In September of 2019 revisions of the Guidance for Industry and Food and Drug Administration (FDA) Staff: Humanitarian Use Device (HUD) Designations and the Guidance for Industry and FDA Staff: Humanitarian Device Exemption (HDE) Program were released. These guidelines were designed to assist the applicants in preparing their submissions of a HUD and HDE, in addition to the FDA reviewers in their evaluation and analysis. While new revisions of the guidelines were released, applicants should still be encouraged to follow the FDA regulations 21 CFR 814, Subpart H – Humanitarian Use Devices when submitting for HUD and/or HDE approval.
HUD designation is required before submitting a Humanitarian Device Exemption marketing application; however it does not guarantee approval of the HDE application. An HDE is an approval process through the FDA in which the applicant is not required to provide clinical trial results demonstrating that the device is effective for its intended purpose prior to marketing. This process was developed under Section 520(m)(1) of the FD&C Act to encourage the discovery and use of new devices in a less affected population. An HDE must meet all of the following criteria in order to be approved:
- The device will not expose patients to unreasonable or significant risk and the probable benefit outweighs the risk of injury or illness
- The device is not available to a person with the disease or condition with the HDE, and no comparable device, other than another HDE or Investigational Device Exemption (IDE), is available
- HUD designation
Before discussing the first steps of the submission and review process, it is important to know what a Humanitarian Use Device is. A Humanitarian Use Device, or HUD, is a medical device intended to benefit patients in the treatment or diagnosis of diseases or conditions that affect or are manifested in no more than 8,000 individuals in the United States per year. When determining the population estimate, it is important to note the difference between disease prevalence and disease incidence:
- Prevalence – the total number of patients with a disease or condition in the population at a given time
- Incidence – the number of new patients diagnosed with a disease or condition during a particular period, such as annually
The phrase “per year” was added in the 1996 Final Rule on HUD designations by the FDA to clarify that the requirement of the disease or condition affecting no more than 8,000 (4,000 at the time the 1996 Final Rule was released) individuals in the U.S. was determined annually, not as point prevalence.
Another thing to keep in mind when calculating the population estimate is whether the proposed HUD is intended for therapeutic or diagnostic purposes. According to the guidelines, therapeutic devices are generally defined as the number of new patients per year who are diagnosed with the disease or condition, and who would be eligible for treatment with the device while diagnostic devices are generally defined as the number of patients per year who would be subjected to diagnosis with the device, regardless of the test results.
When an applicant would like to submit a HUD request, the FDA recommends submitting the following to the Office of Orphan Products Development (OOPD):
- Cover Letter containing a statement that requests either HUD designation for a rare disease or condition, or a specifically identified orphan subset of a non-rare disease or condition
- Title, name, address, telephone number, and e-mail address of the applicant and primary contact person(s)
- Adequate description of the rare disease or condition or documentation of an orphan subset of a non-rare disease or condition
- Proposed indication(s) for use of the device
- Adequate description of the device and scientific rationale for its use as proposed
- Population estimate
- Copies of all cited references separated by tabs
- Appendices, if applicable
Once submitted, the review process can take up to 45 calendar days from the date of receipt by the OOPD. Once the OOPD determines which disease or condition the device treats or diagnosis, that the disease or condition does not affect more than 8,000 individuals in the United States per year, and the scientific rationale for the device is appropriate the approved HUD Designation Letter is granted. Once an HUD request is approved, the applicant is eligible to submit the HDE marketing application.
Do you have any experience submitting a HUD or HDE to the FDA? Are you familiar with any currently approved HUD’s?