Rare disease patients’ participation in research. A Rare Barometer survey

This report presents the in-depth results of the Rare Barometer survey on rare disease patients’ participation in research. Over 3000 people living with a rare disease and their family members responded to this global Rare Barometer survey. - More than one third (37%) of rare disease patients surveyed declare they have already participated in a research study. - Among those who participated in research, 18% specified they have participated in research to develop treatments and therapies. Participation in research to develop treatments and therapies varies significantly across patients’ countries and disease area. Results also show a gender gap in inclusion in clinical studies: more men (21%) than woman (16%) have participated in research to develop treatments. - The main motivations that lead rare disease patients to participate in research are first the fact that they want to help science and the community and then to receive new treatment options. - A quality relationship with researchers is by far the main factor contributing to satisfaction with the participation in a research project. Having the possibility to be closely and regularly followed by a research team is also reported as one of the positive aspect of patients’ participation. - Regarding rare disease patients’ perspectives on rare disease research, among rare disease research areas, therapeutic research is considered as the highest priority. In parallel, the lack of public funding is identified as the main obstacle to rare disease research.