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  1. Center for Drug Evaluation and Research | CDER

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

Investigational Drugs in Emergency Situations

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