Validation of Colorectal Cancer Models on Long-term Outcomes from a Randomized Controlled Trial

Maria DeYoreo, Iris Lansdorp-Vogelaar, Amy B. Knudsen, Karen M. Kuntz, Ann G. Zauber and Carolyn M. Rutter
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Maria DeYoreo: RAND Corporation, Santa Monica, CA, USA
Iris Lansdorp-Vogelaar: Department of Public Health, Erasmus MC, Rotterdam, Zuid-Holland, the Netherlands
Amy B. Knudsen: Institute for Technology Assessment and Harvard Medical School, Massachusetts General Hospital, Boston, MA, USA
Karen M. Kuntz: Department of Health Policy and Management, University of Minnesota, School of Public Health, Minneapolis, MN, USA
Ann G. Zauber: Department of Epidemiology and Biostatistics, Memorial Sloan Kettering Cancer Center, NY, USA
Carolyn M. Rutter: RAND Corporation, Santa Monica, CA, USA

Medical Decision Making, 2020, vol. 40, issue 8, 1034-1040

Abstract: Microsimulation models are often used to predict long-term outcomes and guide policy decisions regarding cancer screening. The United Kingdom Flexible Sigmoidoscopy Screening (UKFSS) Trial examines a one-time intervention of flexible sigmoidoscopy that was implemented before a colorectal cancer (CRC) screening program was established. Long-term study outcomes, now a full 17 y following randomization, have been published. We use the outcomes from this trial to validate 3 microsimulation models for CRC to long-term study outcomes. We find that 2 of 3 models accurately predict the relative effect of screening (the hazard ratios) on CRC-specific incidence 17 y after screening. We find that all 3 models yield predictions of the relative effect of screening on CRC incidence and mortality (i.e., the hazard ratios) that are reasonably close to the UKFSS results. Two of the 3 models accurately predict the relative reduction in CRC incidence 17 y after screening. One model accurately predicted the absolute incidence and mortality rates in the screened group. The models differ in their estimates related to adenoma detection at screening. Although high-quality screening results help to inform models, trials are expensive, last many years, and can be complicated by ethical issues and technological changes across the duration of the trial. Thus, well-calibrated and validated models are necessary to predict outcomes for which data are not available. The results from this validation demonstrate the utility of models in predicting long-term outcomes and in collaborative modeling to account for uncertainty.

Keywords: colorectal cancer; colorectal cancer screening; microsimulation model; model validation (search for similar items in EconPapers)
Date: 2020
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Persistent link: https://EconPapers.repec.org/RePEc:sae:medema:v:40:y:2020:i:8:p:1034-1040

DOI: 10.1177/0272989X20961095

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