Rare disease patients’ participation in research - survey findings

The findings from a EURORDIS Rare Barometer survey of 3213 people living with a rare disease (from 63 countries) aimed to discover more about about their level of participation in research as well as what participants felt were the main obstacles to rare disease research.

Rare disease research faces many challenges as, for example, only small populations are available for clinical trials combined with poor knowledge of the causes and progression of the diseases. With a lack of co-ordinated resources for patient registries and clinical trials, funds are often scarce for research on conditions with small numbers. 

However patient involvement in research is now better targeted to the needs of patients where patients are not just beneficiaries of the research but are valued partners throughout the research process.

Some of the findings showed that:

• 37% had participated in research e.g. clinical trials, quality of life studies, research projects on genetic therapies or medical devices.
• Their motivations for getting involved was: to help the community (53%), to help science (38%), to get new treatment options (31%) and to raise awareness and knowledge (17%).
• Their experiences of being involved showed that:
• A quality relationship with researchers is essential.
• Transparent information on the research project is necessary requirement.
• Therapeutic research is the priority research area for rare disease patients.
• Lack of public funding is the main obstacle to rare disease research.

Read the report: Rare disease patients’ participation in research: A Rare Barometer survey; Eurordis, February 2018