Patient-Reported Experience Measurements From Individuals With Inherited Retinal Disorders Involved in Observational Research.

Purpose: Inherited retinal disorders (IRD) are a complex group of conditions. By developing the first patient-reported experience measurement (PREM) questionnaire tailored for individuals with IRD participating in natural history studies, we gathered information on individuals' views of their experience while they are involved in research.

Methods: Adults with IRD who (i) were enrolled in a natural history study taking place at Moorfields Eye Hospital (London, UK), (ii) had attended at least two study visits, (iii) the most recent one being less than two weeks before the questionnaire, and (iv) who were not involved in interventional research, were considered for participation.

Results: Fifty individuals completed the PREM questionnaire at a mean age of 31.1 ± 11 years old and were diagnosed at a mean age of 14 ± 9.7 years old. Most individuals rated "getting closer to receiving treatment' as their main motivation to enroll in the study, and their biggest influence was their own curiosity. Individuals were more satisfied with the care they received, and least satisfied with the efficiency of the visit. After validity and reliability assessments, the final PREM was created, with 27 questions and five sections, and Cronbach alpha coefficient between 0.316 and 0.756 in each section.

Conclusions: The PREM instrument allowed us to assess the overall satisfaction of individuals with IRD involved in research, detect possible barriers to research participation, and ways of improving our care.

Translational relevance: The final version can be included in future research and other sites worldwide, to maintain high quality standards.