In an unprecedented move, health officials with the Food and Drug Administration (FDA) have approved a new Alzheimer's drug based on results showing only that the drug was "reasonably likely" to help patients. Independent advisors have warned the FDA that the drug has not been shown to help combat the development of the disease.
Critics of the decision worry it could help erode the standards by which the FDA evaluates experimental treatments, some of which have been shown to be only minorly effective.
The new drug, aducanumab, was developed by Biogen
The function of the new drug is to help clear beta-amyloid, a protein, from the patient's brain. Other experimental drugs have performed this same function but did not improve patients' abilities to take care of themselves or think clearly.
In fact, there have been countless Alzheimer's drugs submitted to the FDA and denied in recent years. Biogen's approval is likely to encourage those companies to reinvest in their stalled drug developments.
The drug will go on the market under the name Aduhelm and is taken every four weeks as a living cell infusion. Biogen estimates that a year's supply for one patient will cost $56,000. Those with insurance coverage won't see a price tag anywhere near that high, but insurance companies may attempt to restrict who qualifies. Biogen has also promised not to raise this price for at least four years.
As a part of the FDA approval process, the drug must undergo a follow-up trial which the company hopes to complete by 2030.
The FDA's decision met with immediate backlash from doctors and other health and drug experts regarding the allegedly flimsy evidence that the drug does any good.
"The FDA gets the respect that it does because it has regulatory standards that are based on firm evidence. In this case, I think they gave the product a pass," said Dr. Caleb Alexander, an FDA advisor and medical researcher at Johns Hopkins University.
Alexander said he was "surprised and disappointed" by the drug's approval.
After the FDA's decision was announced, shares of Biogen jumped 38%. Analysts expect the new drug to bring in billions in sales for the pharma company.
However, a non-profit which studies drug values, the Institute for Clinical and Economic Review, has said that the only way to justify the drug's $56,000 price would be if it completely stopped the progression of dementia.
In studies on the drug, those taking aducanumab saw a 22% slower decline in thinking skills compared to patients taking the placebo. However, in practice, this 22% was only a 0.39 improvement on an 18-point cognitive ability test. The test also did not take into account things like patients' ability to care for themselves or remember important details.
Alzheimer's is a brain-destroying disease that slowly diminishes a person's ability to communicate, reason, remember, and even swallow in its later stages. Currently, roughly 6 million Americans have Alzheimer's. As the baby boomer generation ages, analysts expect the number of Alzheimer's sufferers to increase by millions.
While the families of those with Alzheimer's understandably are happy to see the approval of drugs that show any sort of benefit, no matter how small, experts worry about the precedent the decision sets for other hard-to-treat diseases.
"The FDA is empowering the physician to make the decision on diagnosis," the CEO of Biogen, Michel Vountatsos, said in an interview.
