Opinion

Editorial: This isn’t how the FDA should OK Alzheimer’s drugs


The Food and Drug Administration didn’t just make a mistake by approving a new Alzheimer’s disease drug this week. It turned what is supposed to be an objective and science-based process on its head, giving the go-ahead to a drug for which there is far more evidence that it doesn’t work than evidence that it does.

Patient advocates were, of course, avid to see drugmaker Biogen’s aducanumab come to market as a potential treatment for mild stages of the dementing disease, which so far has resisted efforts to combat it. Any hope, no matter how dim, is at least something in the eyes of families who watch their loved ones deteriorate mentally and physically for years before death takes them.

The problem is that in this case, the hopes may be false ones. Aducanumab’s performance in two large clinical trials was so unimpressive that Biogen halted the studies. But then a collaborative effort by the FDA and Biogen teased out additional data from one of the studies that showed a small reduction in cognitive decline among a subset of subjects with mild Alzheimer’s who were given a higher dosage of the drug. And even then, the effects were modest.

No previous trials examining this same therapeutic pathway — using an antibody to remove amyloid beta, a protein associated with the development of Alzheimer’s — succeeded in humans.

It was odd and unsettling for the FDA to collaborate with the drugmaker applying for approval — so much so that the consumer advocacy group Public Citizen appealed to the Department of Health and Human Services to examine what they said represented an overly cozy relationship. The FDA then overruled its own advisory committee, which overwhelmingly recommended against approving the drug.

To be clear, we are all for the federal government throwing its dollars and research might into solving the Alzheimer’s puzzle. That should be a top priority, considering the severity of the disease and the greater toll it is expected to take as Baby Boomers age. But the agency’s job is to ensure the safety and efficacy of drugs, not find ways to make them look better.

Still, if some people might benefit, if only a little bit and only for a short while, why not allow the drug to be used? It does have some side effects in sizable numbers of patients, including brain swelling and bleeding, but those effects were mild and easily controlled. So why not give it a shot when there are no other real options?

The answer is twofold: High cost and misdirection of resources.

Aducanumab is expected to cost $20,000 to $50,000 a year per patient, and 2 million Americans — about a third of all those with Alzheimer’s disease — would be considered candidates to take it. Further, in order to identify and manage the possible side effects, the patients taking aducanumab would need ongoing, expensive screening.

There’s a notion in this country that cost is no object when it comes to new treatments to dread diseases — a nice saying but a false one. Because Alzheimer’s is primarily a disease of age, Medicare would pick up most of the tab for a pharmaceutical that shows little benefit, at the same time that patients who desperately need nursing and memory care get practically nothing in the way of Medicare coverage. Government programs, private insurers and healthcare providers make choices to withhold healthcare all the time; it’s just that the system generally favors covering drugs, devices and surgeries over ongoing caregiving to improve quality of life.

While the cost of healthcare heads inexorably higher, the nation has to start asking itself what to do with its finite healthcare dollars. Massive expenditures on pharmaceuticals that lack robust results in clinical trials aren’t the way.

In addition, the approval of aducanumab could slow the development of other, more effective treatments. If most of the patients with mild Alzheimer’s are on this drug, they’re not good candidates for clinical trials of other drugs.

Various scientists recommended at least a third large clinical trial, using the higher dose, to produce stronger evidence one way or the other. The FDA decided against even that prudent route. This drug needs continued study in the general population of Alzheimer’s patients, and if it isn’t proving its mettle, it should be pulled from market.

The worst scenario of all would be for the approval of aducanumab to represent a lopsided new process for considering drug applications. Yes, the nation developed a streamlined approval path for vaccines against COVID-19, but that was an emergency situation and the vaccines were shown in trials to be extremely effective and safe — which is how they have operated in the general population. That’s not the situation here. The FDA should be casting a rigorous eye on new medications, not collaborating with drug companies to ease the path of drugs with a dubious record of success.




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