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[6]
joint conferences like the 2021 and 2023 RE(ACT) Congress & IRDiRC Conference and jointly produced
[7]
[8]
tools such as the Innovation Management Toolbox and Orphan Drug Development Guidebook .
Patient engagement is a vital component of transnational collaboration in all rare diseases research areas.
The EJP RD adopts a patient-centred approach, by supporting patient engagement and involvement in its
activities and by funding patient organizations in research projects. Moreover, EURORDIS , the patient
[9]
organizations alliance, plays a significant role in the programme. It co-leads the training and empowerment
pillar , oversees training activities like the one on leadership and communication skills for patient
[10]
[10]
[10]
representatives , co-develops EJP RD’s Massive Open Online Courses , and is involved in the governance
structures.
The connection with national stakeholders is essential for aligning European and national research policies
and activities. The EJP RD facilitates this through the establishment of National Mirror Groups (NMGs) in
[11]
participating countries . These groups bring together expertise and knowledge from the RD community in
a specific country by joining national/regional RD programmes, infrastructures, and centres of expertise.
They facilitate the identification of national needs and policy gaps, advocate for reforms conducive to the
EJP RD’s success, and promote national alignment with the European research strategy. To date, four
NMGs have been established in the Netherlands, France, Portugal, and the UK, with an additional ten
expected to benefit member countries by the end of the EJP RD.

EJP RD ACTIVITIES AND ACHIEVEMENTS
Research funding
EJP RD Joint Transnational Calls stem from the three successive ERANETs E-Rare: the first one launched
in 2006, the second (E-Rare-2) in 2010, and the third in 2014 (E-Rare-3, 2014-2019, H2020), with the major
goals being to foster the systematic exchange of information and build a transnational research funding
programme on rare diseases. EJP RD has maintained a steady focus of its predecessors on multinational RD
funding activities. Taken together, from 2006, 207 projects were funded through 15 Joint Transnational
Calls (JTCs) , allocating a budget exceeding 223 million euros. The European Commission (EC) co-funded
[12]
selected research projects in 2015, 2019, and 2020.

The funders within EJP RD allocated EC co-funding to support Patient Advocacy Organisations (PAO) in
2019, 2020, and 2021, resulting in the funding of 44 PAOs - the following JTCs being funded only through
national funding, no central EC budget was available to continue this support, but many national funders
strived to provide some alternatives. Concurrently, collaborations among patients, funders, and researchers
led to the development of a guide on patient partnership in rare diseases research . This initiative was
[13]
successful, with 100% of the projects funded in the JTC-2021 involving at least one patient organisation. In
parallel, EJP RD has laid a strong foundation for patient-centeredness in RD research training activities. A
[14]
total of 425 patients or patient representatives attended specialised workshops . This engagement also
[15]
contributed to the increased participation of patient representatives in EJP RD JTCs and NSS calls . It also
has a possible longer-term impact on the implication of 150 patients in protocol development at the
European Medicines Agency (EMA).

To further strengthen connections within the RD community, EJP RD introduced a new funding model for
networking events, the Networking Support Scheme (NSS) . Since 2020, this scheme has supported 69
[15]
events conducted in-person, online, or in a hybrid format following the COVID-19 crisis. Significantly, 22
(32%) of these networking events were coordinated by PAOs, and 50 (72%) included participants from
under-represented countries (8 being the coordinator of the Networking event), highlighting the scheme’s