It’s been nearly two decades since a new drug to treat Alzheimer’s has been approved by the Food and Drug Administration, and millions of patients and their families are desperate for new therapies against the incurable brain disease.
More than 6 million Americans are living with the memory loss and other debilitating effects of Alzheimer’s, with millions more expected to suffer from the disease as the population ages. Treatments are elusive: 99% of clinical trials of new Alzheimer’s drugs end in failure.
Any drug that proved successful would be a rare scientific breakthrough.
Yet we fear that the celebration among some patient advocates and families over Monday’s FDA approval of aducanumab — the first new Alzheimer’s drug since 2003 — is false hope.
There are simply too many unanswered questions about the drug’s effectiveness and safety, including the threat of potential side effects such as brain swelling or bleeding.
Instead of celebrating, we’re troubled by the FDA’s action.
Consider the evidence for and against the drug, which surely will make billions of dollars for drug maker Biogen, given the company’s current price tag of $56,000 a year for Aduhelm (the brand name for aducanumab).
Biogen’s CEO says that price is “fair,” but we have to ask: For whom? Surely not the families, many of them undoubtedly everyday working people, who will have to cover, somehow, whatever costs are not paid by Medicare or other insurance. Nor for the people struggling already to pay for insurance premiums and will likely see those costs rise now.
All for a drug that the FDA’s own independent advisory board rejected for approval last November.
More than a year before that, in March 2019, two clinical trials of aducanumab were halted after an independent data monitoring board found that there was insufficient evidence of its effectiveness against the devastating cognitive decline that is the hallmark of dementia.
Biogen was able to muster support for aducanumab based on another analysis that found it had a very small promising effect on cognitive decline: a change of 0.39 on an 18-point scale that rates memory, problem-solving skills and function.
But when medical experts take the extraordinary step of calling out the FDA’s decision in news stories and op-eds across the country, how confident can we be? Typical was the reaction of Jason Karlawish, a neurologist and professor of medical ethics and health policy at the University of Pennsylvania, who said he won’t prescribe the drug because of the “incomplete and contradictory” data.
Keep the pressure on
The Institute for Clinical and Economic Review, an independent research organization that conducts cost-benefit analyses of new drugs, said in a statement following the FDA’s approval that the agency had “failed in its responsibility to protect patients and families from unproven treatments with known harms.”
The FDA can still do what’s right by patients and families.
Biogen was granted “accelerated approval,” a move the FDA takes to bring innovative new treatments to patients as quickly as possible. As part of accelerated approval, a drug maker must conduct additional clinical trials to prove conclusively whether or not a drug works and is safe.
It’s up to the FDA to keep the pressure on so those trials are done as quickly as possible.
Patients and families need conclusive proof that aducanumab works — or does not.
False hope is not worth it, at any price.
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