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

Area of Science:

• Hepatobiliary Surgery
• Oncology
• Clinical Trial Design

Background:

• Hepatocellular carcinoma (HCC) is a significant global health concern.
• Small asymptomatic HCCs can be treated with local ablation or surgery.
• Randomized clinical trials (RCTs) are essential for comparing treatment efficacy.

Purpose of the Study:

• To investigate patient reasons for accepting or declining participation in an RCT comparing local ablation and surgery for small asymptomatic HCC.
• To understand patient preferences and decision-making processes regarding treatment options.

Main Methods:

• A questionnaire was administered to patients invited to an RCT comparing local ablation and surgery for small asymptomatic HCC.
• The questionnaire assessed patient perceptions and reasons for accepting or declining trial enrollment.
• For patients with a strong treatment preference, the questionnaire explored the origin and timing of their choice.

Main Results:

• High response rates were achieved from both participant (100%) and non-participant (86%) groups.
• A majority of non-participants (23/30) preferred local ablation due to its less invasive nature and shorter hospital stay.
• Patient treatment preferences were often influenced by clinician consultations and referrals.

Conclusions:

• Patient preference for less invasive treatments poses challenges for recruiting participants into RCTs comparing them with standard therapies.
• Early consideration of RCT design is vital when innovative and less burdensome treatments emerge.
• Future RCTs should proactively address patient preferences to improve recruitment and trial success.