Value-Based Hypothesis Testing for Cardiac Device Clinical Trials: A Pathway for Accelerated Reimbursement Decisions.

Abstract

Medical device sponsors seeking to introduce innovative devices in the United States must clear 2 regulatory hurdles. First, they must provide evidence demonstrating reasonable assurance of safety and effectiveness for adjudication and marketing approval by the Food and Drug Administration (FDA). Second, they must apply for reimbursement coverage through the Centers for Medicare and Medicaid Services (CMS). This second step is based on a different metric that requires demonstration that treatment using the device is reasonable and necessary. Typically, decisions for reimbursement coverage lag well behind the FDA approval for marketing and may result in substantial delays to the availability of some novel therapies. In interventional cardiology, recent examples include a 6-month wait for a coverage with evidence decision after an already-belated FDA approval for the first transcatheter aortic valve and the only recently final reimbursement decision for the Watchman (Boston Scientific, Marlborough, MA) left atrial occlusion device, almost 11 months after FDA-marketing approval. These delays arise in part from the incomplete overlap between safety/effectiveness and reasonable/necessary standards. Pivotal clinical trials are typically designed to support the safety and effectiveness assessment but may lack data necessary to characterize reasonable and necessary. Although reasonable and necessary does not have a strict regulatory definition, it connotes added healthcare value, defined as improved quality of outcome relative to cost.1,2 Harmonizing these goals in clinical trial designs will accelerate the clinical introduction of important new technology. This study will examine the limitations of current cardiac device clinical trials and explore methods for incorporating value-based hypotheses into the design of these studies. Given the frequent requirement for active treatment controls and the difficulty in proving superiority of incremental changes in new devices, noninferiority trial designs have become the mainstay of cardiac device clinical trials. In addition to demonstrating that a new device …