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.
Rare Diseases Information
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)