The FDA Just Approved This Medication Against the Advice of Experts
An outside panel of experts warned that there's not enough evidence the drug is effective.
Approval from the U.S. Food and Drug Administration (FDA) is looked on as the gold standard of knowing you should be able to trust a product. The agency's approval means that a drug has been thoroughly tested and deemed to be safe and effective, with its benefits outweighing any potential risks. Generally speaking, the FDA's decision is in line with advice from doctors and researchers in the field, but there are some exceptions. In fact, the FDA just approved a new medication against the advice of outside experts. Read on to find out more about this controversial drug.
The FDA just approved a new drug for Alzheimer's disease against experts' advice.
On June 7, the FDA approved Aduhelm (aducanumab), a drug designed to treat Alzheimer's disease. According to a statement from the FDA, this is the first time the agency has approved a drug for Alzheimer's since 2003. The approval comes after an outside advisory panel urged the FDA to reject the drug back in November, because it hadn't been shown to help slow the progression of the disease.
NBC News reported that although the drug claims it can slow the rate of decline in Alzheimer's patients, it was only shown to do so in one study, which the panel did not feel was substantial evidence. The panelists also noted multiple "red flags" about the injectable treatment.
Lon Schneider, MD, director of the California Alzheimer's Disease Center at the University of Southern California and one of the site investigators on an aducanumab trial, told The New York Times, "There's so little evidence for effectiveness … I don't know what caught the FDA's fancy here."
The FDA acknowledged the controversy surrounding the approval.
Amid all the controversy, the FDA is requiring the drugmaker, Biogen, to conduct a follow-up study, the Associated Press (AP) reported. If the study doesn't demonstrate effectiveness, the FDA could remove the drug from the market. However, the AP notes that the agency rarely takes this action.
The FDA has already acknowledged the contention surrounding its recent decision. In a statement, the director of the FDA's Center for Drug Evaluation and Research Patrizia Cavazzoni, MD, noted that the agency was "well-aware of the attention surrounding this approval." She added that "the expert community has offered differing perspectives," and the data in the submission left uncertainties regarding the drug's benefit.
Experts say doctors will have to decide whether or not to prescribe the drug.
Jason Karlawish, MD, co-director of the Penn Memory Center and site investigator for Biogen's clinical trials of the drug, told NBC News that he disagrees with the FDA's decision. "It's going to present a real challenge for patients and caregivers and clinicians about whether to prescribe the drug," Karlawish said. "It's going to also present challenges for researchers who are conducting trials and are planning trials to develop better drugs." For his part, he said he would prescribe the drug only "after having had a conversation with the patient and family members about the notable uncertainties that surround whether the drug even works."
According to The New York Times, experts feel even if the medication were able to slow cognitive decline in some, the benefit would be so minimal that it would not outweigh the risk of swelling or bleeding in the brain that the drug caused in some patients during the trials.
The potential high cost of the medication is also causing controversy.
While Biogen has yet to disclose how much the drug will cost, the AP reported that estimates say it could be $30,000 to $50,000 for a year's worth of treatment. Meanwhile, a preliminary analysis was made that the drug should only be priced at $2,500 to $8,300 per year to be a good value based on the "small overall health gains." Per the AP, the nonprofit Institute for Clinical and Economic Review felt that "any price is too high" if the drug's benefit isn't confirmed in follow-up studies.