Earlier this month, the Centers for Medicare and Medicaid Services announced that Medicare would cover Aduhelm, the first Alzheimer’s medication approved by the Food and Drug Administration in nearly two decades. But just a tiny subset of beneficiaries with the neurodegenerative disease will be covered.
The agency’s decision to more or less ration access to Aduhelm could gut research efforts into drugs for other diseases without effective treatments.
Last year, the FDA greenlit Aduhelm through its accelerated approval pathway, which grants patients faster access to drugs that treat serious conditions and fill an unmet need based on a surrogate endpoint. According to the FDA, such an endpoint is a marker that is thought to predict clinical benefit but is not itself a measure of clinical benefit.
In the case of Aduhelm, clinical trials showed that the drug effectively reduced amyloid-beta plaque, a hallmark of Alzheimer’s. But CMS has decided it needs more data on the drug before agreeing to pay for it — and is only covering Aduhelm for the few patients who participate in further clinical trials.
It’s not a stretch to think CMS could similarly restrict future accelerated-approval medications. That could make investors wary of funding the research and development of potentially life-saving treatments.
Developing a drug is a long, risky undertaking that costs an average of $2.9 billion. If CMS, the agency with jurisdiction over the 83 million people on Medicaid and the 63 million people on Medicare, can decide to refuse coverage of FDA-approved medication, then investors could decide that drug development, especially via the accelerated approval pathway, isn’t worth the financial risk.
CMS’s decision may force patients hoping for effective treatments for diseases such as cancer, cystic fibrosis, and Alzheimer’s to wait even longer.
Sally C. Pipes is president, CEO, and Thomas W. Smith fellow in Health Care Policy at the Pacific Research Institute. Her latest book is False Premise, False Promise: The Disastrous Reality of Medicare for All (Encounter 2020). Follow her on Twitter @sallypipes.
