The RealiseD project, funded under the Innovative Health Initiative (IHI), emerged in direct response to the strategic priorities set by the Rare Disease Moonshot. The Moonshot’s Clinical Trials Research Needs Recommendations provided a structured foundation that helped shaping RealiseD’s objectives, methodologies, direction and expected outcomes.
The origins of RealiseD can indeed be traced back to the EFPIA-EURORDIS Joint Statement on Patient Access to Medicines for Rare Diseases released in June 2022. Proposal 6 of this statement called for a Rare Disease Moonshot, recognising the need for a coordinated and ambitious approach to tackling persistent gaps in rare disease research and drug development. This call to action led to the establishment of the Rare Disease Moonshot launched in December 2022 at the European Health Summit, and which in turn produced research needs recommendations, particularly to optimise clinical trials. These recommendations directly informed the design and implementation of RealiseD.
“The Rare Disease Moonshot made it clear that we needed more than just recognition of the problem—we needed structured solutions that could be implemented at scale. RealiseD was conceived as a direct response to that need, building on the recommendations to create practical frameworks for optimising clinical trials and regulatory pathways in rare diseases.” – Solange Corriol-Rohou (AstraZeneca and RealiseD lead)
A key feature of RealiseD is the development of playbooks—structured guides aimed at improving the design and execution of clinical trials for rare diseases. It is also planned to identify solutions to address scientific gaps and technical and operational challenges, and find synergies with relevant existing initiatives (e.g., ERDERA, ERAMET or IDEAL) to establish a new, dedicated, rare-disease-specific and sustainable infrastructure. These playbooks, shaped by the RD Moonshot’s research recommendations, will provide standardised methodologies for researchers and industry stakeholders. The project also prioritises regulatory-grade data integration, responding to the RD Moonshot’s emphasis on embedding regulatory science in trial design to facilitate approval processes and patient access. Furthermore, RealiseD embraces the Moonshot’s model of cross-sector collaboration, bringing together academic institutions and industry leaders under the IHI framework to strengthen translational research and clinical implementation. The public-private spirit of this project is also embodied with the in-kind contribution from the industry matching the public EU funding (with a total budget over 17M€).
This model should not be the future. The needs are for today.
The RealiseD project demonstrates the tangible impact of the Rare Disease Moonshot. By transforming research recommendations into funded initiatives, it highlights how strategic collaboration, regulatory integration, and innovative trial design can help overcome the challenges of rare disease research.
We call on all research funders, public, private, or charities, to prioritise funding opportunities that centre on public-private partnerships and push beyond existing frameworks (such as the IHI). These partnerships have consistently proven their effectiveness and impact. Given the urgent need for innovation in rare disease research and the implementation of its outcomes, it is crucial to utilise these mechanisms. The evidence is clear: strategic multistakeholder collaboration accelerates progress and delivers tangible benefits to patients.
More information: https://realised-ihi.eu
Factsheet RD Moonshot: https://www.rarediseasemoonshot.eu/wp-content/uploads/2024/06/Factsheet-Recommendations-to-advancing-research-in-rare-disease-white-spots.pdf
RD Moonshot Clinical Trials Recommendations: https://www.rarediseasemoonshot.eu/wp-content/uploads/2023/07/Clinical-Trials-Research-Needs-Recommendations-2.pdf
EURORDIS Statement: https://download2.eurordis.org/positionpapers/EFPIA_Eurordis_Statement_FINAL.pdf
Factsheet RealiseD: https://www.ihi.europa.eu/projects-results/project-factsheets/realised