Comparison of Survival Outcomes Among Cancer Patients Treated In and Out of Clinical Trials

• Published in: JNCI : Journal of the National Cancer Institute Vol. 106; no. 3; p. dju002
• Main Authors: Unger, Joseph M., Barlow, William E., Martin, Diane P., Ramsey, Scott D., LeBlanc, Michael, Etzioni, Ruth, Hershman, Dawn L.
• Format: Journal Article
• Language: English
• Published: Cary, NC Oxford University Press 01.03.2014; Oxford Publishing Limited (England)
• Subjects: Biological and medical sciences; Cancer; Cancer Care Facilities; Clinical outcomes; Clinical trials; Clinical Trials as Topic - statistics & numerical data; Comorbidity; Comparative analysis; Humans; Kaplan-Meier Estimate; Medical sciences; Multiple tumors. Solid tumors. Tumors in childhood (general aspects); Multivariate Analysis; National Cancer Institute (U.S.); Neoplasms - diagnosis; Neoplasms - mortality; Neoplasms - therapy; Odds Ratio; Patient Selection; Prognosis; Proportional Hazards Models; SEER Program; Selection Bias; Survival analysis; Survival Rate; Tumors; United States - epidemiology; United States; Human; Cancerology; Prognosis; Treatment; Clinical trial; Evolution; Malignant tumor; Survival; Comparative study; Cancer
• ISSN: 0027-8874; 1460-2105; 1460-2105
• DOI: 10.1093/jnci/dju002

Clinical trials test the efficacy of a treatment in a select patient population. We examined whether cancer clinical trial patients were similar to nontrial, "real-world" patients with respect to presenting characteristics and survival. We reviewed the SWOG national clinical trials consortium database to identify candidate trials. Demographic factors, stage, and overall survival for patients in the standard arms were compared with nontrial control subjects selected from the Surveillance, Epidemiology, and End Results program. Multivariable survival analyses using Cox regression were conducted. The survival functions from aggregate data across all studies were compared separately by prognosis (≥50% vs <50% average 2-year survival). All statistical tests were two-sided. We analyzed 21 SWOG studies (11 good prognosis and 10 poor prognosis) comprising 5190 patients enrolled from 1987 to 2007. Trial patients were younger than nontrial patients (P < .001). In multivariable analysis, trial participation was not associated with improved overall survival for all 11 good-prognosis studies but was associated with better survival for nine of 10 poor-prognosis studies (P < .001). The impact of trial participation on overall survival endured for only 1 year. Trial participation was associated with better survival in the first year after diagnosis, likely because of eligibility criteria that excluded higher comorbidity patients from trials. Similar survival patterns between trial and nontrial patients after the first year suggest that trial standard arm outcomes are generalizable over the long term and may improve confidence that trial treatment effects will translate to the real-world setting. Reducing eligibility criteria would improve access to clinical trials.