European Joint Programme for Rare Diseases Meeting
Research on rare diseases is strongly supported by the European Union (EU) with close to 900 million Euros in more than 160 EU-funded projects. However, the EU finds the current landscape of research on rare diseases very fragmented and calls for stronger coordination of future research (and care) activities across Europe. Integrating rare disease research and care efforts is expected to maximize the potential of funded projects, tools and/or services that remain too fragmented, and to create a sustainable ecosystem for rare disease research allowing a virtuous circle between research, care and innovation.
In order to consolidate the transnational research on rare diseases through existing European platforms, and in the framework of the preparation of the Horizon 2020 calls for 2018-2020 (Societal Challenge 1, Health, Demographic change, and Well-being), the EU has launched a Consultative Group on the European Joint Programme (EJP) for Rare Diseases. An EJP is a Horizon 2020 co-fund action that is designed to support coordinated national research and innovation programmes.
The EU has provided ERNs the opportunity to participate in this Consultative Group to help refine and develop details of three initiatives (Pillars) of the EJP for Rare Diseases. The 3 Pillars are 1) research funding (to support rare diseases research by reinforcing and enlarging participation to transnational E-Rare calls and to develop new funding schemes), 2) organisation and sharing of information (to build a common virtual platform for coordinated access, repository and sharing of data and resources for research on rare diseases), and 3) education & capacity building (to share knowledge and know-how through training and support activities).
The work for the development of the EJP is divided among 4 Working Groups, one Working Group for Pillar 1, two for Pillar 2, and one for Pillar 3. Next, each Pillar of the EJP is organized into different Work Packages (WPs). ERN GUARD-HEART participates in Working Group 1 (Pillar 1, Research Funding), which has 4 WPs: WP 1) transnational calls; WP2) support of networking of new communities; WP3) Small scheme support/brokerage system; and WP4) monitoring of research funded activities.
ERNs are well-placed to contribute to the construction of the EJP for Rare Diseases to participate in the design of the programme of all 3 Pillars and help in specifying to end-user needs. ERNs will also be well-placed to develop implementation pilots for the information and data sharing platform. At the same time, ERN will benefit from the funding schemes, capacity building activities developed in the EJP for rare Diseases. And finally, there is mutual benefit to have a tight collaboration between the EJP and ERNs to foster rare disease research and care.
The kick-off meeting of the working groups took place on 15 June 2017 in Paris, France. On behalf of ERN GUARD-HEART, Dr. Estelle Gandjbakhch (AP-HP Pitié Salpêtrière Hospital, Paris), participated at this meeting in the working group for Pillar 1 (research funding). During this meeting, a time frame for the development of the EJP and different activities of the different working groups has been planned.