Terry H. Schwadron
June 10, 2021
Key to whether we “trust” our institutions is whether we can understand what they are doing, and a distinct sense that they run both fairly and consistently.
This week, we saw some frank public befuddlement over a Food and Drug Administration (FDA) decision that greenlights the use of a new, experimental Alzheimer’s medication that may or not work for a terrible disease besetting millions. Indeed, the news media had no difficulty locating doctors who said they would not prescribe the drug without a lot more testing.
Of course, our hearts go out quickly to anyone who has seen or tried to help someone with Alzheimer’s or other forms of dementia. Experiencing someone, particularly a parent or senior, seemingly physically sound but too often lost in a different world is a heartbreak, and it is difficult to be helpful.
So, yes, any drug that comes along and says it can help reduce the suffering is one that we usually welcome. But the FDA decision came here before the full clinical trials that we expect when the government declares a medication safe and effective.
That, in turn, further undercuts trust for other times when the government tells us that particular drugs are ok, including, as we see, coronavirus vaccines which still carry an “emergency use” approval.
In this case, FDA approval came despite warnings from independent advisers that the much-debated treatment hasn’t been shown to help slow the brain-destroying disease. The FDA approved the drug from Biogen based on study results showing it seemed “reasonably likely” to benefit Alzheimer’s patients. It’s the only therapy that U.S. regulators have said can likely treat the underlying disease, rather than manage symptoms like anxiety and insomnia.
Expense and Payment
The New York Times has reported that the drug, which is more expensive than anticipated — Biogen said it would charge an average of $56,000 a year per patient — will obviously boost profits for that company and for clinics distributing it with most of the cost likely to end up costing taxpayers through Medicare payments. It’s a big enough overall cost to affect private health insurance premiums as well.
That, of course, makes it a political and social issue as well as a medical one.
The drug, which will be called Aduhelm, is a monoclonal antibody administered monthly that targets a protein in the brain that clumps into plaques in patterns that are associated with Alzheimer’s patients. It is the first new Alzheimer’s treatment since 2003 and the first ever approved to attack the biological underpinnings of the disease, instead of merely postponing symptoms.
Basically, it reduces the clumps, and in its testing, Biogen says it has slowed development of Alzheimer’s in about 22 percent of cases against a group given a placebo.
Under terms of this accelerated approval, the FDA is requiring Biogen to conduct a follow-up study to confirm benefits for patients. If the study fails to show effectiveness, the FDA could pull the drug from the market, though the agency rarely does so. Other studies will target side effects.
Alzheimer’s gradually attacks areas of the brain needed for memory, reasoning, communication and basic daily tasks and can affect even the ability to swallow. Patients cannot care for themselves. And, with the aging across the globe, the disease is spreading.
No one has determined the exact cause.
So, we have groups representing Alzheimer’s patients and their families say any new therapy — even one of small benefit — should warrant approval. And we have experts warning that this is not a treatments of proven benefit. The FDA’s own panel of outside neurological expert offered a scathing assessment in November to the data from a single study by Biogen — whose stock is up 40 percent this week.
Without strong evidence that the drug helps patients and with potentially serious side effects, groups including a professional society representing health care providers for older adults, urged the agency not to approve the drug.
In any case, it remains unclear how Biogen’s positive results translate into practical benefits, like greater independence or ability to recall important details.
What is clear to the rest of us citizens is just how dependent we are on the outcome of purely scientific debate. Our generalized assumption until the last few years has been that we should trust the FDA to reach such conclusions.
The FDA doesn’t seem to involve itself in how we pay for new drugs, or whether there is sufficient public interest in changing the speed of disease spread or even demographic change in the country. It has elected to be more scientifically “pure,” as if that is separable from the impact.
But we do expect that the FDA is looking at actual medical effectiveness. If not, why do we have an FDA?
Obviously the fights over the last year over masks, vaccines, the potential off-label use of alternative treatments for coronavirus have all felt much more political than scientific, exactly a reflection that the impact is being seen alongside medical or social benefit. Those experiences mesh with views over environment and climate, over social studies of what’s behind discrimination of different sorts over almost everything that undergirds policy-making in Washington these days.
If we do or don’t like the outcome, the next step routinely has come to attack the “science” behind the conclusion. That spells “trust.”
We need a pill for that.