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.


CONCLUSIONS
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.
Sato
Keiko
K
Genetic Counseling and Clinical Research Unit, Kyoto University School of Public Health, Yoshida Konoe-cho, Sakyo-ku, Kyoto 606-8501, Japan. keiko.sato@kt2.ecs.kyoto-u.ac.jp
Sato
Tosiya
T
Furuse
Junji
J
Kasugai
Hiroshi
H
Konishi
Masaru
M
Kosuge
Tomoo
T
Saito
Akiko
A
Sasaki
Yo
Y
Takasaki
Ken
K
Okusaka
Takuji
T
D006528:Carcinoma, Hepatocellular / Q000601:surgery; D006801:Humans; D008113:Liver Neoplasms / Q000601:surgery; D010349:Patient Compliance / Q000523:psychology / Q000706:statistics & numerical data; D010358:Patient Participation / Q000523:psychology / Q000706:statistics & numerical data; D057240:Patient Preference / Q000523:psychology / Q000706:statistics & numerical data; D016032:Randomized Controlled Trials as Topic / Q000379:methods* / Q000523:psychology*; D011795:Surveys and Questionnaires