Translational research needs recommendations
The Rare Diseases (RD) Moonshot was set up to boost public private collaborations in the areas where there are with no treatment options and there is no R&D going on.
The RD Moonshot partners identified three areas of action where public private collaborations can add most value:
- Optimize the translational research ecosystem to accelerate translation,
- Modernize clinical trials (design, conduct, regulatory sciences) to make them more suitable for very small populations,
- Support infrastructure to shorten the path to diagnosis and treatment
Accelerating translational research for rare diseases white spots by bringing public and private partners together earlier, better, and more often to achieving a common goal in order to bridge the gap between scientific discovery and clinical application. White spots are known to be conditions for which there is no approved treatment option and where development is not currently commercially viable. This is the reason why this paper provides recommendations for a robust collaborative framework involving public-private initiatives to leverage the complementary strengths of various stakeholders, including patients, researchers, clinicians, regulatory authorities, industry and Health Technology Assessment (HTA) bodies. Recommendations for overcoming the barriers to successful translational research include developing a shared vision focused on patient-centric approaches, while ensuring structural access to comprehensive expertise across the value chain and investing in innovative methodologies alongside standardization and awareness efforts and a call for the acceleration of translational efforts through the establishment of cross-sectoral accelerators and the enhancement of Technology Transfer Offices to facilitate effective technology transfer and innovation processes.
