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EJP RD’s alignment with the European Health Data Space ensures interoperability and supports the shared
goal of safely utilising health data for research, innovation, and policymaking.

Moreover, the partnership with the Pan-European Paediatric Clinical Trial Network “connect4children”,
the European Federation of Pharmaceutical Industries and Associations (EFPIA), and the European
Medicines Agency is pivotal in progressing toward clinical trial readiness. This is achieved through
[47]
capacity-building activities that focus on innovative methodologies . Furthermore, the collaboration with
EFPIA, the European Confederation of Pharmaceutical Entrepreneurs, and the European Association of
Bioindustries contributed to shaping the Rare Disease Moonshot. This initiative scales up public-private
partnerships to accelerate scientific discovery and drug development in rare and paediatric diseases for
[48]
which currently there is no therapeutic option .

Building on its achievements and successful collaborations, the EJP RD is set to evolve into the "European
[49]
Rare Diseases Research Alliance (ERDERA)" , encompassing over 170 public and private organizations
across more than 36 countries. This programme is secured with research funding for the next seven years,
extending into clinical trial sponsorships. At the heart of ERDERA are the European Clinical Research
Network and Acceleration Hub, dedicated to accelerating diagnostics, clinical trial readiness, innovative
therapies development, and outcome research. ERDERA will support RD research by providing essential
services such as data processing, regulatory compliance, innovative methodologies, and training support,
ensuring strategic coherence and resource efficiency on a global and national scale.

The RD research ecosystem fostered by EJP RD and expanded by ERDERA aims to significantly accelerate
diagnosis establishment and research enrolment for undiagnosed patients on average within six months of
medical presentation, contribute to the development of effective RD therapies approved in the EU and
internationally, and deepen understanding of RD's societal impact to inform policymaking. From the outset,
ERDERA will leverage EJP RD's sustainability plan and an exit strategy to ensure its long-term viability by
linking development and delivery efforts. This includes establishing a sustainable vision, operationalising
core values like data and expertise infrastructure, clinical research services, and funding support, and
incentivizing knowledge sharing and commercial engagement. The international collaboration and
information sharing fostered by EJP RD will remain key to ERDERA. Its Strategic Research and Innovation
Agenda (SRIA) already lists the pre-identified collaborations with EU programmes, missions, and other
[50]
initiatives. These collaborations will expand to other entities according to arising needs and interests .
Ultimately, ERDERA strives to form a European Federated Rare Disease Research organisation capable of
sustaining long-term public and private support, thereby transforming the landscape of RD research.

DECLARATIONS
Acknowledgements
We are deeply thankful to the entire EJP RD consortium for their unwavering commitment and significant
contributions toward the development and success of this consolidated rare disease research ecosystem.
This work stands on the collective efforts of a diverse group of experts and institutions whose dedication,
interdisciplinary collaboration, and innovative thinking have propelled forwards our understanding of rare
diseases. Their relentless pursuit of excellence, shared knowledge, and resources have been instrumental in
overcoming the challenges associated with rare disease research. We acknowledge the unique role of each
member and the synergy that has enabled us to achieve our shared goals. This achievement shows the power
of collaboration in advancing scientific discovery and improving medicine and care.