Author Affiliations: Department of Bioethics, Clinical Center, National Institutes of Health, Bethesda, Maryland.
A recent randomized controlled trial (RCT) evaluating a self-expanding intravascular stent (the Wingspan stent system), a device intended to open stenotic intracranial arteries via angioplasty and thereby prevent thrombotic stroke, reported surprising results.1 Based on case series data, this type of stent system was expected to be better than usual care; however, patients who underwent the stent procedure were more than twice as likely to experience stroke or death within 30 days compared with patients treated with aggressive medical therapy (14.7% vs 5.8%).1 These “negative” test results were disappointing. A promising new approach to a serious medical problem turned out to be worse than current best practice. However, within this failure was an important lesson worthy of notice and support from patients, clinicians, and policy makers. Through cooperation between the US Food and Drug Administration (FDA) and the Centers for Medicare & Medicaid Services (CMS), this trial represented the successful use of a policy mechanism that can balance the goal of rapid access to innovative medical procedures with the need to obtain rigorous evidence on the risks and benefits of these procedures before they become widespread in clinical practice. The key was to make initial coverage conditional on participation in clinical research.
Miller FG, Pearson SD. Linking Insurance Coverage for Innovative Invasive Procedures With Participation in Clinical Research. JAMA. 2011;306(18):2024-2025. doi:10.1001/jama.2011.1631