The Camargo Blog has published a four-part blog series highlighting those designation programs available specifically for products with rare disease indications. This final installment will explain the criteria, timeline, and benefits of Humanitarian Use Device (HUD) designation.
The HUD designation program is designed for medical devices and is similar to the Orphan Drug Designation (ODD) program for drugs and biological products. The HUD designation provides an incentive for sponsors developing medical devices to be used in the treatment or diagnosis of diseases affecting small populations. Established in 1990 with the passage of the Safe Medical Devices Act, the program creates an alternative pathway for market approval of medical devices that may help people with rare diseases or conditions. It was updated as part of the 21st Century Cures Act and is codified in 21 CFR §814 Subpart H.
Criteria to Obtain the Designation
To obtain HUD classification, a device must meet several criteria:
- It must be intended to benefit patients in the treatment or diagnosis of a rare disease or condition that affects or is manifested in 8,000 or fewer people in the US per year or is a rare subset of a non-rare disease or condition.
- Adequate documentation or prevalence data demonstrating that the intended disease or condition is rare must be provided.
- There must be scientific rationale supporting use of the device in the rare disease or condition.
According to the FDA guidance document on HUD:
“The scientific rationale supporting use of the device for the rare disease or condition, or orphan subset of a non-rare disease or condition (see Section III.C) in the HUD request should contain all relevant nonclinical, clinical, and/or proof-of-principle data pertaining to the device as applicable – whether positive, negative, or inconclusive. It should be noted that the nonclinical information on whether a device has been verified and validated against the proposed device design specifications is reviewed in the HDE marketing application and not in the HUD request.”
Criteria to Obtain the Benefit
After obtaining HUD designation for a medical device, a sponsor can apply for a Humanitarian Device Exemption (HDE), a type of marketing application. The FDA may grant an HDE if the device meets the following criteria:
- The device will not expose patients to an unreasonable or significant risk of illness or injury.
- The probable benefit to health from use of the device outweighs the risk of injury or illness from its use, when considered with the probable risks and benefits of currently available devices or alternative forms of treatment.
- The device would not be available to a person with the disease or condition in question without the HDE.
- No comparable device, other than another device approved under an HDE or Investigational Device Exemption (IDE), is available to treat or diagnose the disease or condition.
- The device is designed to treat or diagnose a disease or condition that affects not more than 8,000 individuals in the US on an annual basis.
Timeline and Maintenance
A request for HUD is submitted to the Office of Orphan Products Development (OOPD) and can be submitted at any time prior to submission of an HDE or other device marketing application. The FDA should respond to the request within 45 days.
There are no apparent requirements for maintaining HUD prior to submission of an HDE or other device marketing application. The sponsor of an approved HDE application is subject to post-approval requirements (such as limitations regarding selling the device for profit) and must submit periodic reports in accordance with the approval order. In addition, the HDE holder is responsible for ensuring that the HUD is administered only in facilities having oversight by an Institutional Review Board (IRB).
Once a medical device is designated as an HUD, the device is eligible for an HDE marketing application. An HDE application is similar in both form and content to a Premarket Approval application (PMA) but is exempt from PMA effectiveness requirements (described in Sections 514 and 515 of the FD&C Act). FDA approval of an HDE application authorizes the sponsor to market the device in accordance with the approved labeling and indication(s) for use.*
*Note: An HUD designation and the subsequent submission of an HDE application do not guarantee FDA approval.
Camargo has a long history of helping sponsors to attain special designations like HUD status and to map the most time- and cost-efficient development plan for regulatory and post-marketing success. Contact us to find out how we can support your program.
Marissa Berry, PhD, RAC
Manager, Regulatory Strategy