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The value of explicitly emulating a target trial when using real world evidence: an application to colorectal cancer screening.

ABSTRACT: Observational analyses for causal inference often rely on real world data collected for purposes other than research. A frequent goal of these observational analyses is to use the data to emulate a hypothetical randomized experiment, i.e., the target trial, that mimics the design features of a true experiment, including a clear definition of time zero with synchronization of treatment assignment and determination of eligibility. We review a recent observational analysis that explicitly emulated a target trial of screening colonoscopy using insurance claims from U.S. Medicare. We then compare this explicit emulation with alternative, simpler observational analyses that do not synchronize treatment assignment and eligibility determination at time zero and/or do not allow for repeated eligibility. This empirical comparison suggests that lack of an explicit emulation of the target trial leads to biased estimates, and shows that allowing for repeated eligibility increases the statistical efficiency of the estimates.

SUBMITTER: Garcia-Albeniz X

PROVIDER: S-EPMC5759953 | biostudies-literature | 2017 Jun

REPOSITORIES: biostudies-literature

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The value of explicitly emulating a target trial when using real world evidence: an application to colorectal cancer screening.

García-Albéniz Xabier X Hsu John J Hernán Miguel A MA

European journal of epidemiology 20170601 6

Observational analyses for causal inference often rely on real world data collected for purposes other than research. A frequent goal of these observational analyses is to use the data to emulate a hypothetical randomized experiment, i.e., the target trial, that mimics the design features of a true experiment, including a clear definition of time zero with synchronization of treatment assignment and determination of eligibility. We review a recent observational analysis that explicitly emulated ...[more]

PMID: 28748498

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Project description:PurposeUsing real-world data (RWD)-based trial simulation approach, we aim to simulate colorectal cancer (CRC) trials and examine both effectiveness and safety end points in different simulation scenarios.MethodsWe identified five phase III trials comparing new treatment regimens with an US Food and Drug Administration-approved first-line treatment in patients with metastatic CRC (ie, fluorouracil, leucovorin, and irinotecan) as the standard-of-care (SOC) control arm. Using Electronic Health Record-derived data from the OneFlorida network, we defined the study populations and outcome measures using the protocols from the original trials. Our design scenarios were (1) simulation of the SOC fluorouracil, leucovorin, and irinotecan arm and (2) comparative effectiveness research (CER) simulation of the control and experimental arms. For each scenario, we adjusted for random assignment, sampling, and dropout. We used overall survival (OS) and severe adverse events (SAEs) to measure effectiveness and safety.ResultsWe conducted CER simulations for two trials, and SOC simulations for three trials. The effect sizes of our simulated trials were stable across all simulation runs. Compared with the original trials, we observed longer OS and higher mean number of SAEs in both CER and SOC simulation. In the two CER simulations, hazard ratios associated with death from simulations were similar to that reported in the original trials. Consistent with the original trials, we found higher risk ratios of SAEs in the experiment arm, suggesting potentially higher toxicities from the new treatment regimen. We also observed similar SAE rates across all simulations compared with the original trials.ConclusionIn this study, we simulated five CRC trials, and tested two simulation scenarios with several different configurations demonstrated that our simulations can robustly generate effectiveness and safety outcomes comparable with the original trials using real-world data.

Project description:IntroductionThe enhanced recovery after surgery (ERAS) protocol provides optimised care guidelines for patients undergoing elective colorectal surgery. To ensure high compliance with active ERAS elements, patients must be educated to actively participate in the perioperative care pathway. Mobile health is a rapidly expanding area of the digital health sector that is effective in educating and engaging patients during follow-up. iColon is a mobile application designed by the Operative Unit of General Surgery of IRCCS Sacro Cuore Don Calabria Hospital of Negrar of Valpolicella, which is specifically targeted at patients undergoing elective colorectal surgery. iColon is organised into ERAS phases, and it provides real-time feedback to surgeons about a patient's adherence to perioperative active ERAS elements.Methods and analysisWe hypothesise that by providing a patient-focused mobile application, compliance with active ERAS elements could be improved.The first coprimary objective is to build patient confidence in using the mobile application, iColon, during perioperative care. The second coprimary objective is to establish patient compliance with active ERAS elements.Secondary objectives include examining: length of stay, 30-day readmission rate, postoperative complications and patient satisfaction of received care.This study is a prospective observational real-world study of patients undergoing elective colorectal surgery who are following the ERAS protocol and using iColon during perioperative periods between September 2020 and December 2022.By educating and engaging patients in the ERAS protocol, the mobile application, iColon, should stimulate patients to be more proactive in managing their healthcare by complying more closely with active ERAS elements.Ethics and disseminationThis study has been approved by the local Ethics Committee with the protocol number 29219 of 25 May 2020. The results will be actively disseminated through peer-reviewed journals, conference presentations and various community engagement activities.

Dataset Information

The value of explicitly emulating a target trial when using real world evidence: an application to colorectal cancer screening.

Publications

The value of explicitly emulating a target trial when using real world evidence: an application to colorectal cancer screening.

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