Statistical analyses
tumor, death
EVENTS, DISEASE, SECONDARY, TUMOR
This study was designed to provide 80% power for assessment of the primary PFS endpoint using the following assumptions to determine the sample size: an overall type I error rate of 0.1 (one‐sided), a 1:1 randomization ratio, a median PFS for the placebo arm of 6.0 months, and a PFS hazard ratio (HR; pamiparib/placebo) of 0.63. A sample size of approximately 128 patients (64 per treatment arm) was required to achieve 85 PFS events within the planned study duration of approximately 26 months after the first patient was randomized to the study, assuming an estimated accrual period of 20 months.The intent‐to‐treat analysis set included all randomized patients who were assigned to receive either pamiparib or placebo; used for PFS, OS, and TSST analyses. The efficacy evaluable analysis set included all randomized patients who had measurable disease at baseline and at least one post baseline tumor assessment (unless treatment was discontinued due to clinical progression or death); used for all efficacy analyses related to ORR, DoR, and TTR. The safety analysis set included all patients in the intent‐to‐treat analysis set who received any dose of pamiparib or placebo.The final PFS analysis was performed when 85 PFS events had occurred. A stratified one‐sided log‐rank test at a 0.1 significance level incorporating the randomized stratification factors was used to compare treatment arms for the primary endpoint. The treatment effect on PFS was estimated by fitting a Cox proportional hazards model to the PFS, including treatment arm as a factor and stratification factors (region and ECOG PS) as strata. From this model, the HR was estimated and presented with a two‐sided 95% confidence interval (CI).For the OS secondary endpoint, a stratified log‐rank test that incorporated the randomized stratification factors was used to compare treatment arms in the intent‐to‐treat analysis set. The HR and its two‐sided 95% CI were estimated using the stratified Cox proportional hazards model. Median OS, TSST, and DoR for each treatment arm was estimated using the Kaplan–Meier method, and the 95% CI was calculated using the Brookmeyer–Crowley method. The ORR and its exact two‐sided 95% CI were calculated for each treatment arm, with the Cochran–Mantel–Haenszel score test was used to compare treatment arms in the efficacy evaluable analysis set. TTR was assessed using descriptive statistics. Only patients with a response of CR or PR during the study were included in the DoR and TTR analyses.