What does the FDA approval of a new drug for Alzheimer’s disease mean?

In a highly controversial decision, the The FDA gave an expedited approval to the first drug that could slow the progression of Alzheimer’s disease. But the approval does not say that the FDA believes the drug will actually treat the disease. Rather, the FDA carefully states that the drug, under the chemical name aducanumab and the trade name Aduhelm, reduces a substance in the brain that is associated with Alzheimer’s disease.

In effect, the FDA appears to have approved the drug, not because it is likely to treat Alzheimer’s successfully, but because it may lead to more research on other drugs that might. This justification will benefit Biogen.

And it could pave the way for major breakthroughs in the future, but its benefits for people with dementia today are likely to be limited.

Despite its questionable immediate value and Biogen’s plan to charge $ 56,000 a year for what it says are necessary infusions, desperate families are likely to sue for the drug. Not only could it put enormous pressure on doctors and family budgets, it could also put greater pressure on Medicare finances.

Unusual approval

The road to drug approval was extremely unusual. Biogen conducted two clinical trials. One tentatively showed a 22 percent reduction in cognitive decline, which would have been an extraordinarily positive result. But the other did not find any benefit at all. After the second result, Biogen dropped the investigation. It then reopened its application for FDA approval, saying that giving high doses of the drug to people with disease at a very early stage could be beneficial.

An independent The FDA study panel strongly recommended that the agency not approve tthe drug, pending further investigation. In an unusual step, the FDA rejected that recommendation and approved the drug. However, it required Biogen to conduct a third clinical trial to determine its effectiveness. It will likely take years before the test is completed. It did not limit approval to early stage patients.

The FDA did not say that Aduhelm can successfully treat Alzheimer’s. Rather, it concluded that the drug could have a positive effect “on a surrogate endpoint that is reasonably likely to predict clinical benefit for patients.” In other words, the FDA believes the drug shows promise in its ability to reduce the substance that could be the cause of Alzheimer’s.

Johns Hopkins epidemiologist G. Caleb Alexander, a member of the FDA advisory committee who rejected the drug, He said The Washington Post, “I am surprised and disappointed. I think this product was driven by the magnitude of the unmet need, which is huge. But unmet needs cannot exceed regulatory standards, and my concern is that in this case they may have. “

Intense lobbying

The FDA approval came after intense pressure from Biogen, the drug’s developer, other manufacturers working on similar drugs, and organizations such as the Alzheimer’s Association, which has close ties to the pharmaceutical industry.

But many dementia experts are highly critical of both the FDA approval process and the decision itself. They say it will do little more than give false hope to millions of people with the disease and their families.

Not only do they question the value of Aduhelm, they argue that the entire field of research that produced him is focused on the wrong cause of Alzheimer’s disease. For decades, a group of scientists has insisted that Alzheimer’s could be treated by breaking down substances called beta amyloid plaque. Still decades of drug trials based on this theory have failed. Now, there is some evidence that Aduhelm can reduce those plates. But will it successfully treat Alzheimer’s disease?

High cost, limited value

Another group of researchers believe that Alzheimer’s disease may be the result of an entirely different cause, called tau tangles, or possibly some combination of substances that interfere with brain function. If they are right, Aduhelm and similar drugs would be of limited benefit to people living with Alzheimer’s, even if they break down beta amyloids.

It is also important to know that there is no evidence that this drug can successfully treat any of the many other forms of dementia. While Alzheimer’s is the most common, there are dozens of others. And some researchers believe that even those diseases that are grouped together as Alzheimer’s may in fact be many different conditions, much like cancers.

Aduhelm will be extraordinarily expensive. Biogen says it will charge patients $ 56,000 a year for the regular infusions needed. Wall Street Journal reports (paywall) that an independent research group estimated that the drug could be profitable between $ 2,500 and $ 8,300 a year. However, based on the FDA’s rationale for approving the drug, Biogen is likely to face competition from other companies with similar products. So you probably want to get performance as fast as possible.

For patients, the cost of the drug itself will be only part of the expense. They will require expensive positron emission tomography (PET) scans to determine if there is any chance that the drug can help them. And they will require continuous imaging to measure whether the drug is successfully reducing amyloid beta plaques.

While some insurances may pay for some drug costs, they are unlikely to pay for necessary tests and scans.

A cure, or at least a treatment to slow the progression of Alzheimer’s, would be an extraordinary step in improving the quality of life for millions of older adults. And after decades of failed research, it’s easy to understand why the FDA wants to push the development of new drugs that could help. But it would be a tragedy if people living with dementia and their families came out of the news today believing that a cure is about to hit the market.

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