In response to recent legislative actions, the US Food and Drug Administration (FDA) issued draft guidance to clarify its regulatory review practices for humanitarian device exemptions (HDE).
The 54-page draft guidance released Tuesday sets forth the criteria—as required by the 21st Century Cures Act of 2016
—that agency staff will consider in making determinations of probable benefit-risk to health for a device reviewed via the Center for Devices and Radiological Health’s (CDRH) HDE program.
As part of such assessments, FDA says its staff will consider the target patient population and size, as well as the device’s intended use and existing treatment options.
The FDA Reauthorization Act of 2017
(FDARA) allows for either an Institutional Review Board (IRB) or an "appropriate local committee" can approve the use of a Humanitarian Use Device (HUD) to provide greater flexibility in making determinations regarding HUD use.
As established in the draft guidance, the term "appropriate local committee" is interpreted by FDA as "a standing committee for the facility that has expertise and experience in reviewing and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.” But the agency is asking stakeholders to provide comments by 13 August on the characteristics that should define this type of committee for purposes of the HDE program.
The new principles were drafted to replace a 2010 Q&A guidance
for HDE holders, IRBs and clinical investigators, to advance the development of the program, which establishes a pathway for firms’ devices intended for small patient populations.
Devices should be intended to treat or diagnose rare diseases or health conditions affecting no more than 8,000 US patients—a limit increased by the Cures Act
—to receive HUD designations under the HDE program.
Other regulatory policies outlined in the guidance document include postapproval requirements, such as those for adverse event reporting and periodic HDE reports to FDA.
Regulatory considerations HDE for designations and applications are detailed in the draft guidance as well. For example, an HDE applicant is required to first receive a HUD designation request from the Office of Orphan Products Development (OOPD) prior to an HDE application submission to FDA.
Further, at least some of the key differences between FDA’s review of HDE applications vs. the premarket approval application (PMA) program are also explained in the draft guidance.
Humanitarian Device Exemption (HDE) Program Draft Guidance for Industry and Food and Drug Administration Staff