By Deena Beasley and Julie Steenhuysen
Jun 11 (.) – By authorizing the first new Alzheimer’s drug in nearly 20 years, the US Food and Drug Administration (FDA) takes even greater risk in its strategy of bringing new therapies to market without resounding evidence about its effectiveness, regulatory and scientific experts said.
Biogen Inc’s drug Aduhelm was licensed based on evidence that it can reduce platelets in the brain, a possible factor in the development of Alzheimer’s, rather than strong evidence that it slows the progression of the fatal, degenerative disease.
The FDA has granted so-called “expedited approvals” in more than 250 instances since 1992, mostly for rare diseases or very small patient populations with no available treatments.
In these cases, the agency requires drug companies to conduct additional clinical trials to prove their therapy is effective, or they could experience a withdrawal of the marketing license.
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 panel members have resigned in protest since the FDA’s decision was announced Monday.
“This decision has shaken the foundations of the scientific process and methods,” said Dr. Jason Karlawish, co-director of the Penn Memory Center in Philadelphia. Karlawish ran one of Biogen’s drug testing centers.
He said the FDA made its decision “by decree” and had not asked its advisers to consider whether the drug’s ability to remove a type of plaque in the brain known as beta amyloid would improve patient outcomes.
“It’s a disturbing set of events, scientifically, clinically and politically,” Karlawish said.
Biogen has said that about 1.5 million Americans with early-stage Alzheimer’s will be eligible for the drug, with an average price of $ 56,000 per year, and the federal Medicare insurance program for the elderly will likely address most of the problems. costs.
FDA approval allows Biogen to sell its product for several years, with potential annual sales forecasts reaching between $ 10 billion and $ 50 billion, until the company completes the required follow-up study.
“No unmet need, however compelling, can replace the requirement for sufficient evidence,” said Johns Hopkins Center professor of public health Dr. Caleb Alexander, a member of the FDA advisory panel.
The number of Americans living with Alzheimer’s disease is expected to double to around 13 million by 2050, according to the Alzheimer’s Association.
Biogen’s head of research, Alfred Sandrock, said the FDA had painstakingly analyzed data from its clinical trials for two years before reaching a conclusion.
“I think they made the right decision on behalf of the American people,” Sandrock said in an interview.
THE FDA’S POSITION
The FDA has defended its decision by saying that Biogen presented clear evidence that Aduhelm, known chemically as aducanumab, cleared beta amyloid from the brains of people with Alzheimer’s. Amyloid has long been a target of experimental therapies for the disease. However, none of the above drugs have shown that amyloid reductions produce significant benefits for patients by slowing the decline in cognition or the ability to function. Dr. Peter Stein, director of the FDA’s Office of New Drugs, told . that Aduhelm showed the clearest correlation yet between a reduction in amyloid beta and a delay in cognitive decline among patients, suggesting that it is a good predictor of clinical benefit. FDA officials said other Alzheimer’s drug developers cannot assume their therapies will be considered under the same framework. Eli Lilly and Co and Roche Holding AG are among the manufacturers also working on drugs that target beta amyloids.
“We cannot say at this time whether this will be a pathway for other Alzheimer’s drugs or therapies for other neurodegenerative diseases,” Dr. Patrizia Cavazzoni, director of the FDA’s Center for Drug Evaluation and Research, told ..
Critics are less convinced that amyloid clearance is an adequate measure to predict clinical benefit.
“We need to rethink the use of these regulations as interpreted and used by the FDA,” Karlawish said.
Nearly half of all drugs that received expedited approval finally received full authorization, after a median of about three years, according to a report from the Institute for Clinical and Economic Review (ICER), a drug cost watchdog. . Despite Aduhelm’s high price, patient advocacy groups praised the FDA’s decision and said it would jumpstart research in the field, including the types of drug combinations that have improved the treatment of complex diseases like cancer and cancer. HIV
“This approval will generate additional investment by other companies in disease-modifying treatments and therapies,” activist group UsAgainstAlzheimer’s said in a statement.
(Reporting by Deena Beasley in Los Angeles and Julie Steenhuysen in Chiacgo. Edited in Spanish by Marion Giraldo)