www.hhs.gov U.S. Department of Health and Human Services

Rare Diseases - Challenges and Progress in Drug Development

Step Status
Educational Content
Starts On: Wed, 6/14/23: 12:00 AM EDT
Ends On: Sun, 6/14/26: 11:45 PM EDT
Type: Enduring Material
Credits: 1
Description: The development of drugs to treat rare diseases is challenging on multiple levels. This online learning will examine these challenges and FDA programs to drive scientific and regulatory innovation and engagement to accelerate the availability of treatments for patients with rare diseases.
  • Describe the challenges involved in developing a drug for a rare disease.
  • Identify key aspects of the FDA regulatory framework that are relevant to rare disease drug development.
  • Summarize the efforts of the FDA Center for Drug Evaluation and Research to accelerate rare disease drug development through innovation and engagement.
Additional Information: Review the Activity Announcement for additional details: Announcement Form

Additional Resources: SLIDES
ACCME Joint Accreditation
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