In its about anti-amyloid monoclonal antibodies for Alzheimer's disease, CMS upheld its proposed plan to cover aducanumab (Aduhelm) only for people in randomized controlled trials.
However, the same restrictions won't necessarily apply to all monoclonal antibodies that target amyloid in Alzheimer's, the agency said.
"We are finalizing the majority of the proposed decision with a slight modification that reflects the feedback we received during the public comment series to create a long-term coverage pathway," said Lee Fleisher, MD, CMS chief medical officer, in a press conference on Thursday.
"Overall, this decision is a two-part final coverage determination: one for drugs that demonstrate effectiveness and clinical benefits as determined through FDA traditional approval, and one for drugs where there is a reasonable assurance of clinical benefit as determined through FDA accelerated approval," Fleisher added.
Under , anti-amyloid monoclonal antibodies approved by the FDA on a surrogate endpoint like amyloid reduction will be covered in an FDA or NIH clinical trial. To date, the only such approved treatment is aducanumab.
Anti-amyloid monoclonal antibodies that show efficacy of a direct measure of clinical benefit may be covered in broader types of CMS-approved research, like registries or other prospective studies.
"This long-term pathway is meant to be nimble and respond to any new drugs in this class that are in the pipeline and demonstrate clinical benefits," Fleisher said.
The agency reviewed more than 10,000 comments and information from more than 250 documents, including the recent publication of the phase III aducanumab trial data, before reaching its decision.
"This final National Coverage Determination reflects CMS' commitment to provide the American public with a transparent, trusted, evidence-based decision -- without regard to cost -- that is made only after a thorough analysis of public feedback," said CMS Administrator Chiquita Brooks-LaSure in a .
"Through this decision, we are creating a pathway for people with Medicare to quickly access drugs the FDA determines have shown a clinical benefit and encourage manufacturers and trial administrators to ensure that the clinical trials recruit racially diverse participants," Brooks-LaSure added.
The Alzheimer's Association, a group that lobbied hard against Medicare's proposed coverage restrictions, pointed out that CMS incorporated some recommendations it and Alzheimer's patients and families had made, including a registry for future treatments that are granted full FDA approval.
But overall, "the decision by CMS is a step backward for families facing Alzheimer's disease," Maria Carrillo, PhD, chief science officer of the Alzheimer's Association, said in a . "Years of increased research funding have led to more progress and innovation than ever before, but today's decision may halt this progress as developers question if there is a pathway forward to coverage."
Others saw the CMS decision as the best choice for people with Alzheimer's at this time. "It restores one's faith in regulators and government doing the right thing for the most people," Lon Schneider, MD, of the University of Southern California in Los Angeles, told 鶹ý.
"The FDA fumbled it," Schneider observed. "CMS saw what most of us saw -- the lack of evidence for substantial efficacy and safety when aducanumab is given to early Alzheimer's patients every month for 18 months -- and therefore, found aducanumab not to be 'reasonable and necessary' treatment that should be covered by Medicare."
The restrictions outlined in the CMS determination, including the distinction between accelerated approval and traditional approval, have never been applied to drugs for other diseases, aducanumab maker Biogen said in a released late Thursday.
"Biogen is carefully considering its options and will provide updates as the company further evaluates the business impact of this decision," the statement read.