This editorial, presented by the International Rare Diseases Research Consortium (IRDiRC), describes how precompetitive collaboration can address shared challenges across the rare disease research and development lifecycle, including diagnosis, clinical development, evidence generation and access to medicines.
It presents systems frameworks, shared platforms and disease-specific collaborations as approaches for developing non-proprietary solutions. Examples include the Critical Path Institute (C-Path),
Rare Care Comprehensive Centre, Rare Disease Moonshot, Together for Rare Diseases, International Rare Diseases Research Consortium (IRDiRC), ACCELERATE and the Innovative Health Initiative (IHI).
The authors identify several principles for effective collaboration: defining patient-centred objectives, bringing together complementary expertise, establishing common terminology and governance, building trust through neutral conveners, remaining flexible, and using existing tools and frameworks. The editorial calls for more sustainable collaboration structures aligned with patients’ needs to support rare disease research, diagnostics and therapeutic development.
