In the US, manufacturers of high risk devices must submit data to the Food and Drug Administration (FDA) to demonstrate safety and effectiveness before the devices can be approved for wide use. The humanitarian device exemption (HDE) was established by the Safe Medical Devices Act of 1990 to encourage device manufacturers to develop products for treating or diagnosing rare diseases. Under the exemption, manufacturers do not have to provide the high quality data on effectiveness that would normally be expected for high risk devices, but they must still prove that the device is safe and that the probable benefits outweigh the risks from its use and that of alternative forms of treatment.

Policymakers in the US and Europe continue to examine and update the regulation of medical devices. Current EU proposals to require manufacturers of high risk devices to submit a clinical investigation report and publish a summary are similar to the requirements for HDE devices in the US. However, the EU proposals apply to all high risk devices not just those for rare diseases. We examine the scientific and regulatory characteristics of devices approved under the humanitarian device exemption since its inception and consider the benefits and risks of this pathway.

(cite: Hwang TJ, Carpenter D, Kesselheim AS. Assessment of US pathway for approving medical devices for rare conditions. British Medical Journal (BMJ) 2014; 348: g217.)