WASHINGTON — When a controversial Alzheimer’s drug won US approval, surprise at the decision quickly turned to shocking how long it might take to find out — nine years.
Drugmaker Biogen has a study to complete by 2030 to confirm whether its new drug EduHelm actually slows the brain-destroying disease. This is under the terms of the Food and Drug Administration. conditional acceptance l of the drug, a decision that has been praised and condemned by patients as an overdose The agency’s own outside experts.
But both camps agree: 2030 is too long to wait for an answer on the $56,000-a-year drug.
“We think nine years is unacceptable and our expectation is that it will happen in a very short time frame,” said Maria Carrillo of the Alzheimer’s Association, an advocacy group that was pushing for approval but now wants the FDA to act quickly. Set a time limit.
Other experts have warned that the 2030 deadline could be pushed back if patients shy away from enrolling in a new study for an already available drug. And the focus on EduHelm — the first new Alzheimer’s drug in 18 years — could spare volunteers from testing other promising treatments.
“If someone can go to their doctor and get an FDA-approved drug, why would they go to a trial where they are at risk of getting a placebo?” Donna Wilcock, an Alzheimer’s researcher at the University of Kentucky, said.
To establish the safety and effectiveness of a new drug, researchers compare the results of those who do not with the same group of people who receive the treatment. This usually means that half the volunteers are randomly assigned to receive a fake treatment instead of the real thing.
Biogen has already conducted two large studies of its drug that require monthly IVs. The study took about four years to run, and participants were followed for about 1 1/2 years. Both were stopped early when it seemed the drug was not working, and the results were so inundated with flaws and inconsistencies that the FDA deemed them too weak to support approval on the grounds of slowing the disease.
Instead, the agency took another approach and gave the drug conditional approval based on one promising indication: its success in getting rid of a build-up of sticky plaque in the brain that is thought to play a role in Alzheimer’s disease.
Under its so-called accelerated approval program, the FDA is requiring Biogen to conduct a new study that is definitively answering whether EduHelm’s effect on plaque actually slows mental decline in patients. Other Alzheimer’s drugs available in the market only temporarily reduce symptoms.
The FDA did not elaborate on how the 2030 target came about, or why it gave such a distant deadline for the drug to be given to millions of patients in the coming years. add billions to the nation’s health care bill.
“The Alzheimer’s trials take time to complete,” the FDA said in a statement in response to questions about the study. The agency said it may be possible to answer important questions about EduHelm before the completion of the study and that Biogen expects to submit the results “as soon as possible.”
But critics of the agency point out that nine years is one of longest follow-up period The agency gives grants to drug manufacturers. Medicines approved under similar conditions usually get six years. And, if anything, those studies tend to lag behind time, not end quickly. If follow-up studies do not have positive results, the FDA may withdraw approval, although it rarely does.
“Just because it says nine years doesn’t mean the evidence will be available in nine years,” said Joshua Wallach, a medical researcher at the Yale School of Public Health. “It’s all back-and-forth discussions that may be going on with the FDA that may delay completion.”
Biogen is not scheduled to submit its initial proposal for study to the FDA until October. Large Alzheimer’s trials often take six or seven years, and FDA-mandated studies may take even longer, the Massachusetts-based company said in a statement.
“We are working with urgency and implementing resources and plans,” to complete the trial ahead of schedule, the company said.
Meanwhile, Alzheimer’s experts like Dr. Samuel Gandy are looking at other drug studies asking patients to opt out so they can receive EduHelm.
“They’ve all said, ‘You know, I can’t stand the thought of being on a placebo,'” said Gandy, who heard from more than 20 families interested in the drug at Mount Sinai Hospital in New York.
When he explained the drug’s unknown benefits and potential side effects—including brain swelling and bleeding—many decided against it. But other patients remain interested.
post-approval studies have become a evenly edged The FDA’s requirement since the 1990s, as regulators accelerate their reviews of drugs for HIV, cancer and other deadly diseases. But the agency has a mixed record of tracking those requirements and penalizing companies that don’t meet them. Government and academic study.
The case for a widely debated drug for muscular dystrophy shows how the system can go awry.
In 2016, the FDA approved first drug of its kind Based on preliminary results from Sarepta Therapeutics that it may help treat degenerative disease by increasing muscle-building proteins.
Like EduHelm, the approval was opposed by outside FDA advisors, who said there is little evidence that the drug actually improved a patient’s health or quality of life. But the FDA granted approval on the condition that Sarepta completes a confirmatory study by May 2021.
However, the trial is still underway, according to the FDA’s website, despite “multiple challenges in overall planning and startup.” The new target date is 2026, a decade after the drug was allowed on the market.
A spokesperson for Sarepta said the company spent years negotiating study details with the FDA, which required higher-dose trials.
Meanwhile, Sarepta has obtained approval for two other dystrophies Follow-up trials are also needed for drugs based on similar results, which the company says are already well underway.
“The FDA took a risk with Sarepta and I think they’re going to be burned by it now,” said Yale’s Dr. Joseph Ross.
Ross and his colleagues have shown that at least quarter Follow-up results are never published, leaving questions for clinicians and patients.
The results of Biogen’s two EduHelm studies have yet to appear in a medical journal. The company says it is “working diligently to publish our data.”
Follow Matthew Perron on Twitter: @ ap – fdawriter
The Associated Press Department of Health and Science receives support from the Howard Hughes Medical Institute’s Department of Science Education. AP is solely responsible for all content.