Advancing treatments for rare diseases requires innovative ways to measure success.
On May 18, 2026, the U.S. Food and Drug Administration and the Triangle Center of Excellence in Regulatory Science and Innovation convened a virtual public workshop, Advancing Novel Surrogate Endpoints for Rare Disease Drug Development. The workshop focused on scientific and regulatory considerations for developing surrogate endpoints for drugs and biological products intended to treat rare diseases, with emphasis on the types of mechanistic, translational, clinical, and methodological evidence that may support their use in regulatory decision-making.
The workshop was held as part of FDA’s broader efforts under the Rare Disease Endpoint Advancement Pilot Program and was intended to help meet commitments under the Prescription Drug User Fee Act VII (PDUFA VII) and requirements of the Food and Drug Omnibus Reform Act of 2022 (FDORA) to advance public discussion on endpoint development for rare diseases.
Across seven sessions, speakers and panelists explored the role of surrogate endpoints in traditional and accelerated approval, pathways for engagement with FDA on biomarker science, regulatory considerations for rare disease biologics, examples of reasonably likely surrogate endpoints, and emerging approaches including new approach methodologies, digital health technologies, artificial intelligence, and real-world data. The workshop materials below include the recording, presentation slides, discussion guide, agenda, speaker biographies, and related resources.
Meeting Materials
Final Agenda
Final Transcript
Speaker, Panelist and Moderator Biographies
Workshop Discussion Guide
Planning Committee Roster
Recording
Please note that captioning is subject to further edits. Once reviewed and approved, a downloadable transcript will be available.
Slides
- Side Deck for Session 1. Mechanistic and Translational Evidence Considerations for Supporting a Novel Surrogate Endpoint Biomarker (Pacanowski, FDA)
- Slide Deck for Session 2. Pathways for Engagement with FDA related to Biomarker Science (Sekar and Hart, FDA)
- Slide Deck for Session 3. Center for Biologics Evaluation and Research (CBER) Biologics License Application (BLA) Approval: Regulatory Considerations in Rare Diseases (Sherafat-Kazemzadeh, Elenburg and Shukla, FDA)
- Slide Deck for Session 4. A Case Study of a Reasonably Likely Surrogate Endpoint (RLSE) in Rare Disease Drug Development (Krudys, FDA)
Session 5. Developing Endpoints Using New Approach Methodologies
- Slide Deck for Talk 1. From Static Biobanks to Dynamic Patient Networks in Rare Disease Research (Kottick, AminoChain)
- Slide Deck for Talk 2. Endpoint-enabling NAMs: Rare Lung Disease Considerations (Hagood, UNC-Chapel Hill)
- Slide Deck for Talk 3. Assembling Mechanistic Evidence for Rare Disease Surrogate Endpoints: The Role of AI and NAMs (Mungall, Lawrence Berkeley National Lab)
Session 6. Use of Digital Health Technologies to Collect Endpoint Data
- Slide Deck for Talk 1. The Digital Physiome: Wearables for Early Disease Detection (Dunn, Duke University)
- Slide Deck for Talk 2. Accelerating Pediatric Rare Disease R&D with Core Set of Digital Measures (Cesnakova, Digital Medicine Society)
- Slide Deck for Talk 3. When Measurement Changes: Digital Biomarkers and the Future of Evidence in Rare Disease Trials (O'Connor, Ethomix)
Session 7. Harnessing Real-World Data to Improve Rare Disease Clinical Trials
- Slide Deck for Talk 1. Clinical Trials in Rare Disease: A Clinician's Perspective on Recruitment, Data Collection, and Identification of Relevant and Surrogate Endpoints (Chatfield, University of Colorado)
- Slide Deck for Talk 2. Using RWD to Generate Collective Patient Signatures (and how to find it) (Lawrence, Datavant)
- Slide Deck for Talk 3. Endpoints in the Wild: Finding Patients, Defining Disease, and Mining Outcomes from EHR Data (Housman, Graticule)
Slide Deck for Talk 4. From Community to Clinic: Patient-Centered Evidence Accelerates Therapeutic Development in Rare Diseases (Brimble, FOXG1 Foundation and Citizen Health)
Workshop Survey closed on May 25th.
Continuing Medical Education
** Up to 6.25 hours of Continuing Medical Education will be available.
CME Accreditation Statement
The School of Medicine of the University of North Carolina at Chapel Hill is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.
Credit Statement
The School of Medicine of the University of North Carolina at Chapel Hill designates this live activity for a maximum of 6.25 AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.
Disclosure Statement
This activity has been planned and implemented under the sole supervision of the Course Director, Christin Daniels, MA (UNC Chapel Hill), co-course director, Melissa Haendel, PhD (UNC Chapel Hill), co-course director, Quyen Tran, PharmD (CDER, U.S. FDA) and co-course director Eleanor Dixon-Terry, MPH (CBER, U.S. FDA) in association with the UNC Office of Continuing Professional Development (CPD). Christin Daniels, Quyen Tran, Eleanor Dixon-Terry and CPD staff have no relevant financial relationships with ineligible companies as defined by the ACCME. Dr. Haendel has an ownership interest in Alamya Health. This relationship has been mitigated; Dr. Haendel will recuse herself from discussing any products or services offered by Alamya. The presenters, panelists, and moderators have no relevant financial relationships with ineligible companies as defined by the ACCME. The content of the presentations relates to research process and procedure, not clinical care of patients.
Funding Acknowledgement: This program was supported by the Food and Drug Administration (FDA) of the U.S. Department of Health and Human Services (HHS) as part of a financial assistance award [Center of Excellence in Regulatory Science & Innovation], U01FD007857 totaling $11,000 from the Office of Regulatory and Emerging Sciences. The contents are those of the author(s) and do not necessarily represent the official views of, nor an endorsement, by FDA/HHS, or the U.S. Government.
