Public meeting: FDA Rare Disease Day 2024 (2024)

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Meeting recording

Watch the full meeting recording or for specific panels, please visit:

• Panel 1: Clinical trial foundations and future directions
• Panel 2: Navigating information – Empowering patients and providers
• Panel 3: Patient engagement at FDA
• Panel 4: FDA initiatives to advance medical product development for rare diseases
  

Summary

FDA will host Rare Disease Day, a virtual public meeting, on March 1, 2024 in global observance of Rare Disease Week. This year’s Rare Disease Day is dedicated to patients and health care professionals. Panels will discuss:

• The legal framework for approving studies and medical products at FDA
• What FDA does during review processes to approve medical products
• Decentralized clinical trials and digital health technologies
• Where to find important information and documents related to clinical trials
• Information that can be obtained from medical product labels
• Legal and ethical requirements for consent forms in clinical trials
• FDA initiatives to advance medical product development for rare diseases
• Ways for patients to engage with FDA

Agenda and materials

View the agenda (PDF, 2.39 MB) for detailed information about this event, including speaker bios. Where available, slides from each speaker are provided below, under Event Materials.

Continuing education credit

Free continuing education (CE) credit is available for physicians, physician assistants, pharmacists, pharmacy technicians, nurses, and certified public health professionals who complete the following enduring material activities on the FDA CE Portal:

FDA Rare Disease Day 2024: Clinical Trial Foundations and Future Directions

FDA Rare Disease Day 2024: Navigating Information. Empowering Patients and Providers

FDA Rare Disease Day 2024: FDA Initiatives

Public docket

Stakeholders are invited to provide their perspectives on the discussion questions through the public docket. All comments must be identified with the docket number FDA-2023-N-5211 through April 5, 2024.

Contact

Participants may submit questions in advance toOOPDOrphanEvents@fda.hhs.gov.

Office of Clinical Policy and Programs,Office of Orphan Products Development | 301-796-7634