During this public health emergency associated with the COVID-19 pandemic, the Office of Orphan Products Development (OOPD) is providing sponsors with increased flexibility for submission of orphan drug designation requests and related submissions (amendments, annual reports, etc.). During this public health emergency, orphan drug designation, humanitarian use device designation, and rare pediatric disease designation requests and submissions may be submitted electronically by email to the OOPD.
When transmitting information to the Orphan Drug Designation Program via email, please utilize the mailbox email@example.com. The use of automated read receipt is recommended to avoid the need to call to verify receipt of the email. Sponsors and others who plan to email information to FDA that is considered to be private, sensitive, proprietary, or commercial confidential are strongly encouraged to send it from an FDA secured email address so the transmission is encrypted. The OOPD will assume that the addresses of emails received or email addresses provided as a point of contact are FDA secure when responding to those email addresses.
Sponsors and others can establish a secure email address link to FDA by sending a request to SecureEmail@fda.hhs.gov. There may be a fee to a commercial enterprise for establishing a digital certificate as part of the set-up process before emails can be sent to FDA encrypted.
On December 13, 2016, Section 3052 of the 21st Century Cures Act (Pub. L. No. 114-255) changed the population estimate required to qualify for Humanitarian Use Device (HUD) designation from "fewer than 4,000" to "not more than 8,000." Accordingly, a HUD is now defined as a medical device intended to benefit patients in the treatment or diagnosis of a disease or condition that affects or is manifested in not more than 8,000 individuals in the United States per year. The full text of the 21st Century Cures Act is available at:https://www.congress.gov/114/bills/hr34/BILLS-114hr34eah.pdf.
The Humanitarian Use Device or HUD program was established in 1990 with passage of the Safe Medical Devices Act and creates an alternative pathway for getting market approval for medical devices that may help people with rare diseases or conditions. As defined by 21 CFR 814.3(n), and updated by the 21st Century Cures Act, a HUD is a “medical device intended to benefit patients in the treatment or diagnosis of a disease or condition that affects or is manifested in not more than 8,000 individuals in the United States per year.” In order to obtain HUD designation, the applicant must provide documentation, with appended authoritative references, to demonstrate that the device meets this definition.
In addition to the documentation describing the disease or condition, the applicant must also provide the proposed indications for use of the device, and the reasons why such a device is needed for the patient population. One aspect that has become increasingly difficult is if the HUD is proposed for an indication that represents a subset of a common disease or condition. In these situations, the applicant must demonstrate that the subset is an “orphan subset.”
An “orphan subset” is a regulatory phrase used to describe the subset of individuals with a non-rare disease or condition on whom use of a device is appropriate, where use of the device on the remaining individuals with that disease or condition would be inappropriate given some intrinsic feature of the device (e.g., adverse event profile or mode of action). An “orphan subset” is not a readily identifiable subset or a group of patients who meet or do not meet the inclusion and exclusion criteria for a clinical study. Likewise, they are not patients with an unmet medical need. If the HUD application is designated, the applicant can then submit the HDE marketing application to the Center for Devices and Radiological Health (CDRH) or Center for Biologics Evaluation and Research (CBER) for marketing review.
Additional Humanitarian Use Device Resources
- Understanding HUDs and HDEs
- Humanitarian Device Exemption (HDE) Program Guidance
- Device Advice: Comprehensive Regulatory Assistance
- Medical Device User Fee
- Listing of CDRH Humanitarian Device Exemptions
- Pediatric Humanitarian Device Exemptions Reviewed by the FDA Pediatric Advisory Committee
- IDE Early/Expanded Access
Request for Humanitarian Use Device designation should be sent to:
The Office of Orphan Products Development (OOPD)
Food and Drug Administration
10903 New Hampshire Avenue
Silver Spring, MD 20993-0002