Gomes, M., Latimer, N. orcid.org/0000-0001-5304-5585, Soares, M. et al. (6 more authors) (2022) Target trial emulation for transparent and robust estimation of treatment effects for health technology assessment using real-world data : opportunities and challenges. PharmacoEconomics, 40 (6). pp. 577-586. ISSN 1170-7690
Abstract
Evidence about the relative effects of new treatments is typically collected in randomised controlled trials (RCTs). In many instances, evidence from RCTs falls short of the needs of health technology assessment (HTA). For example, RCTs may not be able to capture longer-term treatment effects, or include all relevant comparators and outcomes required for HTA purposes. Information routinely collected about patients and the care they receive have been increasingly used to complement RCT evidence on treatment effects. However, such routine (or real-world) data are not collected for research purposes, so investigators have little control over the way patients are selected into the study or allocated to the different treatment groups, introducing biases for example due to selection or confounding. A promising approach to minimise common biases in non-randomised studies that use real-world data (RWD) is to apply design principles from RCTs. This approach, known as ‘target trial emulation’ (TTE), involves (1) developing the protocol with respect to core study design and analysis components of the hypothetical RCT that would answer the question of interest, and (2) applying this protocol to the RWD so that it mimics the data that would have been gathered for the RCT. By making the ‘target trial’ explicit, TTE helps avoid common design flaws and methodological pitfalls in the analysis of non-randomised studies, keeping each step transparent and accessible. It provides a coherent framework that embeds existing analytical methods to minimise confounding and helps identify potential limitations of RWD and the extent to which these affect the HTA decision. This paper provides a broad overview of TTE and discusses the opportunities and challenges of using this approach in HTA. We describe the basic principles of trial emulation, outline some areas where TTE using RWD can help complement RCT evidence in HTA, identify potential barriers to its adoption in the HTA setting and highlight some priorities for future work.
Metadata
Item Type: | Article |
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Authors/Creators: |
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Copyright, Publisher and Additional Information: | © 2022 The Author(s). This is an author-produced version of a paper subsequently published in PharmacoEconomics. Uploaded in accordance with the publisher's self-archiving policy. |
Dates: |
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Institution: | The University of Sheffield |
Academic Units: | The University of Sheffield > Faculty of Medicine, Dentistry and Health (Sheffield) > School of Health and Related Research (Sheffield) > ScHARR - Sheffield Centre for Health and Related Research |
Funding Information: | Funder Grant number Yorkshire Cancer Research S406NL |
Depositing User: | Symplectic Sheffield |
Date Deposited: | 04 Apr 2022 12:57 |
Last Modified: | 25 Mar 2023 01:13 |
Status: | Published |
Publisher: | Springer Nature |
Refereed: | Yes |
Identification Number: | 10.1007/s40273-022-01141-x |
Related URLs: | |
Open Archives Initiative ID (OAI ID): | oai:eprints.whiterose.ac.uk:185430 |