Association of Patient Demographic Characteristics and Insurance Status With Survival in Cancer Randomized Clinical Trials With Positive Findings

Importance Few new treatments tested in phase 3 cancer randomized clinical trials show an overall survival benefit. Although understanding whether the benefits are consistent among all patient groups is critical for informing guideline care, individual trials are designed to assess the benefits of experimental treatments among all patients and are too small to reliably determine whether treatment benefits apply to demographic or insurance subgroups. Objective To systematically examine whether positive treatment effects in cancer randomized clinical trials apply to specific demographic or insurance subgroups. Design, Setting, and Participants Cohort study of pooled patient-level data from 10 804 patients in SWOG Cancer Research Network clinical treatment trials reported from 1985 onward with superior overall survival for those receiving experimental treatment. Patients were enrolled from 1984 to 2012. Maximum follow-up was 5 years. Main Outcomes and Measures Interaction tests were used to assess whether hazard ratios (HRs) for death comparing standard group vs experimental group treatments were associated with age (>= 65 vs <65 years), race/ethnicity (minority vs nonminority populations), sex, or insurance status among patients younger than 65 years (Medicaid or no insurance vs private insurance) in multivariable Cox regression frailty models. Progression- or relapse-free survival was also examined. Data analyses were conducted from August 2019 to February 2020. Results In total, 19 trials including 10 804 patients were identified that reported superior overall survival for patients randomized to experimental treatment. Patients were predominantly younger than 65 years (67.3%) and female (66.3%); 11.4% were black patients, and 5.7% were Hispanic patients. There was evidence of added survival benefits associated with receipt of experimental therapy for all groups except for patients with Medicaid or no insurance (HR, 1.23; 95% CI, 0.97-1.56; P = .09) compared with those with private insurance (HR, 1.66; 95% CI, 1.44-1.92; P < .001; P = .03 for interaction). Receipt of experimental treatment was associated with reduced added overall survival benefits in patients 65 years or older (HR, 1.21; 95% CI, 1.11-1.32; P < .001) compared with patients younger than 65 years (HR, 1.41; 95% CI, 1.30-1.53; P < .001; P = .01 for interaction), although both older and younger patients appeared to strongly benefit from receipt of experimental treatment. The progression- or relapse-free survival HRs did not differ by age, sex, or race/ethnicity but differed between patients with Medicaid or no insurance (HR, 1.32; 95% CI, 1.06-1.64; P = .01) vs private insurance (HR, 1.74; 95% CI, 1.54-1.97; P < .001; P = .03 for interaction). Conclusions and Relevance Patients with Medicaid or no insurance may have smaller added benefits from experimental therapies compared with standard treatments in clinical trials. A better understanding of the quality of survivorship care that patients with suboptimal insurance receive, including supportive care and posttreatment care, could help establish how external factors may affect outcomes for these patients. This cohort study uses pooled patient-level data from the SWOG Cancer Research Network to assess whether positive treatment effects are associated with demographic or insurance subgroups among patients participating in phase 3 cancer randomized clinical trials. Question Do positive treatment effects from cancer clinical trials apply to all sociodemographic groups? Findings In this cohort study using patient-level data recorded for 10 804 patients who participated in cancer randomized clinical trials with positive findings, the receipt of experimental treatment vs standard treatment was associated with improved overall survival among patients 65 years or older compared with patients younger than 65 years. No significant added benefit of experimental treatment was observed among patients having Medicaid or no insurance compared with those with private insurance. Meaning Patients with Medicaid or no insurance may have smaller added benefits from experimental therapies in clinical trials.