Assessing the Validity of a Patient-Trial Generalizability Score Using Real-world Data from a Large Clinical Data Research Network: A Colorectal Cancer Clinical Trial Case Study

Existing trials had not taken enough consideration of their population representativeness, which can lower the effectiveness when the treatment is applied in real-world clinical practice. We analyzed the eligibility criteria of Bevacizumab colorectal cancer treatment trials, assessed their a priori generalizability, and examined how it affects patient outcomes when applied in real-world clinical settings. To do so, we extracted patient-level data from a large collection of electronic health records (EHRs) from the OneFlorida consortium. We built a zero-inflated negative binomial model using a composite patient-trial generalizability (cPTG) score to predict patients' clinical outcomes (i.e., number of serious adverse events, [SAEs]). Our study results provide a body of evidence that 1) the cPTG scores can predict patient outcomes; and 2) patients who are more similar to the study population in the trials that were used to develop the treatment will have a significantly lower possibility to experience serious adverse events.