OBJECTIVES: Despite an increasing recognition of the importance of patient involvement in clinical research, regulation and policy matters, many patients are not well informed in these areas to be able to actively participate. This study evaluated the short-term impact of a training course designed to educate patients and advocates in the field of rare retinal degenerative diseases to enable them to become informed participants.

METHODS: A semi-structured questionnaire was distributed to the course participants post-completion. The questionnaire included items regarding the usefulness of the course, enablers and barriers to utilizing the knowledge in their future work, pre- and post-course knowledge levels, and confidence in discussing the subject matter in decision making panels.

RESULTS: The survey was completed by 75 % of participants who completed the course (9/12). Mean age of the respondents was 44±9 years and 75% were females. Majority responded that they would use the knowledge gained from the course to provide interventions in the organizations they work with (75%) and increase advocacy activities (75%). Many felt inspired to do more research on discussed topics (55%) and believed the course allowed them to be more effective in discussing the subject matter on panels/committees they sit on (75%). There was a significant increase in participants reporting high subject matter knowledge (10% vs 55%, p<0.001) and increased confidence (55% no/slightly confident vs 22% slightly confident, p <0.001) in participating in multidisciplinary panel discussions on the subject after the course.

CONCLUSIONS: In the short-term, the training course had a positive impact, with participants becoming more informed and confident in the subject matter. This study highlights the potential of targeted training programs to empower patients and advocates in research, regulation and policy, particularly in rare diseases.