FDA Finalized Guidance on Humanitarian Device Exemption Program

The US Food and Drug Administration (FDA) on Thursday finalized guidance on the Humanitarian Device Exemption (HDE) program, reflecting changes made by the 21st Century Cures Act and further explaining the criteria to determine “probable benefit” related to its decision-making process for humanitarian use devices (HUDs).

HUDs are medical devices intended to help treat or diagnose diseases or conditions that affect or are manifested in not more than 8,000 individuals in the US per year, a number which was increased by the Cures Act from 4,000 individuals per year.

First drafted in June 2018, the final guidance remains largely the same as the draft, although it includes some additional information and clarifications.

For instance, in the section on “Assessing Probable Benefit and Risk in an HDE Application,” the agency adds a sentence to clarify:

“The types of evidence that may be used to support approval of an HDE application include investigations using laboratory animals, investigations involving human subjects, nonclinical investigations, and analytical studies for in vitro diagnostics.”

FDA also clarifies when an HDE can be suspended or withdrawn, noting, “We intend to discuss the regulatory options with the HDE holder before revoking a HUD designation.”

Under the section on “IRB or Appropriate Local Committee Oversight,” the final guidance also adds an explanation for what such a local committee means.

“FDA interprets the statutory term ‘appropriate local committee’ to mean a standing committee for the facility that has expertise and experience in reviewing and making treatment decisions for clinical care, particularly in applying innovative medical device technologies to clinical care,” - the guidance notes.

“As such, a standing committee for the facility that includes physicians with experience in the treatment of rare diseases or conditions would be considered an appropriate local committee by the Agency. Depending on the facility and the charters of its committees, examples of an appropriate local committee may include a peer review committee, a credentialing committee, or a Quality Care Committee. We recommend that the committee include a senior executive level medical staff or faculty member (e.g., the Chief Medical Officer, Physician-in-Chief, Surgeon-in-Chief, Department Chair).”

The final guidance, like the draft, also includes information on FDA’s reviews of HDE applications, special considerations for devices marketed under an HDE and appendices related to benefit-risk assessments.

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