Recommendations
The Rare Disease Moonshot partners identified three areas of action where public private collaborations can add most value:
CLINICAL TRIALS RESEARCH NEEDS RECOMMENDATIONS
How can public-private partnerships help optimise clinical trials in rare disease?
DIAGNOSTICS RESEARCH NEEDS RECOMMENDATIONS
How can public-private partnerships help optimise diagnostic research in rare disease?
TRANSLATIONAL RESEARCH NEEDS RECOMMENDATIONS
How can public-private partnerships help optimise translational research in rare disease?
How were the recommendations co-created?
The publication of research needs recommendations for clinical trials, diagnosis, and translational research is the result of several months of dedicated collaboration among members of the rare disease community. The stakeholders consulted during the drafting process included patient representatives; global pharma/biotech companies; small innovative enterprises; academic translational research; biobanking, non-profit clinical and fundamental research communities; biobanking community; and non-profit PPPs. This approach highlighted our collective commitment to advancing research outcomes and addressing the challenges of rare diseases.
Clinical Trials
The clinical trials recommendations process started in February 2023, led by C-Path and ECRIN. It brought together 33 stakeholders from industry, patient advocacy groups, and regulatory bodies. Through a series of workshops and consultations, the discussions focused on essential aspects such as disease prevalence, patient access, data standards, and regulatory support.
Diagnosis
The diagnosis recommendations journey kicked off in June 2023 with a workshop that brought together 30 industry participants. The final recommendations came from months of teamwork between the RD Moonshot team and stakeholders, including industry, research community and patient advocacy groups. Additional input came through a series of workshops with industry partners and at the EURORDIS Round Table of Companies, which gathered more than 100 participants, the recommendations were continually enhanced.
Translational Research
The work on translational research recommendations kicked off with a workshop in February 2023, bringing together 33 participants from a broad spectrum of sectors. The initial discussions underscored the importance of interdisciplinary approaches, research coordination, and stakeholder skill enhancement. Throughout the process, workshops, online consultations, and direct feedback sessions, involving over 20 stakeholders, helped to refine the recommendations.