Rare Disease News, Events & Reports
CDER’s ARC Program | Center for Drug Evaluation and Research
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Rare Disease News, Events & Reports by Year:
2026 | 2025 | 2024 | 2023 | 2022
In Case You Missed It: RDEA Pilot Program Workshop Materials Available
The FDA, in collaboration with Triangle CERSI, convened a Rare Disease Endpoint Advancement (RDEA) Pilot Program workshop, Advancing Novel Surrogate Endpoints for Rare Disease Drug Development, on May 18, 2026. The workshop recording and slides are available here.
Events
- Advancing Novel Surrogate Endpoints for Rare Disease Drug Development Workshop – May 18, 2026
- RISE Together: Data Sharing Across the Rare Disease Ecosystem – March 30, 2026
- Public Meeting: FDA Rare Disease Day 2026 – February 23, 2026
News
- Strategic Agenda added to FDA Rare Disease Innovation Hub – January 15, 2025
Events
- Individualized Therapies on the RISE - November 20, 2025
- Regulatory Submissions with Real-World Evidence: Successes, Challenges, and Lessons Learned – September 23, 2025
- Patient-Focused Drug Development: Workshop #2 to Discuss Methodologic and Other Challenges Related to Patient Experience Data – September 18-19, 2025
- Assessing Novel Efficacy Endpoints in Ophthalmologic Rare Disease Drug and Biologics Development – September 17, 2025
- On the RISE: Controls in Rare Disease Clinical Trials for Small and Diminishing Populations – September 3, 2025
Reports
News
- FDA Voices Blog: FDA Takes Exciting Steps Toward Establishing the Rare Disease Innovation Hub – November 11, 2024
- Editorial: 9 Things to Know About CDER’s Efforts on Rare Diseases – August 14, 2024
- FDA Voices Blog: FDA Rare Disease Innovation Hub to Enhance and Advance Outcomes for Patients – July 17, 2024
- Center for Biologics Evaluation and Research and Center for Drug Evaluation and Research initiate Support for clinical Trials Advancing Rare disease Therapeutics (START) Pilot Program – June 27, 2024
Events
- Patient-Focused Drug Development: Workshop to Discuss Methodologic and Other Challenges Related to Patient Experience Data – December 13, 2024
- Opportunities to Improve Dose-Finding and Optimization for Rare Disease Drug Development – October 29, 2024
- Advancing Rare Disease Therapies Through an FDA Rare Disease Innovation Hub – October 16, 2024
- Natural History Studies and Registries in the Development of Rare Disease Treatments – May 13, 2024
- Advancing the Use of Complex Innovative Designs in Clinical Trials: From Pilot to Practice – March 5, 2024
Reports
News
- FDA News Release: FDA Creates New Advisory Committee for Evaluation of Genetic Metabolic Disease Treatments – December 12, 2023
- CDER Statement: CDER Soliciting Comments Regarding Clinical Trial Innovation – October 17, 2023
- Editorial: CDER Continues to Advance Rare Disease Drug Development with New Efforts, Including the Accelerating Rare Disease Cures (ARC) Program – February 28, 2023
Events
- Public Meeting on Advancing the Development of Therapeutics Through Rare Disease Patient Community Engagement – December 14, 2023
- FDA CDER and CBER & Duke-Margolis Center for Health Policy | Rare Disease Endpoint Advancement Pilot Program Workshop: Novel Endpoints for Rare Disease Drug Development – June 7-8, 2023
- FDA CDER & M-CERSI Workshop | Creating a Roadmap to Quantitative Systems Pharmacology-Informed Rare Disease Drug Development – May 11, 2023
- FDA CDER & JHU CERSI Workshop | Addressing Challenges in the Design and Analysis of Rare Disease Clinical Trials: Considerations and Tools – May 2-3, 2023
Reports
News
- FDA Initiates the Lysosomal Diseases Pre-Consortium at the Critical Path Institute – September 1, 2022
Events
- FDA and Duke-Margolis Public Workshop: Translational Science in Drug Development: Surrogate Endpoints, Biomarkers, and More – May 24-25, 2022
- FDA CDER & NIH NCATS Regulatory Fitness in Rare Disease Clinical Trials Workshop – May 16-17, 2022