Enhancing and leveraging principal investigator and patient advocacy group collaboration in rare disease clinical research-meeting report from the rare Diseases Clinical Research Network

The Rare Diseases Clinical Research Network (RDCRN) works toward faster diagnosis and better treatment for people living with rare diseases, specifically by advancing clinical trial readiness. Inclusion of patient advocacy groups (PAGs) is mandated for each RDCRN consortia; principal investigator (PI)-PAG collaboration is expected to accelerate clinical trial readiness. Real-world examples of PI-PAG collaboration in rare disease clinical research (RDCR) are often not documented nor shared. We report on the Spring 2023 RDCRN meeting, which was dedicated to (a) capturing examples of ways that PAGs and PIs in the RDCRN collaborate, and (b) describing challenges and potential best practices for PAG-PI collaboration. PI and PAG attendees included 50 investigators and staff from 19 consortia and 41 PAG members from 21 consortia. Examples of collaboration in Study Design, Planning and Execution, Funding, and Stakeholder Engagement were captured, as were best practices and challenges to PI-PAG collaboration. Strengthening PI-PAG collaboration can accelerate rare disease research. Documenting real-world examples, and barriers and facilitators of collaboration, from across the RDCRN, supports existing frameworks for accelerating clinical trial readiness.