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Projecting effectiveness after ending a randomized controlled trial: a two-state Markov microsimulation model

Part of: Special Collection - Methods

Published online by Cambridge University Press:  03 August 2020

Fei Yuan Open the ORCID record for Fei Yuan [Opens in a new window] ,
Shrikant I. Bangdiwala Open the ORCID record for Shrikant I. Bangdiwala [Opens in a new window] ,
Wesley Tong Open the ORCID record for Wesley Tong [Opens in a new window]  and
Andre Lamy Open the ORCID record for Andre Lamy [Opens in a new window]
Fei Yuan*
Affiliation:
Population Health Research Institute, DBCVSRI, 20 Copeland Avenue, Hamilton, ONL8L 2X2, Canada
Shrikant I. Bangdiwala
Affiliation:
Population Health Research Institute, DBCVSRI, 20 Copeland Avenue, Hamilton, ONL8L 2X2, Canada Department of Health Research Methods, Evidence and Impact, McMaster University, 1280 Main St W, Hamilton, ON L8S 4L8, Canada
Wesley Tong
Affiliation:
Population Health Research Institute, DBCVSRI, 20 Copeland Avenue, Hamilton, ONL8L 2X2, Canada
Andre Lamy
Affiliation:
Population Health Research Institute, DBCVSRI, 20 Copeland Avenue, Hamilton, ONL8L 2X2, Canada Department of Health Research Methods, Evidence and Impact, McMaster University, 1280 Main St W, Hamilton, ON L8S 4L8, Canada Hamilton Health Sciences, 237 Barton St. East, Hamilton, ONL8L 2X2, Canada
*
Author for correspondence: Fei Yuan, E-mail: yuanf@phri.ca; yuanfeifei@gmail.com
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Abstract

Objective

To investigate the behavior of restricted mean survival time (RMST) and designs of a two-state Markov microsimulation model through a 2 × 4 × 2 full factorial experiment.

Method

By projecting patient-wise 15-year-post-trial survival, we estimated life-year-gained between an intervention and a control group using data from the Cardiovascular Outcomes for People Using Anticoagulation Strategies Study (COMPASS). Projections considered either in-trial events or post-trial medications. They were compared based on three factors: (i) choice of probability of death, (ii) lengths of cycle, and (iii) usage of half-a-cycle age correction. Three-way analysis of variance and post-hoc Tukey's Honest Significant Difference test compared means among factors.

Results

When both in-trial events and post-trial study medications were considered, monthly, quarterly, or semiannually were not different from one other in projected life-year-gained. However, the annual one was different from the others: mean and 95 percent confidence interval 252.2 (190.5–313.9) days monthly, 251.8 (192.0–311.6) quarterly, 249.1 (189.7–308.5) semiannually, and 240.8 (178.5–303.1) annually. The other two factors also impacted life-year-gained: background probability (269.1 [260.3–277.9] days projected with REACH-based-probabilities, 227.7 [212.6–242.8] with a USA life table); half-a-cycle age correction (245.5 [199.0–292] with correction and 251.4 [209.1–293.7] without correction). When not considering post-trial medications, only the choice of probability of death appeared to impact life-year-gained.

Conclusion

For a large trial or cohort, to optimally project life-year-gained, one should consider using (i) annual projections, (ii) life table probabilities, (iii) in-trial events, and (iv) post-trial medication use.

Keywords

StatisticsHealth economics/economic evaluationClinical epidemiologyModelingEpidemiology
Type
Method
Information
International Journal of Technology Assessment in Health Care , Volume 36 , Issue 4 , August 2020 , pp. 317 - 324
Copyright
Copyright © The Author(s), 2020. Published by Cambridge University Press

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Footnotes

Abbreviations: ASA, acetyl-salicylic-acid or aspirin; COMPASS, the Cardiovascular Outcomes for People Using Anticoagulation Strategies Study; CAD, coronary artery diseases; CHD, coronary heart diseases; ICER, incremental cost-effectiveness ratio; a modification of the International Society on Thrombosis and Homeostasis (ISTH) criteria for major bleeding (fatal bleeding excluded); MI, myocardial infarction; PAD, peripheral artery diseases; RMST, restricted mean survival time. This measurement is used to measure survivals of two intervention groups (life expectancy) and the incremental survival (life-year-gained) between intervention groups; REACH registry, Reduction of Atherothrombosis for Continued Health Registry; Riva, rivaroxaban.

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