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(Reuters) – By approving the first new drug for Alzheimer’s disease in nearly 20 years, the U.S. Food and Drug Administration is taking its biggest risk yet with a strategy that allows new therapies without strong evidence of their effectiveness, according to regulatory and scientific experts. .
FILE PHOTO: Signage is seen outside the Food and Drug Administration (FDA) headquarters in White Oak, Maryland, US August 29, 2020. REUTERS / Andrew Kelly / File Photo
Biogen Inc’s drug Aduhelm was cleared based on evidence that it can reduce brain plaques, a likely contributor to Alzheimer’s disease, rather than evidence that it slows the progression of the deadly disease that makes you lose your mind.
The FDA has granted so-called accelerated approval in more than 250 cases since 1992, mostly for rare diseases or small patient populations that lacked effective treatment. In these cases, the agency requires the drugmakers to conduct additional clinical trials to prove that their treatment works, or risk withdrawing from the market.
Aduhelm, however, is in a different league in terms of the number of potential patients and the cost to the healthcare system.
Additionally, the FDA approval ignored the recommendation of its external advisers, who said Biogen did not provide sufficient evidence of clinical benefit. Three of the advisory committee members have resigned in protest since the FDA’s decision was announced on Monday.
The move shook the foundations of the scientific process and methods, said Dr Jason Karlawich, co-director of the Penn Memory Center in Philadelphia. Karlawich ran one of the Biogen drug testing sites.
He said the FDA made its decision by executive order and failed to ask its advisers to consider whether the ability of the drugs to remove a type of brain plaque known as beta-amyloid would improve patient outcomes. .
It’s a disturbing set of events, scientifically, clinically, politically, Karlawich said.
Biogen said about 1.5 million Americans with early-stage Alzheimer’s disease would be eligible for the drug, at an average price of $ 56,000 per year, under the federal Medicare insurance program for people. older people probably bear the brunt of the cost.
FDA approval allows Biogen to sell its product over several years – with potential annual sales forecast reaching $ 10 billion to $ 50 billion – until the company completes a required follow-up study.
No unmet need can replace sufficient evidence, said Johns Hopkins Professor of Public Health Dr. Caleb Alexander, a member of the FDA Advisory Group.
The number of Americans living with Alzheimer’s disease is expected to more than double to approximately 13 million by 2050, according to the Alzheimers Association.
Biogen chief researcher Alfred Sandrock said the FDA thoroughly analyzed data from its two-year clinical trials before reaching a conclusion.
I think they made the right decision on behalf of the American public, Sandrock said in an interview.
AN FDA POINT OF VIEW
The FDA defended its decision saying Biogen presented clear evidence that Aduhelm, known chemically as aducanumab, cleared beta-amyloid from the brains of people with Alzheimer’s disease.
Amyloid has long been a target of experimental therapies for the disease. However, none of the previous drugs have shown reductions in amyloid to lead to significant benefits for patients by slowing the deterioration in cognition or the ability to function.
Dr Peter Stein, director of the FDA’s Office of New Drugs, told Reuters that Aduhelm has so far shown the clearest correlation between reduced beta-amyloid and delayed cognitive decline in patients, suggesting that this was a good predictor of clinical benefit.
FDA officials have said that other Alzheimer’s drug developers cannot assume their therapies will be reviewed under the same framework. Eli Lilly and Co and Roche Holding AG are among the drug makers who are also working on drugs targeting amyloid.
We cannot say at this time whether this will be a route for other drugs for Alzheimer’s disease, or drugs for other neurodegenerative diseases, Dr Patrizia Cavazzoni, director of the Assessment Center, told Reuters. and FDA drug research.
Critics are less convinced that elimination of amyloid is an adequate indicator to predict benefit.
We need to rethink the use of these regulations as interpreted and used by the FDA, Karlawich said.
Almost half of all drugs with fast track approval eventually achieved full approval, after a median of about three years, according to a report by the Institute for Clinical and Economic Review (ICER), a watchdog. drug costs.
The exceptions include Lillys Lartruvo for sarcoma, which has been withdrawn, while the FDA has revoked its authorization for Avastin Rocks to treat breast cancer, although it is still used for other cancers.
More than 100 drugs with fast-track approvals have been on the market for just under two years, according to ICER. The report noted that many confirmatory trials are slow to complete and the results are often ambiguous.
The agency has become familiar with the fast-track approval path, said attorney Jim Shehan, head of FDA regulatory practice at law firm Lowenstein Sandler.
Biogen said a confirmatory trial for Aduhelm could take nine years. The FDA called the estimate conservative and said it would support efforts to complete this trial as soon as possible.
Despite Aduhelm’s high price tag, patient advocates hailed the FDA’s move, saying it would revive research in the area, including the types of drug combinations that have improved treatment for complex diseases like cancer. and HIV.
The approval will spark additional investment by other companies in disease-modifying treatments and therapies, advocacy group UsAgainstAlzheimers said in a statement.
Reporting by Deena Beasley in Los Angeles and Julie Steenhuysen in Chiacgo; Editing by Michele Gershberg and Bill Berkrot
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