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Clinical Trial Simulation in Diabetic Retinopathy: Insights from Patients and Site Staff
Stela Vujosevic1,2, Ivana Gunderson3, Asma Burale4
1Department of Biomedical, Surgical and Dental Sciences, University of Milan, Milan, Italy. stela.vujosevic@unimi.it.
Introduction:
High patient burdens from diabetic retinopathy (DR)-associated vision loss and intravitreal therapy (IVT) support patient experience inclusion in DR trial designs. This trial simulation characterized patient and site staff opinions to improve future nonproliferative DR (NPDR) trial designs.
Methods:
Between March 27 and May 31, 2023, survey data were collected from trial simulation participants. After a preread and trial design animation, study features were simulated followed by a 75-90-min web-assisted telephone interview. Patients with NPDR and trial site staff from the United States, United Kingdom, and Germany were included. The likelihood of patient participation and the challenges faced by site staff in conducting the simulated clinical trial at their study site were assessed using a 1-7 scale. Outcomes were evaluated via thematic analysis and descriptive statistics.
Results:
Twenty-two patients aged 36-55 years and mostly female (59.1%), and 16 site staff were interviewed. Mean NPDR duration was 9.3 years; most patients (81.8%) had never participated in a clinical trial. Although eligibility criteria resembled other trials, site staff indicated that restrictive exclusion criteria of the trial simulation could limit recruitment and that endpoints did not match patients' goals, which mainly focused on saving vision. The proposed 4-5-h on-site visits and 72-week trial length were considered "too long" by 45.5% and 50.0% of patients, respectively. For the 1:2 sham or active treatment allocation ratio, responses were 40.9% neutral, 36.4% positive, and 22.7% negative. Some patients misunderstood that sham injections imitate actual injections, expressing concerns about adverse events. Patients reported IVT-related anxieties, particularly IVT-inexperienced patients. Mean patient trial participation interest score was 4.9/7; 62.5% of site staff were interested in conducting the trial. Some proposed adaptations were implemented in the trial protocol (e.g., offering patient/caregiver transportation).
Conclusions:
Insights gained from respondent feedback in this simulation may inform future DR clinical trial design, potentially enhancing recruitment rates and patient experience.
Insights
Patient feedback on a simulated nonproliferative diabetic retinopathy (NPDR) trial highlighted concerns about trial length and endpoints. Incorporating patient insights can improve future diabetic retinopathy (DR) clinical trial designs and recruitment.
Area of Science:
- Ophthalmology
- Clinical Trial Design
- Patient-Reported Outcomes
Background:
- Diabetic retinopathy (DR) causes significant vision loss and treatment burdens.
- Intravitreal therapy (IVT) is a common treatment for DR.
- Patient experience is crucial for effective clinical trial design in DR.
Purpose of the Study:
- To characterize patient and site staff opinions on a simulated nonproliferative diabetic retinopathy (NPDR) trial.
- To identify factors influencing patient participation and site staff challenges in DR clinical trials.
- To inform improvements in future NPDR trial designs for better recruitment and patient experience.
Main Methods:
- A simulated NPDR clinical trial was presented to patients and site staff.
- Surveys and web-assisted telephone interviews were conducted between March and May 2023.
- Data were analyzed using thematic analysis and descriptive statistics to assess participation likelihood and site challenges.
Main Results:
- Patients found the proposed trial length (72 weeks) and visit duration (4-5 hours) excessive.
- Site staff noted restrictive exclusion criteria and misaligned endpoints as potential recruitment barriers.
- Patients expressed anxieties regarding intravitreal therapy (IVT) and sham injections, particularly those new to treatment.
- Patient interest in participation was moderate (4.9/7), while site staff interest was higher (62.5%).
Conclusions:
- Patient and site staff feedback provides valuable insights for optimizing DR trial protocols.
- Addressing patient concerns about trial duration, endpoints, and treatment anxieties can enhance recruitment.
- Adaptations, such as transportation support, can improve the patient experience in DR clinical trials.
