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Last year, we covered the controversy over the FDA’s approval of Aduhelm, the new — and first ever — drug treatment to claim the ability to slow the progress of Alzheimer’s disease. More has happened in the last few weeks.

To summarize, the FDA approved the drug under a process that bypassed its usual “safe and effective” standard. Instead, Aduhelm received “accelerated approval” based on an expectation that its proven ability to remove clumps of amyloid protein in the brains of patients (one of several biomarkers of the disease) ought to reduce patient symptoms.

Unfortunately, the studies on which the approval was based were in conflict on this point. In fact, two early studies of the drug were halted by the manufacturer itself due to disappointing results.

Furthermore, of the 11 medical experts convened by the FDA to consider all the study evidence, 10 indicated the studies failed to show “clinical effects,” and additionally expressed uncertainty even as to the role of amyloid in Alzheimer’s disease.

Not only did the studies not prove Aduhelm to be effective, they revealed some very serious — in one case fatal — side effects, including falls, brain bleeding and brain swelling.

Despite its uncertain (unlikely?) benefit, the drug’s developer, Biogen, proposed to charge patients (in most cases, that means Medicare) $56,000 a year for the product.

Today, more than one out of 10 Medicare recipients suffer from Alzheimer’s. As a result, when Medicare set its premium rates for 2022, it raised them by the largest dollar-amount ever: $21.60 per month, about half of which it said was due to the expected cost of covering this one drug.

That means 56 million Medicare recipients are each being charged $130 more this year to cover the cost of this dubious treatment for eligible patients.

In my view, there are several parties responsible for this egregious situation: The researchers who pushed the FDA to approve the drug despite the absence of the usually required evidence; the patient advocacy groups who have sought a treatment — any treatment! — for Alzheimer’s for decades and lobbied the FDA heavily for approval; the manufacturer, who reportedly priced the drug based on “comparables” from life-saving cancer treatments (Aduhelm is not a life-saver, or even a proven life-extender at present); and even competitor drug manufacturers pursuing similar lines of research who hoped to piggyback on the approval of Aduhelm to promote their own treatments.

Fortunately, though the FDA overlooked its “safe and effective” standards in this case, the Centers for Medicare & Medicaid Services (CMS), which administers Medicare, did not ignore its “reasonable and necessary” standards for covering the drug’s cost.

Normally, Medicare pays for all FDA-approved drugs. This year, however, CMS announced it would cover Aduhelm’s cost only for patients enrolled in an approved double-blind study of the treatment’s effectiveness.

I will also note that, due to a great outcry over Aduhelm’s originally proposed cost, and also to the reluctance of many leading hospitals and medical practices to prescribe the treatment, Biogen cut the price in half. As a result, Medicare is now considering whether to reduce the current premium. They certainly should, not only because of the price drop, but due to the very limited coverage Medicare is offering.

Here’s how I view the characters in this modern-day morality play:

• The FDA, in providing accelerated approval, succumbed to political and other pressures, ignoring long-standing rules, established expectations, and the judgment of its experts.
•  Biogen was guilty of overreach both in pushing for full approval without satisfactory evidence, and aggressively pricing this drug of limited effectiveness. On the plus side, at least the company responded to market pressures and reduced the price, though it’s still exorbitant.
• Medicare made two smart calls: first, in pricing this drug into 2022 premiums, and second, in publicizing the degree to which the new drug was responsible for the increase. This transparency appropriately galvanized public response. I think it would be wise for Medicare to act similarly whenever extraordinarily priced drugs have a measurable effect on premiums.
•  CMS was not wrong to both limit coverage of the drug and to require all patients receiving it to participate in a study. However, I wish those studies were not required to be placebo-controlled, because that means half the patients will not be receiving the real drug, and that the number (and locations) of participants will be highly limited.

Another, less onerous route exists for collecting study data on new drugs: a CMS-approved registry. That would permit more patients to obtain Aduhelm from doctors all over the country, and require those doctors to submit data to CMS that could be used to prove (or disprove) the new drugs’ effectiveness over time in real-world settings.

CMS’s position is that placebo-controlled studies must first establish evidence of effectiveness so that registry data can be compared against those results. Once such studies are complete, the registry can be utilized to gather data more broadly through the population.

That means most Alzheimer’s patients and their caregivers and families will have to wait several more years before finding out if they will get a chance to try the much-hyped, much hoped-for Aduhelm or one of the other treatments still in development.

Let’s hope that day comes. It can’t be too soon.