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FDA considers approving Alzheimer's drug without finished trials nor proof it works

The projected price of the drug, if approved, is about $50,000 a year, Dr. Michael Carome said.

Published: May 22, 2021 9:04pm

Updated: May 23, 2021 11:02am

There has yet to be a cure for Alzheimer's disease, which afflicts about six million people in the U.S., but the FDA is considering approving a partially studied drug that aims to serve that purpose. Sinclair's Sunday TV Program, "Full Measure with Sharyl Attkisson," investigated the story. 

"The only drugs approved to treat Alzheimer's tackle symptoms and don't change the progressive, fatal course of the disease," reported Attkisson. 

A new drug called aducanumab, which would be administered as a shot every four weeks, would be the first to treat the root cause of Alzheimer's. However, some analysts say that the data shows aducanumab doesn't work. 

"The concern is that first of all, that the data isn't there to show that the drug works ... the agency was on the verge of favoring approval of this drug when the evidence simply did not support that conclusion," said Dr. Michael Carome, who heads up the health research group at the watchdog Public Citizen. 

Two years ago, the maker of aducanumab, Biogen, conducted "two large clinical studies on thousands of people," Attkisson reported. 

"But in March of 2019," Carome said, "the company announced that they were stopping both of these trials because an analysis partway through this study, it's called an interim analysis, showed that it would be 'futile' to continue, meaning that if they continued the trials to their planned completion, the expected result was going to be the drugs don't work. And so let's not continue it because it would be futile to do so."

However, nine months later, Biogen said it re-examined the data with the FDA and determined that aducanumab works, so they continued with the approval process. 

When asked if it was common for a drug company to proceed with the approval process with findings from incomplete studies, Carome said, "It's uncommon, and it's certainly uncommon for that type of evidence to be the basis for an eventual FDA approval."

In November 2020, the FDA had an advisers' meeting to consider approving aducanumab. 

Samantha Budd Haeberlein, of Biogen, said in the meeting, "Given the totality of the evidence, we can conclude that the benefit risk profile for aducanumab is favorable and potentially prolongs patients' independence by several months, even a few years, as demonstrated in our long term study. This matters for patients, their loved ones and society."

What was unusual about the meeting was that "Instead of the FDA providing a separate, independent analysis like it usually does, it partnered with Biogen to present a single joint briefing document," Attkisson reported. 

At the meeting, Dr. Billy Dunn, FDA Director of Office of Neuroscience, said, "When considered on its own, Study 302 would appear to be a home run, but we had to sort out the impact of the futility declaration."

Carome remarked: "And that biased review really was so striking that it led to very harsh criticism by the committee members of the way the FDA staff had handled their review of the application from Biogen."

He continued: "They criticized the FDA for its review, for its working collaboratively with Biogen to write these analyses. And the outcome of the analyses, they said, was one-sided, that you were cherry-picking data, that you used an approach that was statistically and scientifically inappropriate."

Dr. Scott Emerson, an FDA Adviser, said, "I was very, very, very disturbed by some of the analyses that were considered … This analysis seems to be subject to the Texas sharpshooter fallacy, a name for the joke of someone first firing a shotgun at a barn and then painting a target around the bullet holes."

The result of the meeting "was nearly unanimous. At least 10 and perhaps with one abstention said, 'there is not sufficient evidence here to justify approval of this drug and what the FDA needs to do is require another large, randomized placebo-controlled trial before allowing this drug to be considered for approval,'" Carome said. 

Regarding the delicate balance between wanting the FDA to approve drugs quickly but also being too friendly with drug companies, Carome commented, "Certainly Congress has passed laws that have encouraged and promoted collaboration between agencies like the FDA and the industries they regulate. So that's certainly part of the problem. Congress has also passed laws that have weakened the standards that FDA uses for approving drugs and medical devices."

"Carome's group has sent a series of letters asking for investigations into what it calls the 'unprecedented close collaboration' between the FDA and Biogen that 'dangerously compromised the independence and objectivity of senior staff and clinical reviewers,'" Attkisson reported. 

The FDA responded, insisting that its "interactions with sponsors [drug companies] are critically important to drug development… [and] essential to set clear goals and expectations…the absence of these interactions would dramatically delay the availability of effective drugs for patients who need them."

Carome explained the price of aducanumab. "They projected that the annual price that they would charge for the drug if it was approved would be about $50,000 per year. And so at 10 million patients, $50,000 a year, that would be about a half trillion dollars a year. And that could lead to extraordinary economic impacts on our healthcare system, bankrupt the Medicare program, bankrupt patients in terms of the copays that they would still need to pay. And if the drug doesn't work, at a great cost, lead to a lot of false hope for millions of patients and their families."

During the controversy, the FDA's final decision on aducanumab was put off in March, but is now expected in June.

Biogen declined Attkisson's interview requests. 

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