Rare Diseases Program
Mission Statement:
To facilitate, support and accelerate the development of drug and biologic products for the benefit of patients with rare disorders.
Overview:
- 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
- Where can I go for help?
- Contact: Rare Diseases Team
- From our perspective: Encouraging drug development for rare diseases
Investigational Drugs in Emergency Situations
- For Physicians: How to Request Single Patient Expanded Access (“Compassionate Use”)
- Physician's Checklist for an IND Application for Emergency Treatment
- Emergency IND Application Timeline