Biogen’s Alzheimer’s drug Aduhelm received FDA approval for less clinical data than is usually required, so the Centers for Medicare and Medicaid Services chose a coverage pathway that allowed it to gather more evidence.The agency Tuesday suggested The drug is only given to those who participate in an eligible clinical trial.
CMS covers items and services deemed reasonable and necessary under the law. There is a provision in the law that allows limited coverage when there is insufficient data to determine whether something meets these standards. “Evidence-Developed Coverage” is a mechanism that enables CMS to cover drugs in the context of clinical studies, thereby generating additional data that can support broader coverage decisions. The pathway CMS proposed for Aduhelm is preliminary, but it applies to all therapies in its drug class. A final decision by CMS is expected on or before April 11.At the same time, this public way for the next 30 days.
Aduhelm is an antibody designed to reduce beta-amyloid plaques that build up in the brains of people with Alzheimer’s disease. Phase 3 testing of the drug produced mixed results about how well the drug worked. In addition to offering unclear patient benefits, the drug carries risks of complications, some of which can be fatal. But instead of asking the Cambridge, Massachusetts-based company to conduct another clinical trial, The FDA granted the drug accelerated approval last June. Such a decision is based on a surrogate endpoint indicating that a drug may be working.The benefit of the drug must be demonstrated in post-marketing studies, which Biogen plans to start later this year.
This Proposed CMS Decision Noting that to date, no clinical trials of antibody drugs targeting amyloid have shown clinically meaningful improvements in cognition and function in Alzheimer’s patients, and that “there is insufficient evidence for the use of monoclonal antibodies against amyloid. is a reasonable and necessary treatment for Alzheimer’s disease.” CMS further noted that the statistical significance of one of the two pivotal studies could only be concluded by a post hoc analysis of the data. A secondary analysis of patients with high doses of the drug raises questions about the reliability of clinical trial results. The agency also pointed to the drug’s safety risks, which have been linked to amyloid-related imaging abnormalities (ARIA), brain hemorrhage and swelling detected on MRI scans.
“Because of the lack of clear clinical benefit and the frequent occurrence of adverse events such as ARIA, the evidence does not support the benefits of (monoclonal antibodies) targeting amyloid therapy (Alzheimer’s disease) outweigh the harms,” the proposed CMS decision states. “Adverse events are monitored and treated more closely in clinical trials than in general practice. Currently, we have additional concerns about harm caused by patients being treated outside of the safety monitoring of controlled trials.”
The proposal that CMS only offer participants Aduhelm is a restrictive one, but not without precedent. The agency has published reports of evidence development decisions in the past. Stem cell transplants and cochlear implants for blood disorders are among the products that have obtained such a determination. Howard Fillit, co-founder and chief scientific officer of the Alzheimer’s Drug Discovery Foundation, said the CMS proposal is appropriate and consistent with the FDA’s accelerated approval decision based on limited data.
“This decision supports additional clinical trials to further understand the clinical efficacy and safety of this drug in real-world populations,” Fillit said in an emailed statement. “This decision has implications for other similar drugs in late-stage development. If these trials show clearer and more robust clinical efficacy, the FDA may fully approve these amyloid monoclonal antibodies, and Medicare may provide full payment .”
Questions about Aduhelm’s benefits swirled around the drug prior to FDA approval, and continue to do so now. Prescribing new drugs has been slow. last month, Biogen slashes the drug’s $56,000-a-year wholesale price in half to boost sales. If CMS proposes to complete Aduhelm only for patients in clinical trials in April, the number of insured patients will continue to be small. CMS will not conduct clinical studies to gather more evidence about Aduhelm. Entities that want to conduct clinical trials will submit proposals to the agency for approval. But as long as the study meets CMS standards, the agency will provide Aduhelm to study participants. Based on the results of these studies, CMS could revisit its coverage decision for the drug.
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