The Rare Disease (RD) Moonshot steering group has just published its recommendations for research funders and health research players from the industry and public sector on research needs that can be best addressed by public-private collaborations to optimise clinical trials in small populations.
The 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. Modernising clinical trials (design, conduct, regulatory sciences) to make them suitable for very small populations was identified by the RD Moonshot partners as one of the three areas where public private collaborations can add most value.
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.
The deployment of these recommendations will help prioritising disruptive and impactful actions to close the gap between academic knowledge generation and the next development steps, address the asymmetry of knowledge and skills, and develop regulatory science, amongst others.