Quantitative bias analyses to address measurement error in time-to-event endpoints
Year: 2026
Type: article
Abstract: When using real-world data to construct an external comparator arm for a single-arm trial, there may be differences in how and when patients are assessed for disease between trial and real-world settings. Such differences can generate outcome measurement error when comparing time-to-event endpoints and lead to biased findings. Recent methods have been developed to mitigate measurement error bias in real-world endpoints; however, they rely on the existence of a validation sample, ie, data on a set of patients where both the “true” trial-like and “mis-measured” real-world measures are collected. We demonstrate how novel statistical methods can be leveraged as quantitative bias analyses (QBA) to contextualize real-world evidence findings when outcome measurement error is of concern, but validation samples are infeasible to collect. Quantitative bias analyses allow researchers to set plausible ranges for the amount of error when not directly measurable. We highlight how to conduct QBA with two recent methods, cumulative incidence curve correction and survival regression calibration, and illustrate how to generate plausible parameter values through simulation. We provide an illustrative QBA example in a cohort of real-world patients with newly diagnosed multiple myeloma and provide practical guidance to apply QBA for outcome measurement error and interpret results.
Source: UNC Libraries
DOI: 10.17615/khta-2m20
Authors: Batavia, Ashita S, Weberpals, Janick, Giri, Smith, Roose, James, Stürmer, Til, Brayer, Jason, Wang, Jocelyn R, Ailawadhi, Sikander, Nadeem, Omar, Ackerman, Benjamin, Hester, Laura, Siddique, Juned, Hayden, Jennifer, Zhang, Youyi, Gan, Ryan W, Meyer, Craig S, Raje, Noopur, Schneeweiss, Sebastian, Lund, Jennifer L
Institution: University of North Carolina at Chapel Hill
Language: English
Subfield: Statistics and Probability
Field: Mathematics
Domain: Physical Sciences
Open Access status: green
Funders: Howard Hughes Medical Institute, Smithsonian Institution, National Aeronautics and Space Administration, American Heart Association, University of Texas Southwestern Medical Center, Education Department of Hunan Province, Wellcome Trust, UK Research and Innovation, Zegar Family Foundation, Global Brain Health Institute, Chinese Association of Integrative Medicine, Paul M. Angell Family Foundation, Carnegie Institution for Science, Chongqing Municipal Health Commission, Rotary Yoneyama Memorial Foundation, Health Commission of Heilongjiang Province, Volgenau Foundation, Leverhulme Trust, Deutsche Forschungsgemeinschaft, National Natural Science Foundation of China, Austrian Science Fund, Bundesministerium für Gesundheit, Fraunhofer-Gesellschaft, Guangxi Zhuang Region Health Department, Hellenic Society for the Study of Bone Metabolism, Princess Nourah Bint Abdulrahman University, Shin Kong Wu Ho-Su Memorial Hospital, Tatung University, Cheng Hsin General Hospital Foundation, Earth and Life Systems Alliance, Natural Science Foundation of Shaanxi Province, Tri-Service General Hospital, Army Medical University, Natural Science Foundation Project of Chongqing, Chongqing Science and Technology Commission, Ministry of Science and ICT, South Korea, Health Commission of Jiangxi Province, National Institutes of Health, Centers for Disease Control and Prevention, Agency for Healthcare Research and Quality, U.S. Fish and Wildlife Service, National Institute of Food and Agriculture, Center for Clinical and Translational Sciences, University of Texas Health Science Center at Houston, Materials Research Science and Engineering Center, Northwestern University, NIHR Maudsley Biomedical Research Centre, Biotechnology and Biological Sciences Research Council, Natural Environment Research Council, Zhejiang Traditional Chinese Medicine Administration, West China Hospital, Sichuan University, National Institute for Health Research Applied Research Collaboration South London, National Institute on Aging, National Heart, Lung, and Blood Institute, National Institute on Drug Abuse, National Institute of Neurological Disorders and Stroke, National Institute of Environmental Health Sciences, Division of Civil, Mechanical and Manufacturing Innovation, Division of Electrical, Communications and Cyber Systems, Air Force Office of Scientific Research, UK Prevention Research Partnership
OpenAlex ID: W7212321934