Rare Diseases Program
To facilitate and support the research, development, regulation and approval of drug and biologic products for the treatment of rare disorders.
- Coordinate the development of the Center for Drug Evaluation and Research (CDER) policy, procedures and training for the review and approval of treatments for rare diseases.
- Assist in outside development and maintenance of good science for rare diseases.
- Work collaboratively with external and internal rare disease stakeholders to support the development of treatments for rare disorders.
- Serve as CDER’s focal point to the rare disease drug development community on how best to interact with CDER.
Rare Diseases Information
Meetings and Workshops -Rare Diseases
Lists upcoming meetings and workshops.
Guidances: Annotated Listing for Rare Diseases Report to Congress-Improving the Prevention, Diagnosis, and Treatment of Rare and Neglected Diseases(PDF - 431KB) Where Can I Go For Help: Rare Diseases(PDF - 93KB) Contact: Rare Diseases Program
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)