John Bayley quoted, “She is not sailing into the dark: the voyage is over, and under the dark escort of Alzheimer’s she has arrived somewhere. So have I.”
Alzheimer’s disease is a progressive neurologic disorder that causes the brain to shrink (atrophy) and brain cells to die. Alzheimer’s disease is the most common cause of dementia, a continuous decline in thinking, behavioral and social skills that affects a person’s ability to function independently.
The Food and Drug Administration approved a new drug for Alzheimer’s disease on Monday, the first such approval the agency has made since 2003. The U.S. Food and Drug Administration said it granted approval to the drug from Biogen based on results that seemed “reasonably likely” to benefit Alzheimer’s patients. Approximately 5.8 million people in the United States age 65 and older live with Alzheimer’s disease. Of those, 80% are 75 years old and older. Out of the approximately 50 million people worldwide with dementia, between 60% and 70% are estimated to have Alzheimer’s disease.
The decision, which could impact millions of older Americans and their families, is certain to spark disagreements among physicians, medical researchers and patient groups. It also has far-reaching implications for the standards used to evaluate experimental therapies, including those that show only incremental benefits. The new drug, which Biogen developed with Japan’s Eisai Co., did not reverse mental decline, but the studies show it slows down the rate. The medication, aducanumab, will be marketed as Aduhelm and is to be given as an infusion every four weeks.
“In determining that the application met the requirements for Accelerated Approval, the agency concluded that the benefits of Aduhelm for patients with Alzheimer’s disease outweighed the risks of the therapy,” the FDA stated.
The FDA acknowledged the gravity of its decision and said it is “well-aware of the attention surrounding this approval.” The agency said that, given the seriousness of Alzheimer’s, “it makes sense that so many people were following the outcome” of the FDA’s review. The regulator said the data included in the review was “highly complex and left residual uncertainties regarding clinical benefit.”
“There has been considerable public debate on whether Aduhelm should be approved. As is often the case when it comes to interpreting scientific data, the expert community has offered differing perspectives,” the agency added.
The FDA said it considered all data, followed the usual course of action, examined clinical trials and sought input from the Peripheral and Central Nervous System Drugs Advisory Committee, as well as patients.
“With all this said,” the FDA added that it is “extremely aware of the gradual and cumulative devastation that Alzheimer’s disease causes.” With more than 6 million Americans living with the devastating disease, the FDA called the need for treatments “urgent.”
“Although the Aduhelm data are complicated with respect to its clinical benefits, [the] FDA has determined that there is substantial evidence that Aduhelm … is reasonably likely to predict important benefits to patients,” the agency concluded. “As a result of FDA’s approval of Aduhelm, patients with Alzheimer’s disease have an important and critical new treatment to help combat this disease.”
Biogen did not immediately disclose the price, though analysts have estimated the drug could cost between $30,000 and $50,000 for a year’s worth of treatment.
A preliminary analysis by one group found that the drug would need to be priced $2,500 to $8,300 per year to be a good value based on the “small overall health gains” suggested by company studies. The non-profit Institute for Clinical and Economic Review added that “any price is too high” if the drug’s benefit isn’t confirmed in follow-up studies.
