Description
This guidance provides recommendations to industry, researchers, patient groups, and other stakeholders (collectively referred to in this guidance as “stakeholders”) interested in requesting a meeting, including a teleconference, with the Food and Drug Administration’s (FDA’s) Office of Orphan Products Development (OOPD) on issues related to orphan-drug designation requests, humanitarian use device (HUD) designation requests, rare pediatric disease designation requests, funding opportunities through the Orphan Products Grants Program and the Pediatric Device Consortia Grants Program, and orphan product1 patient-related topics of concern. It is also intended to assist OOPD staff in addressing such meeting requests. This guidance describes procedures for requesting, preparing, scheduling, conducting, and documenting such meetings.
Scope & Applicability
Product Classes
1Includes drugs, devices, biological products, or medical foods for rare diseases.
Stakeholders
5Entity submitting development data and knowledge; Entity performing the work process for change
Entity responsible for submitting applications under section 524B
Stakeholders interested in requesting meetings with OOPD.
Researchers are responsible for aligning QC and QA procedures with data holders.
Guidance for Industry
Regulatory Context
Attributes
4Timeframe to submit written IND after emergency authorization
Valid orphan subset for pediatric subpopulations
A metric used in calculating population estimates for designations.
Estimated number of people with a disease, used to determine clinical investigation enrollment.
See Also (8)
- Submission and Resolution of Formal Disputes Regarding the Timeliness of Premarket Review of a Combination Product: Guidance for Industry and FDA Staff (Status: Final)
- Clinical Development Programs for Drugs, Devices, and Biological Products for the Treatment of Rheumatoid Arthritis (RA) (Status: Final)
- Prussian Blue Drug Products — Submitting a New Drug Application (Status: Final)
- Calcium DTPA and Zinc DTPA Drug Products-Submitting a New Drug Application (Status: Final)
- Interpreting Sameness of Monoclonal Antibody Products Under the Orphan Drug Regulations (Status: Final)
- Tropical Disease Priority Review Vouchers (Status: Final)
- Rare Pediatric Disease Priority Review Vouchers: Draft Guidance for Industry (Status: Draft)
- Questions and Answers on Biosimilar Development and the BPCI Act Guidance for Industry: Guidance for Industry (Status: Final)