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

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.

KEYWORDS:

• Inherited retinal degeneration
• patients’ willingness for clinical trial participation
• multi-center clinical trial planning
• patient compliance

Supplemental data

Supplemental data for this article can be accessed on the publisher’s website.

Additional Contributions

We would like to thank Todd Durham, PhD. and Brian Mansfield, PhD. (Foundation Fighting Blindness), for their input on the questionnaire and for helping disseminate the survey to RP patients enrolled in the My Retina Tracker Registry of the Foundation Fighting Blindness. We also greatly appreciate the patients and families for sharing their opinions through participating in the survey.

Declaration of interest

There is no conflict of interest to disclose for XK, CD, ST, FN and CAL. The disclosure for PAC is as below: Personal financial interest with Aerpio Pharmaceuticals, Allegro, Applied Genetic Technologies Corporation, Asclepix Therapeutics, Ashvattha Therapeutics, Bausch and Lomb, Clearside, CUREVAC, Exonate Ltd., Genentech/Roche Inc, Graybug Vision, Merck & Co., Novartis Pharmaceuticals, Perfuse, Wave Life Sciences; Financial support with Asclepix Therapeutics, Genentech/Roche Inc, Mallinckrodt Pharmaceuticals, Oxford Biomedica, Regeneron Pharmaceuticals, Inc., RegenixBio Sanofi/Genzyme.

Additional information

Funding

This work was supported by grant R34EY031429 from the National Eye Institute and funds from the Research to Prevent Blindness awarded to the Wilmer Eye Institute.