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Biotechnology company Biogen and its regulator, the Food and Drug Administration, worked together, ignoring internal company concerns and circumventing the agency’s own written guidance, to allow Alzheimer’s treatment Aduhelm to receive accelerated approval and at a premium on the drug Market gets patients $56,000 a year, according to a scathing report released Thursday by two House committees.
The “unusual” collaboration, which Aduhelm revived three months after Biogen canceled clinical trials, unfolded through at least 115 meetings, phone calls and email exchanges between the company and the FDA over a year, the committee’s report said for oversight and reform. and energy and trade.
The combined effort culminated when agency staff helped Biogen author a document that informed the FDA’s Advisory Committee ahead of its November 6, 2020 meeting to discuss Aduhelm. Although the FDA often follows the recommendation of an advisory committee, this time it didn’t. After no member of the Advisory Committee recommended Aduhelm, the FDA changed course and allowed Biogen to move its drug to an accelerated approval process.
At the FDA’s suggestion, the drug was labeled for use by the country’s more than 6 million Alzheimer’s patients, even though it had only been tested on people with early-stage Alzheimer’s and mild symptoms, the report said.
“This is the worst decision the FDA has ever made,” said Sidney Wolfe, founder of the advocacy group Public Citizen’s Health Research Group. “It was an unprecedented alliance between the company and the FDA.”
“We have fully cooperated in the committees’ review and continue to review their findings and recommendations,” the FDA said in a statement in response to the report. “It is the agency’s job to interact frequently with companies to ensure we have adequate information to make our regulatory decisions. We will continue to do so as it is in the best interests of patients. However, the Agency has already started to implement changes in line with the Committee’s recommendations.”
The agency had previously conducted an internal investigation into its handling of Aduhelm and concluded more than a year ago that while the collaboration “exceeded the norm in some respects”, there was “no evidence” that the deals between the company and the regulator were “other than” reasonable.”
The internal report said the decision to “proactively” work with Biogen “is consistent with FDA guidance,” given both the “large unmet medical need” for the treatment of Alzheimer’s and the view of an FDA official, that one of the Aduhelm studies “hit a home run” in terms of safety and effectiveness.
The report by the two House Committees also found fault with the company, saying Biogen knows the initial price of $56,000 per year — which was lowered to $28,000 in January 2022 — would place a heavy burden on patients. But the Cambridge, Massachusetts-based company estimated the treatment could bring in up to $18 billion a year for Biogen, and in a slide presentation to its board of directors, happily said, “Our goal is to make history” and “too.” establish [the drug] as one of the best pharmaceutical launches of all time.”
In fact, Aduhelm turned out to be a financial dud, raking in $3 million in revenue for the full year of 2021.
In a statement in response to the report, Biogen said it has worked with the committees and “stands by the integrity of the actions we are taking.” Biogen’s statement also cited the FDA’s internal investigation, which concluded that there was no evidence of impropriety in dealings between the agency and the company.
Biogen stuck to the initial price of $56,000 per year, despite forecasts that the drug could cost Medicare as much as $12 billion in a single year. Other Alzheimer’s treatments sell for a lot less. A year’s supply of Aricept costs less than $8,000; Exelon, a drug in the same family, costs about $8,800 for a year’s supply; and Namenda costs less than $3,000 a year.
The report includes recommendations the FDA should follow to “restore the confidence of the American public” and actions Biogen and other drug companies should take to “meet their responsibilities to patients and families.” Because the recommendations leave it up to the FDA and the company to change their policies, it’s unclear if they’ll actually prevent future episodes like this one.
The committees recommended that the FDA document all communications with drug sponsors, establish a system for working with companies to produce the reports used to inform their own advisory committees, and formal guidelines for the development and review of new Alzheimer’s disease update medication.
Drug sponsors, the committees recommended, should raise any concerns about the safety and efficacy of treatments openly and clearly with the FDA, and should consider the opinions of outside experts when setting prices for new drugs.
Aduhelm, a lab-made protein that’s given directly into a patient’s vein, is said to work by reducing a sticky substance in the brain called amyloid beta, which clumps between neurons and disrupts their function. Some scientists have theorized that accumulation of amyloid beta in the brain causes Alzheimer’s disease.
In September 2015, Biogen began enrolling patients in two Phase 3 clinical trials, testing the safety and efficacy of a drug and comparing it to standard care. Three and a half years later, in March 2019, the company announced it was terminating both studies after receiving an independent report that concluded the treatment reduced the memory loss, confusion and other symptoms associated with Alzheimer’s disease brain damage caused would probably not slow down.
But the drug’s death was short-lived.
The report shows that two months after the trials ended, representatives from Biogen and the FDA met at a neurology conference in Philadelphia and discussed the results of the trials. The FDA official suggested that the agency and the company call a special meeting to discuss the data from the studies.
FDA documents reviewed in the new report show that Biogen has entered into informal discussions with the agency to determine whether the data from the ongoing trials indicate a benefit for patients. A meeting between the FDA and Biogen in mid-June 2019 resulted in both agreeing to form a joint “working group.”
The collaboration would result in the FDA and Biogen moving forward with the drug, though employees at both the agency and the company have expressed reservations about some of the decisions made.
For example, the FDA granted accelerated approval for Aduhelm despite failing to garner the support of a single member of its own Advisory Committee and without bringing the idea up for discussion from an internal or external panel.
In addition, the FDA’s approval violated its own guidelines for early treatment of Alzheimer’s disease, which stated that “there is not enough reliable evidence” that a drug’s effect on amyloid beta alone would be sufficient to kill the patient help. Scientists have expressed conflicting opinions as to whether amyloid beta is a cause of Alzheimer’s or simply a consequence of the disease.
The report also noted that a team of Biogen employees studied the financial impact that the initial price of Aduhelm would have on patients and concluded that the country’s “over 65 population will face challenges.” [their] Ability to pay.” The team estimates that two-thirds of Medicare patients at risk of developing Alzheimer’s would have to pay some of the cost themselves, although more than half earn less than $50,000 a year and more than a third have assets of less than $5,000.
Although the report noted that the company “appeared to have developed financial assistance programs for eligible patients,” investigators wrote that “these programs would leave significant gaps in coverage.”
Despite the expected hardship the price would impose on patients, Biogen expected to spend “between $500 million and $600 million expanding its sales force” to commercialize the drug, the report said.
Five months after the drug’s launch, the Centers for Medicare and Medicaid Services said monthly Medicare Part B premiums will increase 14.5 percent in 2022, half of that in anticipation of higher costs from the new Alzheimer’s treatment.
According to the report, the percentage increase resulted in a $21.60 increase in monthly premiums for Medicare Part B beneficiaries, “reportedly the largest dollar increase in the history of the program.”
For its part, Biogen introduced a broad label that was Aduhelm for “people with Alzheimer’s disease,” despite staff reservations about the lack of evidence of clinical benefit for patients in more advanced stages of the disease than those involved in clinical management were attempts. Some within the company even expressed concern that moving forward with the labeling plan “could damage the credibility of the company,” according to the report.