An article published by Julkowska, Austin et al., in Gene Therapy describes the role and impact of strengthened cooperation not only in research in rare diseases (RD), but also at regulatory, funding and health care levels, not only in Europe but globally.
For more than two decades, the EU has funded cross-border research in RD through its Framework Programmes for Research and Technological Development (FP) with complementary projects and actions implicating partners from wide geographical areas that include North America, Africa, Asia and Australasia. The funding of collaborative research through joint transnational calls was seen as the most effective method to enhance cooperation among scientists. For this reason, the European Research Area Network (ERA-Net) for Rare Diseases, E-Rare, was implemented in 2006. Since then, E-Rare has launched eight Joint Transnational Calls for projects, investing over €92 million in research on RD. Currently, E-Rare-3 is composed of 26 public bodies, ministries and research funding organizations from 18 countries, including a number of non-EU countries – Switzerland, Israel, Turkey, Canada and Japan.
Additionally, within the EU, the European Reference Networks (ERNs) have been set up under the 2011 Directive on Patient Rights to Cross-Border Healthcare to bring together medical specialists across Europe to tackle rare or complex diseases. Healthcare providers who are members of ERNs share their expertise via dedicated IT system, allowing the knowledge to travel instead of the patients. Research is also a key element of the ERNs, which enables joint research efforts – among others – through structured knowledge hub, support of cross-border registries, and recruitment of patients to observational and clinical studies.
More recently, the EU also initiated the International Rare Diseases Research Consortium (IRDiRC) together with the National Institutes of Health in the USA to favour cooperation and collaboration on a global scale among the many stakeholders active in RD research and to maximize the output of RD research efforts around the world. The success of this initiative depends on the integration of the activities of funding agencies, academic researchers, companies, regulatory agencies, and patient advocacy organizations.
Orphanews June 6, 2017