The FDA Believes in a Breakthrough Alzheimer's Drug. It's Time CMS Believed in it, Too.
Medicare recently announced that it will broadly cover Leqembi, a breakthrough treatment that slows memory loss for people with early-stage Alzheimer's.
The announcement puts the medicine within reach for roughly 1.5 million Medicare beneficiaries in the early stages of that fatal dementia. Previously, the drug was functionally off-limits -- even though the FDA approved it back in January -- due to barriers imposed by Medicare officials.
Unfortunately, those barriers remain in place for future Alzheimer's treatments that hit the market in the months and years to come. Unless Medicare officials remove those barriers, beneficiaries will needlessly struggle to obtain lifesaving medicines. And time is of the essence, since at least one other promising Alzheimer's treatment is expected to win FDA approval soon.
The Centers for Medicare & Medicaid Services -- the federal agency that oversees both health insurance programs -- first announced those barriers more than a year ago, before Leqembi ever came to market. The agency's actions were likely driven by concerns over the FDA's June 2021 "accelerated" approval of a different "monoclonal antibody" used to treat Alzheimer's.
At the time, CMS announced that it would cover any monoclonal antibodies that received accelerated approval from the FDA in the future -- but only if patients enrolled in approved clinical trials. Since only a few thousand people, at most, could possibly enroll in those trials, CMS's coverage policy amounted to a de facto denial of the treatment for nearly all Medicare beneficiaries.
CMS simultaneously agreed to cover monoclonal antibodies, such as Leqembi, that received traditional approval from the FDA in the future -- but only if those patients' doctors enroll them in a registry. CMS released few details about the registry process, leaving many patients worried that it would pose a significant bureaucratic hurdle.
Luckily, the registry for Leqembi seems easily navigable.
But the fact remains that CMS restricted access to an entire therapeutic class of drugs, all because it questioned the process that led to the FDA's approval of the first drug in that class.
It's now clear that there was no good reason for CMS's skepticism of all monoclonal antibodies. The FDA's traditional approval of Leqembi came by way of a rigorous confirmatory study. In June, an FDA advisory committee voted unanimously to recommend Leqembi for full approval.
Put another way, the FDA amply verified the drug, which successfully met gold-star safety and efficacy standards. For every other drug in history, the FDA's stamp of approval -- whether via the accelerated or traditional pathways -- has been enough for CMS to cover the medicine. But this time, CMS second guessed the FDA, establishing a registry so it could collect more real-world data.
As one researcher at the University of Southern California noted, "second guessing by CMS is unproductive."
In fact, it's actively harmful. Biotech companies make research investments with the implicit assumption that, if researchers beat the odds and manage to develop a medicine that's safe and effective enough to win FDA approval, it'll be covered by virtually all insurers.
By setting themselves up as secondary authorities on prescription drugs -- even though they're neither scientists nor drug experts -- CMS officials are calling that assumption into question. It's likely to have a chilling effect on research and development.
For generations, Alzheimer's patients have waited for the day when the FDA would approve safe and effective treatments. With the advent of multiple promising monoclonal antibodies, patients, doctors, and FDA scientists believe that day is finally here. It's time CMS believed it, too.
Howard Dean is the former chair of the Democratic National Committee and former governor of Vermont.
