(For use with 21 CFR 814.102)
To provide a more expeditious review of a HUD designation request, the FDA Office of Orphan Products Development (OOPD) suggests the following be provided as part of the request.
- Two copies of the request (that is, the original and one additional copy).
- Cover letter with a statement that the applicant is requesting HUD designation for a rare disease or condition or a valid (medically plausible) subset of a disease or condition.
- Table of contents to include the following subsections:
- Name and Address of the Applicant and Contact Person
- Indication for Use of the Device
- Description of the Rare Disease
- Description of the Device and the Scientific Rationale for its use as proposed
- Population Determination
- Copies of all cited References
- Appendices (if applicable)
- Pagination/Tabs corresponding to the Table of Contents. Please be sure that the following details are contained within each subsection:
- Contact Person: Include telephone and facsimile numbers and E-mail address
- Description of Disease: Include the current standard treatments
- Description of Device and its Use: Include drawings/diagrams, etc. For the specific proposed indication and disease, explain the scientific rationale for the use of the device for the disease and why this device is needed, e.g., "it can be used in place of more invasive procedures," etc.
- Population Estimate (based on the incidence of the condition in the United States):
- If the device is proposed for an indication that represents a subset of a common disease, justify why that the subset is an orphan subset, e.g., "the device cannot be used to treat/diagnose other subsets because..."
- If the device is for a diagnostic purpose, demonstrate that not more than 8,000 patients per year would be subjected to diagnosis by the device in the United States.
- Provide the authoritative references to demonstrate the number of patients that will use this device per year. The disease must be one that affects or is manifested in fewer than 8,000 people in the United States per year.
- Note: supporting population documentation may include peer-reviewed literature citations, textbooks, specialized medical society proceedings, or governmental statistics publications (National Hospital Discharge Survey, National inpatient Profile, etc.).
When adequate studies or literature citations do not exist, OOPD will accept written statements from three independent experts in the field if they contain adequate justification for the population estimates.