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House investigation says FDA approval process of Alzheimer's drug was 'rife with irregularities'

(CNN) A congressional investigation found that the US Food and Drug Administration's "atypical collaboration" to approve a high-priced Alzheimer's drug was "rife with irregularities."

The report, released Thursday, was the result of an 18-month investigation by two House committees. It is sharply critical of Biogen, maker of the medication 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 priced Aduhelm at $56,000 per year, even though its actual effects on a broad patient population were unknown.

More than 6.5 million people in the US live 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. Before Aduhelm's approval in June 2021, the FDA had not approved a novel therapy for the condition since 2003.

Expecting pushback

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 2½ times what it spent to develop Aduhelm.

Dementia, including Alzheimer's, is one of the "costliest conditions to society," according to the Alzheimer's Association. In 2022 alone, Alzheimer's and other dementias cost the US $321 billion, including $206 billion in Medicaid and Medicare payments, the association says.

Aduhelm's cost 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, the report says, Biogen also prepared a narrative to sell the value of the drug.

An unusual collaboration

The Committee on Oversight and Reform and the Committee on Energy and Commerce found that the collaboration between the FDA and Biogen in the approval process of the drug "exceeded the norm in some respects."

Biogen had initially discontinued Aduhelm's clinical trials in March 2019 after an independent committee found that it probably would not 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 "working group" to see whether the effort could be saved.

The investigation found that the FDA and Biogen engaged in at least 115 meetings, calls and substantive email discussions from July 2019 to July 2020, including 40 meetings to guide Aduhelm's potential approval. There may have been even more meetings, but the committees say the FDA failed to follow its own documentation protocol.

The agency then collaborated with Biogen to draft a document used to brief an independent advisory committee that met in November 2020. The trial results were mixed, with only one showing a small benefit to patients.

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

The meeting was unusual, according to one former FDA adviser who had sat 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 a strange dynamic compared to the other advisory committee meetings I've attended," the professor at Harvard Medical School said. "Usually, there's some distance between the FDA and the company, but on this one, the company and the FDA were fully 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 labeled it "probably the worst drug approval decision in recent US history."

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

The committee members said senior FDA leadership told them that the shift in how the drug would be approved came after an FDA expert council meeting in April 2021 provided "unfavorable feedback" for the traditional approval process, according to the new report.

Approved for a broader population

The FDA also approved the drug for "people with Alzheimer's disease," a far broader population than was studied in Biogen's clinical trials.

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

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

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

"The American people rely on FDA for assurance on the safety and efficacy of the medications they take. The number of patients and families impacted by Alzheimer's disease will continue to increase, and it is crucial that FDA and drug companies adhere to established procedures and conduct themselves with the transparency necessary to earn public trust," the report says.

FDA, Biogen respond

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 is described in the approval materials."

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

"It is the agency's job to frequently interact with companies in order to ensure that we have adequate 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 started 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 been focused relentlessly on innovation to address this global health challenge, and have adapted to both successes and setbacks," it said. "Biogen stands by the integrity of the actions we have taken."

CNN's Jacqueline Howard, Brenda Goodman and Tami Luhby and Jeffrey Toobin contributed to this report.