| アブストラクト | Methods have been developed for transporting evidence from randomised controlled trials (RCTs) to target populations. However, these approaches allow only for differences in characteristics observed in the RCT and real-world data (overt heterogeneity). These approaches do not recognise heterogeneity of treatment effects (HTE) according to unmeasured characteristics (essential heterogeneity). We use a target trial design and apply a local instrumental variable (LIV) approach to electronic health records from the Clinical Practice Research Datalink, and examine both forms of heterogeneity in assessing the comparative effectiveness of two second-line treatments for type 2 diabetes mellitus. We first estimate individualised estimates of HTE across the entire target population defined by applying eligibility criteria from national guidelines (n = 13,240) within an overall target trial framework. We define a subpopulation who meet a published RCT's eligibility criteria ('RCT-eligible', n = 6497), and a subpopulation who do not ('RCT-ineligible', n = 6743). We compare average treatment effects for pre-specified subgroups within the RCT-eligible subpopulation, the RCT-ineligible subpopulation, and within the overall target population. We find differences across these subpopulations in the magnitude of subgroup-level treatment effects, but that the direction of estimated effects is stable. Our results highlight that LIV methods can provide useful evidence about treatment effect heterogeneity including for those subpopulations excluded from RCTs. |
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| ジャーナル名 | Health economics |
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| 投稿日 | 2024/9/27 |
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| 投稿者 | Lugo-Palacios, David G; Bidulka, Patrick; O'Neill, Stephen; Carroll, Orlagh; Basu, Anirban; Adler, Amanda; DiazOrdaz, Karla; Briggs, Andrew; Grieve, Richard |
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| 組織名 | Department of Health Services Research and Policy, London School of Hygiene &;Tropical Medicine, London, UK.;Department of Non-Communicable Disease Epidemiology, London School of Hygiene &;The Comparative Health Outcomes, Policy & Economics (CHOICE) Institute,;University of Washington School of Pharmacy, Seattle, Washington, USA.;Diabetes Trials Unit, The Oxford Centre for Diabetes, Endocrinology and;Metabolism, University of Oxford, OCDEM Building Churchill Hospital, Headington,;UK.;Department of Statistical Science, University College London, London, UK. |
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| Pubmed リンク | https://www.ncbi.nlm.nih.gov/pubmed/39327529/ |
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