Rare Disease Moonshot: Clinical trials 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 

The Clinical Trials Research Needs Recommendations aim to inform funders of research programmes at national, European and international levels, as well as health research players from public and private sectors who plan projects in the RD white spots areas.  

We believe that the deployment of these recommendations will help prioritising disruptive and impactful actions to close the gap between academic knowledge generation and next development steps, address the asymmetry of knowledge and skills, and develop regulatory science, amongst others.