More than 6 million Americans suffering from Alzheimer’s just received a gut-punch from the federal agency that oversees Medicare. So did their loved ones.
In mid-January, the Centers for Medicare and Medicaid Services made an unprecedented decision to cut off most seniors’ access to an entire class of Alzheimer’s treatments. CMS’s actions will have a significant impact on dementia research — and could dash Americans’ hopes for a cure. And this decision will hit vulnerable minority and other underrepresented populations especially hard.
The decision most immediately impacts Medicare enrollees who were considering Aduhelm, a medicine that the FDA approved in June. The drug reduces amyloid plaque in the brain, which many — but not all — scientists believe is a trigger for Alzheimer’s.
The FDA’s approval sparked debate — partly due to the drug’s side effects and cost, but also due to concerns over its effectiveness. But the FDA weighed the science and determined that an accelerated approval — which the agency has used for promising treatments for HIV and cancer in the past — offered the best path forward. It gives patients, many of whom are hardest hit by a disease, access to a needed therapeutic option, while additional studies are done.
The bureaucrats at CMS evidently disagree with FDA scientists’ approach, which has long been viewed as the gold-standard for science-based safety and efficacy standards.
The Medicare administrator announced it would restrict access to Aduhelm — and any other future “monoclonal antibodies that target amyloid for the treatment of Alzheimer’s disease” — to a minuscule sliver of people enrolled in CMS-approved clinical trials, all of which could take several years. The proposal excludes millions of Medicare beneficiaries currently living with the disease, and it will impact people of color particularly hard.
Studies show that over the last two decades, even though minorities are at an increased risk of developing Alzheimer’s, 94.7 percent of clinical trial participants for developmental Alzheimer’s treatments have been white.
The announcement also has implications beyond Medicare. Commercial insurers typically mimic the agency’s coverage decisions, so the pronouncement will adversely impact patients with mild cognitive impairment (MCI) and mild Alzheimer’s who have private insurance.
The decision marks a significant setback for Alzheimer’s research. Nearly 100 Alzheimer’s programs have failed in the past decade. So the FDA’s accelerated approval of Aduhelm, the first new Alzheimer’s treatment since 2003, wasn’t just hopeful news for patients; it was a historic event that gave researchers, and investors, confidence to continue plugging away in search of new treatments.
Today, there are companies of all sizes working to develop treatments to stop, prevent, or slow the progression of Alzheimer’s using a variety of novel strategies. Small biopharma companies are leading the majority of these programs.
If Medicare — which pays for the vast majority of Alzheimer’s treatment — won’t cover a new drug, despite the FDA’s experts ruling that the medicine has great potential, companies would be foolish to keep investing in risky, hugely expensive Alzheimer’s research.
CMS officials are, in effect, setting themselves up as a second drug-approval agency. This sets a terrible and potentially far-reaching precedent. The staff at the FDA is approximately three times the size of the CMS. They are scientifically trained at judging the benefit and risk of drugs and have a long history of getting academic feedback. CMS is not equipped for this. They lack the expertise to tackle these scientific questions — and legally, the agency appears to have exceeded its authority.
In its announcement, CMS made clear that it would only be satisfied by clinical trials of Aduhelm that are done under its own aegis, and that its determination to restrict access to the drug was the result of its own “thorough review process.” These words and actions are a dangerous rebuke to the FDA’s scientific autonomy and decision-making authority.
If unchallenged, this reproach — and this unilateral assertion of drug-evaluation powers — could harden into a precedent that could damage our treatment-approval process irreparably. And the onslaught of Alzheimer’s and other difficult to treat diseases will continue unchecked for generations to come.
Michelle McMurry-Heath is a physician-scientist and president and CEO of the Biotechnology Innovation Organization.
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