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, Carolyn M. Rutter

Research output: Contribution to journalArticlepeer-review

6 Scopus citations


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.

Original languageEnglish (US)
Pages (from-to)1034-1040
Number of pages7
JournalMedical Decision Making
Issue number8
StatePublished - Nov 2020

Bibliographical note

Funding Information:
The authors disclosed receipt of the following financial support for the research, authorship, and/or publication of this article: Financial support for this study was provided by a grant from National Cancer Institute as part of the Cancer Intervention and Surveillance Modeling Network (CISNET) U01 CA199335 and cancer center support grant P30 CA008748. The funding agreement ensured the authors’ independence in designing the study, interpreting the data, writing, and publishing the report. The contents of this article are solely the responsibility of the authors and do not necessarily represent the official views of the National Cancer Institute.

Publisher Copyright:
© The Author(s) 2020.


  • colorectal cancer
  • colorectal cancer screening
  • microsimulation model
  • model validation


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