What is a key difference between a humanitarian device exemption and a traditional PMA?

The key difference between a humanitarian device exemption (HDE) and a traditional premarket approval (PMA) is that the HDE does not require the typical level of effectiveness data that is mandated for a PMA.

The HDE is specifically designed to facilitate the availability of devices intended to treat or diagnose diseases or conditions that affect fewer than 8,000 individuals in the United States per year. Due to the limited patient population and the unique nature of these devices, the FDA allows for a different approach in terms of demonstrating effectiveness. Instead of requiring substantial evidence of effectiveness, as is common with traditional PMAs, the HDE process allows manufacturers to show that the device is safe and probable benefits outweigh any risks, even if definitive effectiveness data are not available.

This approach acknowledges the need for certain medical devices that may not meet the rigorous effectiveness standards typically necessary for broader market approvals but are nonetheless important for rare conditions.