Abstract 224: Strength of Evidence for Cardiovascular Devices Approved by the FDA's Humanitarian Device Exemption Pathway
Introduction: Cardiovascular devices have important benefits on morbidity and mortality in patients with heart disease. High-risk devices must show evidence of safety and effectiveness to receive Food and Drug Administration (FDA) approval. However, the FDA’s Humanitarian Device Exemption (HDE) pathway regulates devices which are intended to benefit patients by treating or diagnosing a disease or condition that affects fewer than 4,000 U.S. patients annually, and this pathway does not require evidence of effectiveness. We aimed to characterize the type and quality of evidence on which FDA HDE approval for cardiovascular devices is based.
Methods: We performed a systematic review of all cardiovascular devices that have received HDE approval from 1999-2013. We analyzed the FDA’s “Summary of Safety and Probable Benefit” for these devices and examined the studies and primary endpoints for methodological characteristics considered essential to minimize confounding and bias.
Results: Twelve cardiovascular devices have received FDA HDE approval. Eighteen studies form the basis of the clinical evidence justifying approval for these twelve devices. None of the eighteen studies used to support FDA HDE approval were randomized and none were blinded. One trial (6%) compared the study population to controls, and those controls were retrospectively chosen. Twelve studies (67%) stated the mean age of patients. Ten studies (56%) stated the sex distribution. Four studies (22%) looked for differences in device safety or efficacy by sex. Five (28%) studies clearly stated a primary endpoint, and one of the five (20%) employed surrogate measures for the primary endpoint. Three of the twelve devices (25%) were reviewed by the Circulatory System Devices Panel prior to FDA HDE approval.
Conclusions: HDE cardiovascular devices used to treat patients with heart disease are commonly approved without randomized studies or control groups. Primary endpoints are not often clearly delineated in HDE studies. As there may be limited data before approval, it is important to monitor the post-market safety and effectiveness of these high-risk devices.
Author Disclosures: R.F. Redberg: G. Consultant/Advisory Board; Modest; FDA Circulatory System Devices Panel. S.S. Dhruva: None.
- © 2014 by American Heart Association, Inc.