The specter of Alzheimer’s disease and its harsh reality make the Food and Drug Administration’s approval of the first new drug to treat it since 2003 seem like a ray of hope. It may prove to be little more than a windfall for its maker, a financial blow to Medicare and a black eye for the FDA.
On June 21, the FDA granted accelerated approval for aducanumab, an intravenous drug that targets Alzheimer’s development rather than its symptoms.
The accelerated process is intended to speed potentially valuable therapies to patients with serious needs for which there are no available effective treatments. Alzheimer’s patients certainly fit that category.
Drug company Biogen, which will market the drug under the brand name Aduhelm, hailed the approval. Immediately after, Biogen announced a list price of $56,000 a year. Additional expensive diagnostic testing will also likely be necessary. The drug will be administered through monthly infusions.
Most of the treatment costs will fall on taxpayers through Medicare. Families that can afford it will face years of what could be staggering out-of-pocket costs. Aduhelm use should begin in the earliest stages of the disease but would have to be continued indefinitely.
This might seem like a reasonable trade-off if Aduhelm actually slowed or reversed the progression of Alzheimer’s disease. The scientific community, including the FDA, is not convinced.
The drug was effective in reducing amyloid beta plaques in the brain. Since the plaques are assumed to be responsible for at least some of the cognitive decline in Alzheimer’s patients, it seems as if the drug will prevent that decline. There just aren’t clinical trial results that prove that case.
One of two late stage trials showed a slight slowing of cognitive decline. The other showed no such effect. That trial was stopped early.
On the FDA website, the director for Drug Evaluation and Research admitted, “The data included in the applicant’s submission were highly complex and left residual uncertainties regarding clinical benefit.”
The FDA did require Biogen to conduct a new trial to show effectiveness. Once the drug is widely available, however, patients will be less willing to risk being placed in a placebo group, so that trial may never happen.
Few suffering from Alzheimer’s disease think about efficacy rates, long-term costs or follow-up clinical trials. They only see a new drug as a glimmer of hope.
The FDA seems to be the one hoping that this new drug works. It should have been more certain before approving it.
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