How does a humanitarian device exemption differ from a traditional PMA?

A humanitarian device exemption (HDE) is specifically designed to facilitate the approval of devices intended to treat or diagnose diseases or conditions that affect fewer than 8,000 individuals in the United States annually. One of the defining characteristics of an HDE is that it allows for the approval of devices with less stringent requirements concerning effectiveness data when compared to traditional premarket approval (PMA) processes.

The critical distinction lies in the fact that traditional PMA submissions necessitate robust evidence demonstrating both the safety and effectiveness of the device for the intended use. In contrast, the HDE does not require demonstration of effectiveness, though it still mandates that the device be safe for use. This regulatory pathway acknowledges the unique challenges of providing treatments for rare conditions, where traditional clinical trials may not be feasible due to limited patient populations.

Therefore, the statement that effectiveness data are not required accurately captures the primary difference between an HDE and a traditional PMA, highlighting the flexibility provided to facilitate innovation in treating rare medical conditions. This difference makes it easier for manufacturers to bring humanitarian devices to market, ensuring that patients with rare diseases have access to potentially life-saving technologies.