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ORIGINAL RESEARCH

Oncology Drug Effectiveness from Electronic Health Record Data Calibrated Against RCT Evidence: The PARSIFAL Trial Emulation

ORCID Icon, , , , & ORCID Icon
Pages 1135-1144 | Received 04 May 2022, Accepted 20 Jul 2022, Published online: 10 Oct 2022

References

  • Cartwright TH, Clayton M, Garey JS, Boehm KA. Use of an electronic health record (iKnowMed), in conjunction with evidence-based pathways, for data capture and outcome measurement in colorectal cancer patients treated with first-line therapy in the US Oncology Network. Am J Clin Oncol. 2010;28(15_suppl):3626. doi:10.1200/jco.2010.28.15_suppl.3626
  • Parikh RB, Galsky MD, Gyawali B, et al. Trends in checkpoint inhibitor therapy for advanced urothelial cell carcinoma at the end of life: insights from real-world practice. Oncologist. 2019;24(6):e397–e399. doi:10.1634/theoncologist.2019-0039
  • Administration FaD. Real-World Evidence; 2021. Available from: https://www.fda.gov/science-research/science-and-research-special-topics/real-world-evidence. Accessed September 29, 2022.
  • Califf RM, Robb MA, Bindman AB, et al. Transforming evidence generation to support health and health care decisions. N Engl J Med. 2016;375(24):2395–2400. doi:10.1056/NEJMsb1610128
  • Eichler HG, Koenig F, Arlett P, et al. Are novel, nonrandomized analytic methods fit for decision making? The need for prospective, controlled, and transparent validation. Clin Pharmacol Ther. 2020;107(4):773–779. doi:10.1002/cpt.1638
  • Brufsky A, Liu X, Li B, McRoy L, Layman RM. Real-world tumor response of palbociclib plus letrozole versus letrozole for metastatic breast cancer in US clinical practice. Target Oncol. 2021;16(5):601–611. doi:10.1007/s11523-021-00826-1
  • Carrigan G, Whipple S, Capra WB, et al. Using electronic health records to derive control arms for early phase single-arm lung cancer trials: proof-of-concept in randomized controlled trials. Clin Pharmacol Ther. 2020;107(2):369–377. doi:10.1002/cpt.1586
  • Paul SK, Montvida O, Best JH, Gale S, Pethoe-Schramm A, Sarsour K. Effectiveness of biologic and non-biologic antirheumatic drugs on anaemia markers in 153,788 patients with rheumatoid arthritis: new evidence from real-world data. Semin Arthritis Rheum. 2018;47(4):478–484. doi:10.1016/j.semarthrit.2017.08.001
  • Tan K, Bryan J, Segal B, et al. Emulating control arms for cancer clinical trials using external cohorts created from electronic health record-derived real-world data. Clin Pharmacol Ther. 2021;111:168–178. doi:10.1002/cpt.2351
  • Booth CM, Karim S, Mackillop WJ. Real-world data: towards achieving the achievable in cancer care. Nat Rev Clin Oncol. 2019;16(5):312–325. doi:10.1038/s41571-019-0167-7
  • Lin KJ, Glynn RJ, Singer DE, Murphy SN, Lii J, Schneeweiss S. Out-of-system care and recording of patient characteristics critical for comparative effectiveness research. Epidemiology. 2018;29(3):356–363. doi:10.1097/EDE.0000000000000794
  • Jemielita T, Li XN, Piperdi B, Zhou W, Burke T, Chen C. Overall survival with second-line pembrolizumab in patients with non-small-cell lung cancer: randomized phase III clinical trial versus propensity-adjusted real-world data. JCO Clin Cancer Inform. 2021;5:56–65. doi:10.1200/CCI.20.00099
  • Jemielita T, Widman L, Fox C, Salomonsson S, Liaw KL, Pettersson A. Replication of oncology randomized trial results using Swedish registry real world-data: a feasibility study. Clin Pharmacol Ther. 2021;110(6):1613–1621. doi:10.1002/cpt.2424
