About FDA
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Rare Diseases Program
Mission Statement:
To facilitate and support the research, development, regulation and approval of drug and biologic products for the treatment of rare disorders.
Overview:
- 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.
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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
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Investigational Drugs in Emergency Situations
Investigational New Drug Applications for Clinical Treatment with Investigational Drugs in Emergency Situations 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 New Drug Application Eligibility Tool (text version)
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