Committee, released Thursday, criticizes the FDA for “atypical cooperation and interaction” with the drug’s maker, Biogen.
In statements, Biogen and the FDA both said their interactions leading up to Aduhelm’s green light were appropriate in the context of the approval process.
“One of my top priorities as Chair of the Oversight and Reform Committee is to ensure that the American people have access to effective and affordable medicines,” said Rep. Carolyn Maloney, DN.Y., Chair of the House Oversight and Reform Committee. in the statement.
The FDA “remains committed to the integrity of our drug approval process, which includes making safe and effective new treatment options available to the millions of people living with Alzheimer’s disease in the United States,” the agency said in a statement to ABC News.
“The FDA’s decision to approve Aduhelm is based on our scientific evaluation of the data contained in the application, which are described in the approval materials. … the statement said, in particular:
The report also criticized Biogen for its “unreasonably high price for Aduhelm,” which it said was designed to “make history.” A year of Aduhelm treatment initially cost $56,000 for an average person, the report said.
“Biogen stands by the integrity of the actions we take,” the company said in a statement to ABC News. “As the congressional report states, the FDA’s review concluded that “There is no evidence that these pre-application interactions with the sponsor are anything but appropriate in this situation.”
“Alzheimer’s is a very complex disease and we have learned from the development and launch of Aduhelm. That process continues to inform our work as Biogen brings new innovative treatments to market,” the statement said.
Aduhelm was considered for FDA approval under the agency’s traditional pathway before the agency moved to consider the drug under its Accelerated Approval Program, the report said.
The change in course, which a congressional inquiry said was “dramatic,” came after the FDA found internally that Aduhelm lacked the “demonstrated clinical benefit necessary for traditional approval.” are the investigators of the House of Representatives.
Under the fast-track program, the FDA can approve certain drugs that meet both “unmet medical needs” and “treat serious conditions,” according to the agency’s website. Aduhelm is not the only drug approved through this pathway, which the FDA developed specifically to provide more options to patients with diseases for which there is virtually no cure. Accelerated approval is usually contingent upon larger, longer-term studies by the drug company.
During the months-long approval process, the FDA and Biogen worked closely together. FDA and Biogen officials met, spoke or otherwise had significant e-mail exchanges at least 115 times, according to a congressional investigation.
While it is typical for drug companies to work closely with the FDA in the approval process, such cooperation between Biogen and its federal regulators “in some ways exceeded the norm,” an internal FDA review said, according to the report.
The collaboration comes after an independent review of Aduhelm’s effects in people with Alzheimer’s disease questioned its ability to “effectively slow down cognitive and functional impairment” in patients.
And when the FDA initially approved the drug for all Alzheimer’s patients, a congressional investigation found that the move concerned some Biogen consultants who were wary of such broad approval. The advisers did not disclose such reservations to regulators, the report said.
The FDA later declined broad approval of the drug, eventually recommending the drug for use only in a narrower subset of early-stage patients that more closely mirrored the group included in Aduhelm’s clinical trials.
Following the FDA’s accelerated approval of Aduhelm, the Centers for Medicare & Medicaid Services stipulated that Medicare will pay for the drug only in limited circumstances when patients are part of approved studies.
That move severely limits Aduhelm’s availability and practical implications, essentially making it available only to people who can pay for the treatment out of pocket.
And as part of the accelerated approval provisions, Biogen has more than six years to complete the final trial, according to the report. Aduhelm remains available until then.
Thursday’s report comes as the FDA evaluates two additional Alzheimer’s drugs, with a green light from the agency possible in the coming months. Biogen helped develop one of those drugs.
“The FDA must take swift action to ensure that its processes for future reviews of Alzheimer’s treatments do not lead to the same doubts about the integrity of the FDA’s review,” the congressional report said.
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