  • Soni PD, Hartman HE, Dess RT, et al. Comparison of population-based observational studies with randomized trials in oncology. J Clin Oncol. 2019;37(14):1209–1216. doi:10.1200/JCO.18.01074
  • Rugo HS, Finn RS, Dieras V, et al. Palbociclib plus letrozole as first-line therapy in estrogen receptor-positive/human epidermal growth factor receptor 2-negative advanced breast cancer with extended follow-up. Breast Cancer Res Treat. 2019;174(3):719–729. doi:10.1007/s10549-018-05125-4
  • Llombart-Cussac A, Perez-Garcia JM, Bellet M, et al. Fulvestrant-palbociclib vs letrozole-palbociclib as initial therapy for endocrine-sensitive, hormone receptor–positive, ERBB2 -negative advanced breast cancer. JAMA Oncol. 2021;7:1791. doi:10.1001/jamaoncol.2021.4301
  • Rubin DB. Inference and missing data. Biometrika. 1976;63(3):581–592. doi:10.1093/biomet/63.3.581
  • Azur MJ, Stuart EA, Frangakis C, Leaf PJ. Multiple imputation by chained equations: what is it and how does it work? Int J Methods Psychiatr Res. 2011;20(1):40–49. doi:10.1002/mpr.329
  • Rubin DB. Multiple Imputation after 18+ Years. J Am Stat Assoc. 1996;91(434):473–489. doi:10.1080/01621459.1996.10476908
  • Rubin DB. Multiple Imputation for Nonresponse in Surveys. Hoboken, NJ: Wiley-Interscience; 2004.
  • Cox DR. Regression models and life-tables. J R Stat Soc Ser B Methodol. 1972;34:187–220.
  • Brookhart MA, Schneeweiss S, Rothman KJ, Glynn RJ, Avorn J, Sturmer T. Variable selection for propensity score models. Am J Epidemiol. 2006;163(12):1149–1156. doi:10.1093/aje/kwj149
  • Zhu Y, Hubbard RA, Chubak J, Roy J, Mitra N. Core concepts in pharmacoepidemiology: violations of the positivity assumption in the causal analysis of observational data: consequences and statistical approaches. Pharmacoepidemiol Drug Saf. 2021;30(11):1471–1485. doi:10.1002/pds.5338
  • Austin PC. Balance diagnostics for comparing the distribution of baseline covariates between treatment groups in propensity-score matched samples. Stat Med. 2009;28(25):3083–3107. doi:10.1002/sim.3697
  • Franklin JM, Pawar A, Martin D, et al. Nonrandomized real-world evidence to support regulatory decision making: process for a randomized trial replication project. Clin Pharmacol Ther. 2020;107(4):817–826. doi:10.1002/cpt.1633
  • White IR, Carlin JB. Bias and efficiency of multiple imputation compared with complete-case analysis for missing covariate values. Stat Med. 2010;29(28):2920–2931. doi:10.1002/sim.3944
  • Schulte PJ, Mascha EJ. Propensity score methods: theory and practice for anesthesia research. Anesth Analg. 2018;127(4):1074–1084. doi:10.1213/ANE.0000000000002920
  • Sjolander A, Dahlqwist E, Zetterqvist J. A note on the noncollapsibility of rate differences and rate ratios. Epidemiology. 2016;27(3):356–359. doi:10.1097/EDE.0000000000000433
  • Hernan MA, Hernandez-Diaz S, Robins JM. A structural approach to selection bias. Epidemiology. 2004;15(5):615–625. doi:10.1097/01.ede.0000135174.63482.43
  • Young JG, Stensrud MJ, Tchetgen Tchetgen EJ, Hernan MA. A causal framework for classical statistical estimands in failure-time settings with competing events. Stat Med. 2020;39(8):1199–1236. doi:10.1002/sim.8471
  • Mathur MB, Ding P, Riddell CA, VanderWeele TJ. Web site and R package for computing E-values. Epidemiology. 2018;29(5):e45–e47. doi:10.1097/EDE.0000000000000864
  • VanderWeele TJ, Ding P. Sensitivity analysis in observational research: introducing the E-value. Ann Intern Med. 2017;167(4):268–274. doi:10.7326/M16-2607