Final Device Guidance Released!



The U.S. Food and Drug Administration (FDA) released a final guidance, “Humanitarian Device Exemption (HDE) Program.” The guidance is in alignment with requirements from the 21st Century Cures Act and amendments to the Federal Food, Drug, and Cosmetic Act by the FDA Reauthorization Act of 2017 (FDARA).



A humanitarian use device (HUD) is intended to treat or diagnose people with a rare disease or condition that affects fewer than 8000 per year. The HUD program is an alternative pathway toward device approval. After obtaining a HUD designation, the next step in the process for this approval pathway is requesting an HDE, which is the focus of the final guidance. An HDE exempts the effectiveness requirements as long as: 1) it poses no unreasonable or significant risk, 2) no comparable device is available, and 3) it is used for a rare disease or condition.

Summary of the Guidance:

  • The guidance describes steps the FDA uses to determine if an HDE application will be approved. One of these considerations is an assessment of benefit-risk, specifically the:

    • type, level, and duration of benefit,

    • likelihood the patient will benefit,

    • likelihood the patient will be harmed,

    • severity, type, and duration of harm, and

    • patient and caregiver perspectives on benefit or risk.

  • A HUD with an approved HDE is FDA approved for marketing, but it must have IRB approval and oversight prior to use, unless for emergency purposes.

  • Expected or unexpected adverse events must be reported under 21 CFR 803 in the same manner as for other medical devices.

  • Periodic reports to the FDA are required as a condition of the HDE.

  • The HDE may be sold to recuperate the costs of development and manufacture, but not for profit unless certain criteria are met within the pediatric population.

  • Labelling to identify the device as a humanitarian device and that effectiveness has not been established is required.

This guidance aligns with the FDA’s current focus on broadening and improving access to humanitarian use devices and clarifies areas that were frequent questions, including the criteria for “probable benefit” determination. It replaces the 2010 Q&A “Guidance for HDE holders.”

You may find one of our interactive eLearning courses helpful, particularly the Medical Device vs. Drug: Comparing and Contrasting course.

- The Clinical Pathways Team

Enjoy this blog? Please like, comment, and share with your contacts.