For the first time in 18 years, the Food and Drug Administration has approved a new drug to treat Alzheimer’s disease patients. But the FDA’s decision is controversial because there is strong disagreement in parts of the medical community about whether the drug even works.
Aducanumab, which goes by the brand name Aduhelm, is a once-monthly intravenous infusion intended to slow cognitive decline in mild cases of Alzheimer’s. It will cost around $56,000 per year, not including the tests and brain scans that will be required.
The agency’s independent advisory committee said the clinical trials were not conclusive enough to warrant approval. Other opponents include some doctors who treat Alzheimer’s patients and several prominent experts in the field.
But the biggest boosters of the drug are patient advocacy groups that point out that no new drug has been approved in 18 years while the disease ravages more and more elderly Americans.
FDA director of the Center for Drug Evaluation and Research, Dr. Patrizia Cavazzoni, said the trials left “residual uncertainties” about the efficacy of the drug.
But, she said, the agency had decided to approve the drug through a program called accelerated approval, which is designed “to provide earlier access to potentially valuable therapies for patients with serious diseases where there is an unmet need, and where there is an expectation of clinical benefit despite some residual uncertainty regarding that benefit.”
Michel Vounatsos, Biogen’s chief executive, hailed the approval and said in a statement, “We are committed to sharing our future insights about Aduhelm with the scientific community as we collect more data from the real-world use of this treatment.”
“Once the product is approved, the cat’s out of the bag, the horse is out of the barn,” said Dr. G. Caleb Alexander, a member of the FDA advisory committee. “There’s no way to recover the opportunity to understand whether or not the product really works in the post-approval setting.”
That’s probably why the FDA has ordered Biogen, the drug’s maker, to conduct another trial. But there are several unique problems for doing a post-approval clinical trial.
Alzheimer’s trials are already challenging to conduct because it is often difficult to recruit enough participants. Because the condition can progress very gradually, trials need to be large and continue for many months in order to be able to see if a drug is slowing cognitive decline.
Several experts expressed skepticism that Biogen would be able to recruit many participants in the United States for a post-market trial because patients who can get a drug from their doctors are often reluctant to take the chance of receiving a placebo in a clinical trial.
“Although the Aduhelm data are complicated with respect to its clinical benefits, FDA has determined that there is substantial evidence that Aduhelm reduces amyloid beta plaques in the brain and that the reduction in these plaques is reasonably likely to predict important benefits to patients,” Dr. Cavazzoni, of the FDA, wrote on the agency’s site.
For caregivers and the families of Alzheimer’s patients who, in many cases, are desperate for help, Aduhelm is a godsend. There is such a helpless feeling watching a loved one succumb to the disease that any positive effects at all are most welcome.
There are other Alzheimer’s drugs undergoing trial, but none of them is expected to be approved for three or four years.