Back last June, the FDA made the controversial decision to approve a medication intended to treat Alzheimer’s disease, despite the lack of solid evidence of that drug’s effectiveness, and the strong appearance that it had cherry-picked studies to include only a single trial and exclude those which showed no effectiveness. What’s more, the drug, Aduhelm, had significant side effects, a list price of $56,000 per year, and even its theoretical mechanism of action did not square with emerging developments in the research. From all reports, the drug was approved because of the strong wish for an effective treatment rather than because this particular drug was effective.
But earlier this month, the Centers for Medicare and Medicaid Services (CMS) provided Medicare-watchers with some good news: they plan only to cover this drug only through “coverage with evidence development (CED),” which means that “FDA-approved drugs in this class would be covered for people with Medicare only if they are enrolled in qualifying clinical trials.” This remains only a draft proposal, with a 30 day comment period before it is finalized, expected to be on April 11.
As Rachel Sachs at Health Affairs explained immediately following the announcement,
“By law, Medicare only covers products and services that are “reasonable and necessary” for diagnosis or treatment. CMS is able to use the NCD [National Coverage Determination] process to evaluate the evidence in support of a new product or service and determine whether this standard is met. Although most products that meet the FDA’s standard of “safe and effective” are likely to meet the “reasonable and necessary” bar, the two are in fact different. Yesterday’s NCD provides a strong, clear statement of CMS’ independence and willingness to enforce its own legal standards for its own agency priorities.”
And the New York Times reported that this was, in fact, “the first time that C.M.S. limited Medicare beneficiaries’ access to an F.D.A.-approved drug in this way.”
This followed prior reports that nearly half the 2022 Medicare Part B premium increase, or about $10 per month, was due to Aduhelm costs, though subsequently manufacturer Biogen announced a price cut down to $28,000.
In the meantime, the public comments submitted thus far can be viewed by the public at the CMS website, and a skim through those comments posted as of this writing (and largely dating to the period just after approval, in July/August of 2021) by medical professionals, shows widespread criticism of the FDA approval.
And Biogen has responded to the CMS decision by increasing the enrollment levels of its confirmatory trial, though only from 1,300 to 1,500 participants, a far cry from the expected 50,000 patients if it was widely available. At the same time, a rival drugmaker, Eli Lilly, has submitted initial data for its own similar drug, donanemab, for which it hopes to receive approval and launch at the end of this year. If Eli Lilly’s data is similarly inconclusive, of course, it is deserving of the same caution.
So far, so good — in fact, a rare win for common sense.
Unfortunately, though, the Alzheimer’s Association disagrees, and is engaged in a campaign to call on the public to oppose this decision — a campaign which readers who spend time on twitter will have noticed in the form of frequent ads urging people to “tweet at the president” to tell him to reverse the decision. They imply that this is discrimination, and that people with Alzheimer’s are being treated unfairly, saying in their alert that “No president has allowed this to happen with treatments for diseases like cancer, heart diseases and HIV/AIDS, and we can’t let it happen for Alzheimer’s.” And similarly, in the news update on their website, they write, “Medicare has always covered FDA-approved treatments for those living with other conditions like cancer, heart disease and HIV/AIDS. For CMS to treat those with Alzheimer’s disease differently than those with other diseases is unprecedented and unacceptable” — wholly failing to mention the very real concerns about whether this drug is effective in the first place. And, yes, if a drug with similarly-questionable effectiveness were on the table for cancer or heart disease, I would hope that CMS would be just as cautious.
And, finally, a few words of international comparison: as Medicare was deliberating on its decision, December, the European Medicines Agency voted against approval of Aduhelm (which is most likely binding for the UK as well), and Japan likewise decided to require additional data, a step that could require several further years as they wait for the same additional study results as CMS is requiring.
Whether CMS stands its ground or succumbs to public pressure remains to be seem, but their decision on Aduhelm is an encouraging sign.
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