In addition, FDA could take other steps to make the HDE process worth further consideration by potential sponsors. It could offer additional guidance and assistance to make it easier for sponsors and IRBs to manage the requirement for IRB review of HDEs. As discussed earlier, recent CDRH guidance is helpful but still leaves areas of confusion and uncertainty. The agency could also clarify existing guidance on the very specific details of the process for applying for designation of a Humanitarian Use Device. For example, it could provide further guidance on the evidence needed to support claims of probable benefit and the calculation of the size of the target patient population, and it could also offer consultation to help sponsors understand what data will be responsive and justifiable.

RECOMMENDATION 7-4: FDA should take steps to reduce the burdens on potential sponsors of Humanitarian Use Devices, including

  • assigning an ombudsman to help sponsors navigate the regulatory process for these applications;

  • providing more specific guidance and technical assistance on the documentation of the size of the patient population as required for humanitarian use designations; and

  • developing better guidance (including step-by-step instructions and sample documents) for sponsors and IRBs on their roles and responsibilities related to IRB review of HDEs.

In another area, CDRH could also develop new guidance on the use of surrogate endpoints in medical device trials (see discussion in Chapter 5). As noted earlier in this chapter, the planned analysis of the clinical safety and efficacy data submitted in support of PMAs and the planned guidance document on device clinical trials could contribute useful information and perspectives in this area, even though neither activity will focus on HDEs specifically.



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