Does the new Alzheimer’s drug work? Answers may be missing in the 2030 target


This image provided by Biogen on Monday, June 7, 2021 shows a bottle and packaging for the drug Aduhelm. Pharmacist Biogen has until 2030 to complete a study that confirms whether the new drug really slows down the disease that destroys the brain. This is in line with the terms of the Food and Drug Administration’s conditional approval of the drug, a decision that patients have praised as overdue and condemned by the agency’s own external experts. Credit: Biogen via AP, File

When a controversial Alzheimer’s drug got U.S. approval, surprise at the decision quickly turned into a shock as to how long it can take to see if it really works, nine years.

Pharmacist Biogen has until 2030 to complete a study to confirm if it is new Aduhelm really slows down the disease that destroys the brain. This is under the terms of conditional approval from the Food and Drug Administration, a decision that patients have praised as overdue and condemned by the agency’s own external experts.

But the two camps agree: 2030 is too long to wait for answers about the $ 56,000 a year drug.

“We believe nine years is unacceptable and our expectation is that it will happen in a much shorter period of time,” said Maria Carrillo of the Alzheimer’s Association, a advocacy group that pushed for approval, but now wants it to the FDA sets a faster deadline.

Other experts warn that the 2030 timeline could slip if patients refuse to enroll in a new study of a drug that is already available. And the focus on Aduhelm, the first new drug against Alzheimer’s in 18 years, could turn volunteers away from the trial of other promising treatments.

“If someone can go to their doctor and get the FDA-approved drug, why would they participate in a trial where they run the risk of receiving a placebo?” said Donna Wilcock, an Alzheimer’s researcher at the University of Kentucky.

To establish the safety and efficacy of a new drug, the researchers compared the results in people receiving treatment with a similar group of people who did not. Overall, this means that half of the volunteers are randomly assigned to receive fake treatment instead of the actual one.

Biogen has already conducted two such important studies on its drug, which requires monthly IVs. The studies took about four years to run and followed the participants for about 1 year and a half. Both stopped early when it appeared the drug was not working and the results were so wrapped up in flaws and inconsistencies that the FDA considered them too weak to support approval on the basis of slowing the disease.

Instead, the agency took a different approach and gave conditional approval to the drug based on a promising sign: its success in getting rid of an accumulation of adhesive plaque on the brain that is he believes it will play a role in Alzheimer’s disease.

Under its so-called accelerated approval program, the FDA requires Biogen to conduct a new study that will definitely answer whether the effect of Aduhelm on plaque really slows the mental decline of patients. Other anti-Alzheimer’s drugs on the market only temporarily relieve symptoms.

The FDA has not detailed how the 2030 target was produced or why such a long-term deadline was granted for a drug that could be administered to millions of patients in the coming years, adding billions to the drug bill. health care in the country.

“Alzheimer’s trials take a long time to complete,” the FDA said in a statement in response to questions about the study. The agency added that it might be possible to answer key questions about Aduhelm before the study is completed and that Biogen is expected to present results “as soon as possible.”

But critics of the agency point out that nine years is between them longer follow-up periods the agency grants drug manufacturers. Drugs approved in similar circumstances are usually six years old. And, in any case, these studies tend to run late and do not end soon. If follow-up studies do not have positive results, the FDA may withdraw approval, although it rarely does.

“Just saying nine years doesn’t mean the tests will be available in nine years,” said Joshua Wallach, a medical researcher at the Yale School of Public Health. “There’s all this back-and-forth discussion that can happen with the FDA that can delay its completion.”

Biogen is not scheduled to submit its initial proposal for the study to the FDA until October. The Massachusetts-based company said in a statement that major trials on Alzheimer’s usually take six to seven years and that FDA-required studies may take even longer.

“We are working urgently and establishing resources and plans,” to complete the trial ahead of schedule, the company said.

Meanwhile, Alzheimer’s specialists, such as Dr. Samuel Gandy, are seeing patients in other drug studies ask themselves to abandon them and get Aduhelm.

“Everyone has said,‘ You know, I can’t stand the idea of ​​being on a placebo, ’” said Gandy, who has listened to more than 20 families interested in drugs at New York’s Mount Sinai Hospital.

After explaining the unknown benefits and possible side effects of the drug, including brain swelling and bleeding, several decided against it. But other patients remain interested.

Post-approval studies have become one increasingly common FDA requirement since the 1990s, as regulators have accelerated their reviews of drugs for HIV, cancer and other deadly diseases. But the agency’s mixed record of monitoring these requirements and penalizing companies that do not meet them has been analyzed. government i academic studies.

The case of a widely debated drug for muscular dystrophy illustrates how the system can go wrong.

In 2016, the FDA approved Sarepta Therapeutics ’first unique drug based on preliminary results that could help treat degenerative disease by increasing a muscle protein.

As with Aduhelm, external FDA advisers opposed the approval, saying there was little evidence that the drug actually improved the patient’s health or quality of life. But the FDA granted approval on the condition that Sarepta complete a confirmatory study in May 2021.

The trial, however, is still underway after “multiple challenges in overall planning and implementation,” according to the FDA website. The new target date is 2026, a decade after the drug was allowed on the market.

A Sarepta spokeswoman said the company spent years negotiating the details of the study with the FDA, which required testing a higher dose.

Meanwhile, Sarepta has obtained approval for two other dystrophic drugs based on similar results that also require follow-up trials, which the company says are already on track.

“The FDA took a risk with Sarepta and I think it’s burning them now,” said Dr. Joseph Ross of Yale.

Ross and his colleagues have at least proved it a quarter of follow-up results are never published, leaving questions for doctors and patients.

The results of Biogen’s two Aduhelm studies have not yet appeared in a medical journal. The company says it “works diligently to publish our data.”

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