CMS investigates health outcomes in cerebrovascular disease treatment studies

Sept. 20, 2021

On September 22, 2021, the Centers for Medicare & Medicaid Services (CMS) will convene a panel of the Medicare Evidence Development and Coverage Advisory Committee (MEDCAC). The MEDCAC panel will examine what health outcomes should be of interest to CMS in studies for cerebrovascular disease treatment with a focus on new technologies.

Given the increased emphasis on new and innovative medical products for treating diseases that have few proven therapies, studies of cerebrovascular disease treatment technologies submitted through the Investigational Device Exemption (IDE) pathway have focused on shorter term data with greater reliance upon intermediate and surrogate outcomes.

Although such foci may help in answering Food and Drug Administration (FDA) questions on safety and efficacy, they are generally less helpful for CMS whose focus is on health outcomes associated with the technologies. As a result, there are more frequent evidence gaps with respect to the clinically meaningful health outcomes for CMS beneficiaries in assessments of these kinds of medical technologies.

The MEDCAC panel will examine the growing challenges associated with these gaps in the evidence used to support requests for CMS coverage of new and innovative cerebrovascular disease treatment technologies. By voting on specific questions, and through their discussions, MEDCAC panel members will advise CMS about the ideal health outcomes in research studies of cerebrovascular disease treatment technologies, appropriate measurement instruments and follow-up durations to help to provide clarity and transparency of IDE analyses and National Coverage Analyses (NCAs). MEDCAC panels do not make coverage determinations, but CMS benefits from their advice.

Although there is general agreement on the importance of using mortality as an outcome measure in cerebrovascular disease clinical research, there is little or no consensus on which other outcomes measures to include, such as stroke status and recurrence, hospitalization and healthcare resource utilization, clinician-reported patient functioning, as well as patient-reported outcome measures (PROMs). CMS is seeking input on whether, how, and when to include these additional outcomes.

CMS release