BACKGROUND: In a multinational trial designed to determine the efficacy of epoetin alfa in chemotherapy‐induced anemia, patients receiving non‐platinum chemotherapy having a hemoglobin 10.5 g/dL or less, or a decline in hemoglobin of 1.5 g/dL or greater were randomized (2:1) to epoetin alfa or placebo. A total of 375 patients (251 epoetin alfa, 124 placebo) were assessed for survival status twelve months after completing the protocol, but prior to unblinding. A log‐rank test showed a trend in survival favoring epoetin alfa (median of 17 vs. 11 months, p = 0.128). OBJECTIVES: The primary efficacy endpoint for many cancer clinical trials is median survival. In preparing for an economic analysis, however, we analyzed mean survival, the appropriate survival endpoint for a C/E analysis. METHODS: Sampling with replacement, we conducted a post hoc analysis that examined the difference in mean survival by drawing 10,000 samples in a bootstrapping simulation. Within each sample the survival curves were truncated to maintain identical follow‐up periods between treatment groups. The difference in mean survival was computed for each sample. The probability of superior efficacy was obtained by sorting the results from the samples. RESULTS: The average mean survival difference, across the 10,000 samples, showed a 0.212‐year survival benefit for epoetin alfa. The probability that the difference in mean survival favors epoetin alfa was 0.965. CONCLUSIONS: Comparing differences in median and mean survival may lead to different conclusions about the value of a therapy. Given that mean survival is the appropriate effectiveness endpoint for survival‐based C/E analyses, a non‐significant difference in median survival does not preclude full C/E analyses. Specifically, the mean survival results from this trial warrant a full C/E analysis of epoetin alfa in treating anemia for patients receiving non‐platinum chemotherapy.
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