The day – We may never know if the $ 56K a year Alzheimer’s drug works


The Food and Drug Administration’s approval in June of a drug believed to slow the progression of Alzheimer’s disease was widely celebrated, but it also raised alarm bells. The scientific community was concerned about the drug’s mixed results in studies. The FDA’s own expert advisory group was almost unanimous in opposing its approval. And the $ 56,000 annual price of the infusion drug, Aduhelm, has been criticized for potentially increasing Medicare and Medicaid costs by tens of billions of dollars.

But lost in this discussion is the underlying problem with the use of the FDA’s “fast track” to approve drugs for conditions like Alzheimer’s disease, a slow degenerative disease. Although patients are starting to take it, if the past is any guide, the world may have to wait many years to find out if Aduhelm is working – and maybe they will never know for sure.

The fast-track approval process, which began in 1992, is a consequence of the HIV / AIDS crisis. The process was designed to approve for the temporary sale of drugs that studies had shown may be promising but that had not yet reached the agency’s gold standard of “safe and effective”, under conditions. situations where the drug offered a potential benefit and there was no other option.

Unfortunately, the process has too often been reduced to a commercial end revolving around the agency.

The FDA has explained its controversial decision to give the green light to the latest product from pharmaceutical company Biogen: Families are desperate and there is no other treatment for Alzheimer’s disease. Importantly, when drugs receive this kind of fast-track approval, manufacturers are required to perform further controlled studies “to verify the clinical benefit of the drug.” If these studies fail to “verify clinical benefit, the FDA may” – may – withdraw them.

These subsequent studies often take years to complete, if they are completed at all. That’s because of the FDA’s notoriously lax monitoring and because drugmakers tend to drag their feet. When the drug is in use and the benefits are good, why would a manufacturer want to find out that a lucrative blockbuster is failing?

Historically, to date, most of the new drugs that have received accelerated approval treat severe malignancies.

Follow-up studies are much easier to do when the disease is cancer, and not a neurodegenerative disease like Alzheimer’s disease. In cancer, “no benefit” means tumor progression and death. Mental decline in Alzheimer’s disease often takes years and is much more difficult to measure. So years, or even decades, later, Aduhelm’s studies may not provide a clear answer, even if Biogen does manage to enroll a significant number of patients in follow-up trials.

Now that Aduhelm is on the market, enrolling in the required follow-up trials will likely be difficult, if not impossible. If your loved one suffers from Alzheimer’s disease, with its continued decline in mental function, you would want drug treatment to start now. How likely would you be to sign up and risk being placed in a placebo group?

The FDA gave Biogen nine years for follow-up studies, but acknowledged the timeline was “conservative.”

Even when the required additional studies are done, the FDA has historically been slow to respond to disappointing results. In a 2015 study of 36 anti-cancer drugs approved by the FDA, only five ultimately showed evidence of life extension. But making this decision took more than four years, and during that time the drugs were sold, at a handsome profit, to treat countless patients. Few drugs are removed.

In April, the FDA said it would be a better policeman for cancer drugs that hit the market with fast-track approval. But time, like delays, means money for drug makers.

A consultant who had worked with pharmaceutical companies on marketing drug treatments for hemophilia told me that the industry has called this serious bleeding disorder a “high value disease” because drugs to treat it can exceed. $ 1 million a year for a single patient. .

Aduhelm, at $ 56,000 a year, is a steal, but hemophilia is a rare disease and Alzheimer’s disease is terribly common. Medicines to combat it will be sold and taken. The crucial studies that will define their true benefit will take many years or may never be completed. And from a business standpoint, it doesn’t really matter.

KHN (Kaiser Health News) is a national newsroom that produces in-depth journalism on health issues.

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