Specific impact 4: Improve healthcare systems’ capacity to take up research results

The EJP RD pipeline translated by its interlocked Pillars and transversal activities fosters effective and efficient implementation of research and innovation outcomes in the daily life of rare disease patients. The participation and strong involvement of the European Reference Networks, linking centres of expertise and professionals in different countries, will be the key element to improve the uptake of research results into health care.

The funding of socio-economic and health care/health services related projects in Pillar 1 (WP6) will facilitate a better understanding of the real-life problems and possible solutions for better uptake of research results in the general practice. In addition, the expected exchange of knowledge facilitated through the Networking schemes (WP7) and close connections between researchers, doctors and patients will also lead to an increased uptake of research results by providing the space to share diagnostic practices and guidelines, put more light on research results and innovative solutions and strengthen the collaborations by different stakeholders.

The Pillar 2 strategy is based on giving the European Reference Networks a leading role in driving the way VP is built, with the ultimate aim to accelerate the take up of research results in clinical practice for the benefit of patients. ERNs ensure the usability and the usefulness of the VP by testing it against real-life use cases, by bringing research questions and establishing, together with Pillar 2 partners, sustainable mechanisms for ensuring a virtuous cycle between care and research. In particular, ERNs will make their registries FAIR at the source in collaboration with EJP RD partners, so as to improve data sharing and re-use, under conditions, to increase health data secondary use for research purposes.

The translation from research to healthcare cannot be done without relevant education. Pillar 3 will train on how research results can be exploited in other settings like diagnosis, drug development, etc. It will also provide dedicated trainings developed by and targeted to ERNs, thus closely connecting the needs of healthcare providers and research. Close collaboration with Pillar 2 and Pillar 4 will enabled to identify emerging needs in new technologies and methodologies which will be addressed either through adapting exiting training or developing new ones.

Pillar 4 is dedicated to patient-centrism, and in so doing will ensure that all projects traversing the chasm between lab and clinic are designed for optimal patient uptake. This will be achieved by ensuring strong technical, product development, regulatory, HTA and funding support is available, so that projects are structured with patient uptake at their heart, and can benefit from follow-on funding to get them closer to the patient. Moreover, the methodological work being done to improve statistical methods for small populations will allow clinical uptake for interventions designed for rare diseases, thereby also improving ability to implement results into the clinic. The close involvement of ERNs and of patients groups is a key factor for this implementation, and these will be well integrated in Pillar 4.

Yearly updates on impact 4