Using crowdsourcing to understand patients attitudes toward a clinical trial for retinitis pigmentosa requiring 4 years of participation

• Authors: Xiangrong Kong, Johns Hopkins University, Baltimore; Cory Dorfman, Emory University; Susie Trotochaud, USHER 2020 Foundation; Fahd Naufal, Johns Hopkins University, Baltimore; Carl A Latkin, Johns Hopkins University, Baltimore; Peter A Campochiaro, Johns Hopkins University, Baltimore
• Journal Title: OPHTHALMIC GENETICS
• Volume: Volume 43, Number 1
• Publisher: Taylor & Francis | 2022-02-01, Pages 36-41
• Type of Work: Article | Post-print: After Peer Review
• Publisher DOI: http://doi.org/10.1080/13816810.2021.1961282
• Subjects: Health Sciences, Epidemiology; Biology, Biostatistics
• Keywords: inherited retinal degeneration; patients’ willingness for clinical trial participation; multi-center clinical trial planning; patient compliance

Abstract

Background: Clinical trials for retinitis pigmentosa (RP) likely require long follow-ups because of the slow progression of RP. Understanding patients’ attitudes toward participation in a long trial and their acceptability of strategies aimed at promoting retention/compliance is important for assessing feasibility and resource needs and optimizing trial design. Methods: A crowdsourcing survey to adult RP patients was administered on social media in 2020 July-November. Patient enthusiasm level of study participation, acceptability of attending clinic visits every 4–5 months for 45-months, tele-visits with doctors, and of receiving text messages for medication reminders and for reporting missed dosages were surveyed. Results: Among the 1473 respondents, over 95% use email or a mobile phone and receive text messages; 1157 (79%) respondents were very/somewhat enthusiastic about participation, among them, 80.6% were “very willing” to attend clinic visits every 4–5 months for 45 months; 90.3% were “very willing” to have tele-visits; 64.7% and 77.1% were willing to receive text reminders to take medication and messages surveying missed doses, respectively. The youngest age group (18–30) (22.1%) and oldest age group (70+) (26.1%) compared to the 41–50 years age group (14%) and women (23.5%) compared to men (14.2%) were statistically significantly more likely not to report high willingness to participate in clinical visits for 45 months. Conclusions: A trial requiring 4-years of commitment is feasible although retention can be challenging. Strategies including supplementing in-clinic visits with tele-visits and frequent communications may facilitate retention. This study also demonstrates a methodology useful for planning clinical trials for chronic diseases.