Analysing electronic health records: The benefits of target trial emulation

Bakker, L. J.; Goossens, Lucas; O’Kane, Maurice; Groot, Carin A. Uyl–de; Redekop, W.K.

doi:10.1016/j.hlpt.2021.100545

Cited by 4 publications

(5 citation statements)

References 29 publications

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“…Study strengths include a national surgical registry and reliable assessment of vaccination and infection. The target trial emulation was chosen to reduce certain biases . However, some bias will remain due to unmeasured covariates and a selection bias related to patients who did not undergo surgery due to clinical status.…”

Section: Discussionmentioning

confidence: 99%

“…The target trial emulation was chosen to reduce certain biases. 5 However, some bias will remain due to unmeasured covariates and a selection bias related to patients who did not undergo surgery due to clinical status. Nevertheless, current evidence suggests the decision to proceed should be based on clinical expertise rather than a fixed time interval after infection, consistent with current guidelines.…”

Section: Discussionmentioning

confidence: 99%

See 1 more Smart Citation

Estimated Risk of Adverse Surgical Outcomes Among Patients With Recent COVID-19 Infection Using Target Trial Emulation Methods

O’Brien

Itani²

2023

JAMA Netw Open

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Estimated Risk of Adverse Surgical Outcomes Among Patients With Recent COVID-19 Infection Using Target Trial Emulation Methods

O’Brien

Itani²

2023

JAMA Netw Open

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“…The target trial framework applies the study design principles of RCTs to observational studies which helps to minimise biases that can arise due to study design and analysis choices. [1][2][3][4][5] Target trial emulation has been used successfully in other disease areas to replicate the results from existing RCTs, for example, cardiovascular disease 22 23 and diabetes. 24 The UK CF Registry is cited as an exemplar patient registry in the NICE real-world evidence framework, 25 holds pharmacovigilance credentials and hosts post-authorisation phase IV pharmacovigilance studies.…”

Section: Discussionmentioning

confidence: 99%

“…An alternative is to use observational data to study treatment effects, and it is increasingly recognised that emulating a target trial when using observational data helps to clarify the research question and avoid common biases. [1][2][3][4][5] In this study, we use registry data to emulate a target trial designed to compare multiple treatment strategies on health outcomes in people with cystic fibrosis (CF).…”

Section: Introductionmentioning

confidence: 99%

Emulated trial investigating effects of multiple treatments: estimating combined effects of mucoactive nebulisers in cystic fibrosis using registry data

Granger

Davies

Keogh

2023

Thorax

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IntroductionPeople with cystic fibrosis (CF) are often on multiple long-term treatments, including mucoactive nebulisers. In the UK, the most common mucoactive nebuliser is dornase alfa (DNase). A common therapeutic approach for people already on DNase is to add hypertonic saline (HS). The effects of DNase and HS used alone have been studied in randomised trials, but their effects in combination have not. This study investigates whether, for people already prescribed DNase, adding HS has additional benefit for lung function or use of intravenous antibiotics.MethodsUsing UK CF Registry data from 2007 to 2018, we emulated a target trial. We included people aged 6 years and over who were prescribed DNase without HS for 2 years. We investigated the effects of combinations of DNase and HS over 5 years of follow-up. Inverse-probability-of-treatment weighting was used to control confounding. The period predated triple combination CF transmembrane conductance regulator modulators in routine care.Results4498 individuals were included. At baseline, average age and forced expiratory volume in 1 s (FEV1%) predicted were 21.1 years and 69.7 respectively. During first year of follow-up, 3799 individuals were prescribed DNase alone; 426 added HS; 57 switched to HS alone and 216 were prescribed neither. We found no evidence that adding HS improved FEV1% at 1–5 years, or use of intravenous antibiotics at 1–4 years, compared with DNase alone.ConclusionFor individuals with CF prescribed DNase, we found no evidence that adding HS had an effect on FEV1% or prescription of intravenous antibiotics. Our study illustrates the emulated target trial approach using CF Registry data.

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“…Key elements of the target trial-including the eligibility criteria, treatment strategies, treatment assignment procedures, assignment of time zero, follow-up period, outcomes, causal contrasts and estimands, and analysis plan-are then emulated using RWD. Several published studies have demonstrated how applying the TTE framework can successfully reduce the impact of important biases and promote transparency in observational analyses García-Albéniz et al, 2017;Bakker et al, 2021).…”

Section: Application Of the Target Trial Emulation Framework To Exter...mentioning

confidence: 99%

Application of the target trial emulation framework to external comparator studies

Arnold,

Antunes,

Coles

et al. 2024

Front. Drug Saf. Regul.

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External comparator (EC) studies are increasingly being used to generate evidence that supports the evaluation of emerging pharmacological treatments for regulatory and health technology assessment (HTA) purposes. However, the reliability of evidence generated from EC studies can vary. In this paper, we outline how an existing framework for causal inference, the target trial emulation (TTE) framework, can be appropriately applied to improve the design and analysis of EC studies. Applying the TTE framework involves specifying the protocol of an ideal target trial which would answer the causal question of interest, then emulating its key elements under real-world (RW) settings. We describe each component of the original TTE framework and explain how it can be applied to EC studies, supplemented with practical recommendations. We also highlight special considerations and limitations in applying the TTE framework to EC studies. We describe how the TTE framework can be applied to improve the clarity, transparency, and reliability of evidence generated from EC studies.

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Analysing electronic health records: The benefits of target trial emulation

Cited by 4 publications

References 29 publications

Estimated Risk of Adverse Surgical Outcomes Among Patients With Recent COVID-19 Infection Using Target Trial Emulation Methods

Estimated Risk of Adverse Surgical Outcomes Among Patients With Recent COVID-19 Infection Using Target Trial Emulation Methods

Emulated trial investigating effects of multiple treatments: estimating combined effects of mucoactive nebulisers in cystic fibrosis using registry data

Application of the target trial emulation framework to external comparator studies

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