The control rate was significantly related to the risk difference in 31% of meta-analyses, compared to 13% for relative risk and 14% for odds ratio, indicating it predicts treatment efficacy.
Does the control rate predict treatment efficacy in meta-analyses of clinical trials?
The control rate is significantly correlated with treatment effect measures, particularly risk difference, suggesting investigators should explore causes of heterogeneity related to patient characteristics and treatment protocols rather than just pooling estimates.
If the control rate (CR) in a clinical trial represents the incidence or the baseline severity of illness in the study population, the size of treatment effects may tend to very with the size of control rates. To investigate this hypothesis, we examined 115 meta-analyses covering a wide range of medical applications for evidence of a linear relationship between the CR and three treatment effect (TE) measures: the risk difference (RD); the log relative risk (RR), and the log odds ratio (OR). We used a hierarchical model that estimates the true regression while accounting for the random error in the measurement of and the functional dependence between the observed TE and the CR. Using a two standard error rule of significance, we found the control rate was about two times more likely to be significantly related to the RD (31 per cent) than to the RR (13 per cent) or the OR (14 per cent). Correlations between TE and CR were more likely when the meta-analysis included 10 or more trials and if patient follow-up was less than six months and homogeneous. Use of weighted linear regression (WLR) of the observed TE on the observed CR instead of the hierarchical model underestimated standard errors and overestimated the number of significant results by a factor of two. The significant correlation between the CR and the TE suggests that, rather than merely pooling the TE into a single summary estimate, investigators should search for the causes of heterogeneity related to patient characteristics and treatment protocols to determine when treatment is most beneficial and that they should plan to study this heterogeneity in clinical trials.
Schmid et al. (Tue,) reported a other. Control rate (CR) was evaluated on Linear relationship between the control rate (CR) and treatment effect (TE) measures (RD, RR, OR). The control rate was significantly related to the risk difference in 31% of meta-analyses, compared to 13% for relative risk and 14% for odds ratio, indicating it predicts treatment efficacy.