Earlier this year, the Centers for Medicare & Medicaid Services (CMS) broke new ground in several important respects when it issued a national coverage determination for antiamyloid monoclonal antibodies, a promising new class of drugs for the treatment of Alzheimer’s disease. Despite FDA’s approval of Aduhelm as safe and effective for its indicated use — it was the first drug in the class to obtain FDA approval — CMS decided that the drug had not been shown to be sufficiently safe and effective for its intended use to qualify for Medicare coverage outside the confines of the randomized controlled trials that had been conducted under CMS’s “coverage with evidence development” framework. Furthermore, CMS’s decision applied not only to Aduhelm but also to any future drugs in this class. It now seems likely the future antiamyloid antibodies approved by FDA through the accelerated approval pathway will also be ineligible for Medicare coverage except in randomized controlled trials. It also appears that future antiamyloid antibodies approved by FDA through the traditional pathway will be eligible for Medicare coverage only in CMS-approved prospective comparative studies that meet certain criteria specified by CMS.
CMS’s decision was troubling in that it departed from the agency’s past practice of deferring to FDA’s decisions regarding the safety and effectiveness of drugs. Historically, CMS’s policy has been that drugs approved for marketing by FDA were considered safe and effective for purposes of Medicare coverage when used for the indications specified on the label. Without acknowledging that policy, and without explaining why it was inapplicable or ought to be changed, CMS decided that FDA’s finding that Aduhelm was safe and effective for its labeled use provided insufficient assurance of clinical benefits to satisfy the Medicare requirement that a drug must be “reasonable and necessary” for the treatment of an illness. In another first, CMS restricted Medicare coverage for an entire class of FDA-approved drugs to randomized controlled trials, preemptively determining that even future antiamyloid antibodies approved by FDA would not be found to be sufficiently safe and effective to qualify for unrestricted Medicare coverage. The result is to severely restrict patient access to the first new FDA-approved Alzheimer’s treatment in years.
It will be important to watch whether CMS seeks to apply this new approach to other drugs or whether the decision will be treated as a one-time exception arising from the unique circumstances surrounding FDA’s approval of Aduhelm. If CMS does seek to apply the Aduhelm precedent more broadly, there may be grounds to challenge it on the basis that CMS is improperly usurping FDA’s role and prejudging the safety and effectiveness of drugs before FDA's approval process is even complete. We are already seeing drug approvals in other categories for which CMS might seek additional studies in the Medicare population to assess effectiveness. In any event, CMS’s decision underscores the importance of ensuring that the evidence supporting FDA’s approval decisions is as robust as possible and considering of CMS coverage issues early in the product lifecycle.