To facilitate, support and accelerate the development of drug and biologic products for the benefit of patients with rare disorders.
- Coordinate the development of CDER policy, procedures and training for the review of treatments for rare diseases.
- Assist in outside development and maintenance of good science as the basis for the development of treatments for rare diseases.
- Work collaboratively with external and internal rare disease stakeholders to promote the development of treatments for rare disorders.
- Maintain collaborative relationships with CDER's review divisions to promote consistency and innovation in the review of treatments for rare disorders.
- Work collaboratively with international regulatory agencies to discuss and exchange scientific and regulatory information related to rare diseases.
Rare Diseases Information
Guidances: Annotated Listing for Rare Diseases
- Report: Complex Issues in Developing Drugs and Biological Products for Rare Diseases and Accelerating the Development of Therapies for Pediatric Rare Diseases (PDF - 1.5MB)
- Where can I go for help?
Contact: Rare Diseases Program From our perspective: Encouraging drug development for rare diseases
Investigational Drugs in Emergency Situations
IND Applications for Clinical Treatment: Treatment of a Single Patient in Emergency Setting Physician's Checklist for an IND Application for Emergency Treatment(PDF - 102KB) Emergency IND Application Timeline(PDF - 30KB) Emergency New Drug Application Eligibility Tool(PDF - 45KB) Emergency Investigational New Drug Application Eligibility Tool (text version)