The Rare Disease Cures Accelerator-Data and Analytics Platform initiative (RDCA-DAP®) hosted an all-day workshop and annual meeting September 13 and 14 in Washington. The in-person and livestream event for rare disease stakeholders featured expert presentations, panel discussions, platform demonstrations, and was attended by more than 200 patients, researchers, clinicians, biopharmaceutical company representatives, regulatory reviewers, and scientists.
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RDCA-DAP was established through a partnership between Critical Path Institute (C-Path), National Organization for Rare Disorders (NORD®) and the U.S. Food and Drug Administration (FDA) and has grown to include dozens of collaborations between a variety of stakeholders throughout the rare disease community. The platform serves as a centralized and standardized infrastructure to host integrated and shared data and to support and accelerate rare disease medical product development.
The workshop’s keynote address was delivered by Theresa Mullin, Ph.D., Associate Director for Strategic Initiatives, FDA, Center for Drug Evaluation and Research (CDER). “Building this platform was about better informing drug development programs to get drugs that are effective and safe to patients with rare diseases as soon as possible and as efficiently as possible,” Mullin said. “It’s taken a multistakeholder effort and now we’re seeing tools and learnings that can be shared across rare diseases.”
The keynote was followed by a panel discussion on the impact of RDCA-DAP, which featured representatives from each organization engaged in this effort: Klaus Romero, M.D., M.S. F.C.P. and Alexandre Bétourné, Ph.D., Pharm.D., from C-Path; Pamela Gavin, MBA, from NORD; and Theresa Mullin, Michelle Campbell, Ph.D., and Billy Dunn, M.D., from FDA.
Additional key speakers and panelists were featured from industry, academia and patient organizations. The robust agenda included case studies shared by users of the platform, sessions covering critical topics on the impact of RDCA-DAP on accelerating drug development for rare diseases, as well as active discussions around data sharing, privacy and standards, and much more. A significant portion of the agenda featured case studies on the use of the platform to further clinical research in sickle cell disease, Duchenne muscular dystrophy, Friedreich’s ataxia and transplant therapeutics.