Earlier this month, the FDA released programmatic guidance intended to clarify the current review practices for the Humanitarian Device Exemption (“HDE”) Program (“Guidance”) reflecting recent changes in the HDE Program resulting from statutory amendments made by the 21^st Century Cures. The Guidance addresses frequently asked questions about FDA actions on HDE applications, post-approval requirements, and includes a filing checklist to clarify the required information for the FDA to consider whether an HDE application is ready for substantive review.

Unlike premarket applications (“PMAs”) or De Novo Submissions, an HDE application is not required to include data that demonstrate the subject device has a reasonable assurance of effectiveness – instead the review standard requires the applicant show a probable benefit that exceeds the risk of using the device. An applicant must also demonstrate (among other things) that no similar device exists and that there is no other way to bring the device to market, and that the device would be used diagnose or treat rare diseases or conditions affecting less than 8,000 individuals in the United States per year (up from the longstanding limit of 4,000 that existed prior to passage of 21^st Century Cures at the end of 2016).

The Guidance has not changed much since the June 2018 draft guidance. However, there are a few additional clarifications. For instance, the Guidance acknowledges “that investigations using laboratory animals or human subjects, as well as nonclinical investigations and analytical studies for in vitro diagnostics,” not just human subject testing may be used as evidence to support approval of an HDE application. The FDA also clarified the conditions under which an HDE can be suspended or withdrawn.

Overall, the FDA Guidance may be helpful in preparing HDE submissions, and may acknowledge additional flexibility with respect to HDE data requirements.  However, the fundamental limitations with the HDE pathway, such as limitation to very small populations and extensive post-approval requirements, remain unchanged.