A conundrum for randomized controlled trials: experience from a small hepatocellular carcinoma trial.

Objective: The aim of this study was to explore why patients accepted or declined to participate in a randomized clinical trial, which was subsequently discontinued because of a low recruitment rate.

Methods: Forty-one patients were invited to participate in a randomized clinical trial that aimed to compare local ablation therapies and surgery to treat small asymptomatic hepatocellular carcinomas. These patients were then asked to answer a questionnaire that assessed patient perception and reasons for accepting or declining to enroll in the randomized clinical trial. When patients had a strong preference for a specific treatment, the questionnaire assessed why, how and when they had chosen it.

Results: The response rate was 6/6 (100%) and 30/35 (86%) for the participant and non-participant groups, respectively. Among the 30 non-participants, 23 had a strong preference for local ablation therapies, which was less invasive and offered shorter hospitalization. Patient preference for a specific treatment often stemmed from their consultations with a clinician who referred them to a specialist hospital. Patients without strong preference for a specific treatment participated in the randomized clinical trial because of altruistic motivations.

Conclusion: When new treatments that are innovative and less burdensome become widespread, they are difficult to compare with standard therapy utilizing a well-designed randomized clinical trial. Consequently, when an innovative treatment is developed, investigators should consider designing a randomized clinical trial as early as possible.