FDA Announces Changes to Humanitarian Device Exemption Regulation

As a result of the 21st Century Cures Act, the US Food and Drug Administration (FDA) announced changes in the Humanitarian Device Exemption (HDE) regulations, which provides manufacturers with regulatory relief for devices intended to diagnose and/or treat rare diseases.

Devices that qualify are exempt from the requirement to demonstrate effectiveness prior to marketing the product. For rare conditions, the clinical studies necessary to demonstrate effectiveness might be logistically or cost prohibitive, or both. Enrollment times to generate statistically valid sample sizes could delay availability of the device for many years.  In addition, the small returns based on the limited number of potential users often made investing in clinical trials economically impractical. The HDE provides a mechanism to reduce costs to market.

A device may be marketed under an HDE for one indication, and under a 510(k) or premarket approval for another. Under new HDE regulations, the incidence of the condition for which an HDE was granted will increase from 4,000 to 8,000 individuals. This change in the threshold will increase the number of eligible conditions for consideration of an HDE. This also means that the pool of potential patients, and therefore the potential return on investment, will be larger for manufacturers. 

Previously, HDE regulations required that a local Institutional Review Board (IRB) oversee the use of a device. Therefore, manufacturers required a local IRB for each potential patient. Changes to the regulations will allow the use of a non-local or central IRB for oversight. This should provide more flexibility and availability for the use of a device, and promote more consistency in how a device is used. Moreover, the oversight of a central IRB should assist in the consolidation and faster detection of safety signals. This is expected to open a new niche market for central IRBs.

The changes, announced in the 06 June 2017 Federal Register (Docket # FDA 2017-N-0011), will take effect immediately upon publication.

If you require assistance in understanding the new regulation and how it will impact your business, contact ICON at www.iconplc.com/devices for more information. 

Gordon MacFarlane, Ph.D., RAC
Senior Manager Regulatory Affairs 


  • Medical Device