RD Moonshot – Research Needs Recommendations

Despite advancements, most rare diseases lack effective therapies, underscoring the necessity for a strategic overhaul in the approach to research and development. To address the multifaceted challenges in rare disease research, a unified framework that encompasses the recommendations across clinical trials, diagnostics, and translational research is proposed by the Rare Disease Moonshot. Central to this framework is the role of public-private partnerships in bridging gaps in research and development. By aligning efforts, leveraging resources, and fostering innovation, significant strides can be made towards erasing the “white spots” in rare disease research.

Read their research needs recommendations:
– one pager/factsheet: https://www.rarediseasemoonshot.eu/wp-content/uploads/2024/06/Factsheet-Recommendations-to-advancing-research-in-rare-disease-white-spots.pdf
– and in greater details:
1) About clinical trials: https://www.rarediseasemoonshot.eu/wp-content/uploads/2023/07/Clinical-Trials-Research-Needs-Recommendations-2.pdf
2) About diagnosis: https://www.rarediseasemoonshot.eu/wp-content/uploads/2023/12/DIAGNOSTIC-RESEARCH-NEEDS-RECOMMENDATIONS.pdf
3) About translational research: https://www.rarediseasemoonshot.eu/wp-content/uploads/2024/04/TRANSLATIONAL-RESEARCH-NEEDS-Recommendations.pdf