The Aduhelm verdict is in—but what now? The Centers for Medicare and Medicaid Services will only cover aducanumab in the context of a clinical trial. Impassioned lobbying on both sides of the issue moved the agency to make a few changes from its draft decision laid out in January, but as per the final ruling, all anti-amyloid immunotherapies approved by the Food and Drug Administration will be subject to “coverage with evidence development.” CED is a restrictive process CMS uses when it wants more data about the effects of a new treatment.
CMS spelled out three questions it wants answered before providing full coverage for anti-amyloid antibodies.
The questions address whether the benefits of treatment outweigh harms in the general Medicare population.
CMS promised to swiftly reconsider its decisions once sponsors generate new data on clinical benefit.
The April 7 announcement sparked headlines and strong reactions, and yet it left many researchers confused about what this National Coverage Determination (NCD) actually means for the development of these therapies. The 67-page decision memo was written by CMS analysts led by Tamara Syrek Jensen. It contains a wealth of detail that explain key issues and is worth a deeper examination. Here’s an Alzforum primer to help you understand the nuances of this important decision.
For starters, the final decision did answer some questions researchers had asked about how the proposed CED process would work. For example, CMS will cover Aduhelm and tests ascertaining the presence of brain amyloid in FDA-required confirmatory trials, and it loosened proposed restrictions such as requiring the drug to be administered in a hospital.
Essentially, the final decision creates two pathways for obtaining full coverage. One is for antibodies that have accelerated approval based on amyloid removal, the other is for antibodies with traditional approval based on clinical efficacy data. The former need to complete at least one additional randomized controlled trial (RCT), but the latter can use registry-based comparative studies or pragmatic trials, which compare treatment options in routine clinical practice.
Why ask for additional information on anti-amyloid antibodies with demonstrated efficacy? Because CMS wants answers to three questions: whether the treatment’s clinical benefit replicates in the general Medicare population, whether certain subgroups experience different benefits and harms, and how those benefits and harms change over time.
CMS acknowledged that these requirements would limit Medicare participants’ access to anti-amyloid immunotherapies in the near future. The agency said this is necessary due to the high risk of the brain edema and microhemorrhages known as ARIA, which in the aducanumab Phase 3 trials occurred in 41 percent of people on drug (Oct 2021 news; Dec 2021 news).
“It is appropriate access that matters, and we have real concern about potential harms to Medicare patients … CMS needs evidence that an anti-amyloid monoclonal antibody for the treatment of AD demonstrates improved health outcomes, with reasonable risk of harm, in broader community practice and in the broader Medicare population,” the agency wrote. This evidence would enable anti-amyloid immunotherapy to meet the “reasonable and necessary” criteria CMS uses for coverage of new treatments, it added.
Read the rest of this article on the Alz Forum Website – https://www.alzforum.org/news/community-news/drilling-down-cms-aduhelm-decision-primer
Read more on the trials and tribulations of Biogen’s Aducanumbal on our website – https://www.dementiaresearcher.nihr.ac.uk/tag/aducanumab/