Meta-analysis of risks and benefits of anticancer drugs in advanced cancer patients
Randomized clinical trials of anticancer drugs without active comparators in patients who have exhausted standard of care treatment options are debated. A systematic review and meta-analysis of 128 trials involving 47,432 of these patients found that the pooled HRs for PFS and OS were 0.58 (95% CI 0.53-0.63) and 0.82 (95% CI 0.78-0.85). The absolute benefits however were limited wit PFS and OS gains of 2.1 and 0.5 months. The absolute excesses in all grade, grade III-IV, and grade V adverse events between the two arms were +13.9%, +10.2%, and +0.5%. The correlation between the excess of severe toxicity and efficacy was weak (all R2<0.2). The authors conclude that anticancer drugs evaluated in RCTs against no active treatment benefited trial participants. Severe toxicity was not significantly correlated with efficacy.