When are clinical trials beneficial for study patients and future patients? A factorial vignette-based survey of institutional review board members.

ABSTRACT

OBJECTIVE: The ethicists believe that the goal of clinical research is to benefit future and not current (trial) patients. Many clinicians believe that the clinical trial enrolment offers best management for their patients. The objective of our study was to identify the situations when a clinical trial is beneficial for the patients enrolled in the trial and future patients. DESIGN: Factorial vignette-based cross-sectional survey via the internet. PARTICIPANTS: Institutional review board (IRB) members of the US Medical Schools. MAIN OUTCOME MEASURES: Each participant was invited to review 9 clinical vignettes related to (1) study approval and (2) the assessment if the study is designed to help future or current patients more. RESULTS: A total of 232 IRB members from 42 institutions participated. When considering approval of the trial, we found that uncertainty about treatment effects (OR=1.13; 95% CI 1.08 to 1.19) and requirement for continuation of standard therapy (OR=3.84; 95% CI 2.7 to 5.55) were the only statistically significant factors affecting IRB members' decisions to approve the study. The odds of IRB members approving a trial increased when a trial proposed to enrol patients with life-threatening versus chronic debilitating disease (OR=2.04; 95% CI 1.47 to 2.86). We also found that similar factors affected judgements related to the assessment whether the trial will benefit future or current patients more-(1) future patients uncertainty (OR=1.27; 95% CI 1.18 to 1.37); continuation of standard treatment (OR=1.66; 95% CI 1.07 to 2.56); seriousness of condition (OR=1.78; 95% CI 1.15 to 2.28); (2) current patients uncertainty (OR=1.54; 95% CI 1.4 to 1.7); continuation of standard therapy (OR=2.17; 95% CI 1.39 to 3.44). CONCLUSIONS: IRB members view the proposed studies to be beneficial for current patients and future patients if there is uncertainty regarding treatment effects and if studies do not require stopping the current treatment. This finding supports the design and use of pragmatic trials which may reduce therapeutic misconception and improve trial enrolment, speeding up therapeutic discoveries.