One of RareKids-CAN’s key resource platforms focuses on patient registries and how they can support research, clinical trials, and therapy development. Their goal is to connect patients and families with research opportunities, provide physicians with better tools and resources, help researchers identify patient populations for studies and clinical trials, and support industry efforts to develop therapies that improve lives.
As part of this initiative, more than 600 rare disease registries have been contacted, with approximately 60 organizations already sharing their data dictionaries for review. The effort is focused on identifying common data elements, improving how registries work together and sharing information, and establishing templates and best practices for future rare disease registries to strengthen rare disease research worldwide.
This collaboration represents an important opportunity for the GSA Natural History Study to contribute to broader rare disease research efforts and help shape future approaches to clinical trials and treatment development for rare disease communities around the world.