Aduhelm: House probe says FDA approval process for Alzheimer’s drug was ‘riddled with irregularities’



CNN

A congressional investigation found the US Food and Drug Administration’s “atypical collaboration” to approve a high-priced Alzheimer’s drug was “riddled with irregularities.”

The report, released Thursday, was the result of an 18-month investigation by two House committees. He is very critical of Biogen, manufacturer of the drug Aduhelm.

The report says Biogen set an “unjustifiably high price” for Aduhelm to “make history” for the company and thought of the drug as an “unprecedented financial opportunity.” Biogen cost Aduhelm $56,000 a year, even though its actual effects on a broad patient population were unknown.

More than 6.5 million people in the United States are living with Alzheimer’s, and that number is expected to grow to 13.8 million by 2060, according to the Alzheimer’s Association. The disease is the sixth leading cause of death in the United States. There is no cure and effective treatments are extremely limited. Prior to Aduhelm’s approval in June 2021, the FDA had not approved a new therapy for the disease since 2003.

The investigation found that Biogen planned an aggressive marketing campaign to launch the drug, intending to spend more than $3.3 billion on sales and marketing between 2020 and 2024, more than two and a half times what it spent to develop Aduhelm.

Dementia, including Alzheimer’s, is one of the “costliest conditions for society,” according to the Alzheimer’s Association. In 2022 alone, Alzheimer’s and other dementias cost the U.S. $321 billion, including $206 billion in Medicaid and Medicare payments, according to the association.

The cost of Aduhelm to patients and to Medicare would be significant, the new report says. It was one of the key factors behind a big increase in Medicare premiums in 2022, according to the Centers for Medicare and Medicaid Services.

In anticipation of “pushback” from providers and payers, Biogen also cooked up a narrative to sell the drug’s value, according to the report.

The Committee on Oversight and Reform and the Committee on Energy and Commerce found that the collaboration between the FDA and Biogen in the drug approval process “went above the norm in some respects.”

Biogen had initially halted clinical trials of Aduhelm in March 2019 after an independent committee found it was unlikely to slow the cognitive and functional impairment – the decline in memory, language and judgment – that comes with Alzheimer’s. But in June 2019, the FDA and Biogen started a “task force” to see if the effort could be saved.

The investigation found that the FDA and Biogen engaged in at least 115 substantive meetings, calls and email discussions between July 2019 and July 2020, including 40 meetings to guide Aduhelm’s potential approval. There may have been even more meetings, but the committees say the FDA did not follow its own documentation protocol.

The agency then collaborated with Biogen to draft a paper used to inform an independent advisory committee that met in November 2020. Results from the trial were mixed, with only one showing a small benefit for to the patients

At that meeting, none of the committee members voted to say that the studies presented strong evidence that the drug was effective in treating Alzheimer’s.

The meeting was unusual, according to a former FDA adviser who had been on the committee for several years. Dr. Aaron Kesselheim told CNN in 2021 that the relationship between the FDA and the company was out of the ordinary.

“There was an odd dynamic compared to the other advisory committee meetings I’ve attended,” said the Harvard Medical School professor. “Usually there’s some distance between the FDA and the company, but in this case, the company and the FDA were completely in line with each other in support of the drug.”

When the FDA approved the drug, Kesselheim and two other members of the advisory committee resigned in protest. He later called it “probably the worst drug approval decision in recent American history.”

The FDA often follows the independent panel’s recommendations, but in this case, it reversed course and used its accelerated approval pathway, which sets a different standard of proof that a treatment could work.

Committee members said senior FDA management told them the change in how the drug was approved came after an April 2021 FDA expert panel meeting provided “feedback unfavorable” for the traditional approval process, according to the new report.

The FDA also approved the drug for “people with Alzheimer’s disease,” a much broader population than was studied in Biogen’s clinical trials.

Internal company documents said Biogen agreed to this broader indication “despite internal reservations about the lack of evidence of clinical benefit for patients in disease stages outside of clinical trials and an unknown safety profile.” says the report. Leaders expressed concern that the company could lose credibility and developed a communications strategy to deal with the “anticipated consequences,” the report said.

The committees recommended that the FDA document all of its meetings with drug sponsors, establish a protocol for briefing papers and advisory committees, and update its guidance on how Alzheimer’s drugs are developed and reviewed.

The committees also recommend that companies clearly communicate safety and efficacy concerns to the FDA and consider value assessments made by outside experts when setting drug prices.

“The American people trust the FDA to ensure the safety and effectiveness of the medicines they take. The number of patients and families affected by Alzheimer’s disease will continue to rise, and it is crucial that the FDA and drug companies “adhere to established procedures and conduct themselves with the transparency necessary to gain public trust,” the report says.

The FDA said in a statement that its “decision to approve Aduhelm was based on our scientific evaluation of the data contained in the application, which are described in the approval materials.”

The agency says it is reviewing the committees’ findings and recommendations and says its own review found the interactions with Biogen were appropriate.

“It’s the agency’s job to interact frequently with businesses to ensure we have the right information to inform our regulatory decision-making. We will continue to do so as it is in the best interest of patients. That said, the agency has already begun implementing changes consistent with the Committee’s recommendations.”

Biogen said in a statement Thursday that it has been working “cooperatively” with the investigation.

“Biogen has been committed to researching and developing treatments for Alzheimer’s disease for more than a decade. We have relentlessly focused on innovation to address this global health challenge and have adapted to both successes and setbacks “, he said. “Biogen stands by the integrity of the actions we have taken.”

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