Estimating treatment effects using parametric models as counter-factual evidence

Randomisation controlled trial are the gold standard for causal inference, however the rapidly increasing development of new treatments and the movement towards personalised medicine mean there is a need to measure efficacy outside of the costly and time-consuming RCT.Here we propose a method of estimating treatment effects using parametric models to act as control against which to compare data from an experimental arm. This allows for treatment effects to be estimated where data are only available from an experimental arm and can be a tool useful in the analysis of observational cohorts or for the design and analysis of RCTs.We develop this approach using Bayesian estimation procedures and compare this approach against other casual inference tools. We then demonstrate how this may be used to estimate the efficacy between two treatment in different RCTs for the analysis of Pancreatic Cancer.It is proposed that with reasonable assumptions, this approach can produce a reliable estimate of efficacy and can have applications in both evaluating currently available data and in the efficient design of future trials.

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Journal
BMC Medical Research Methodology
Published
2025-04-09
DOI
https://doi.org/10.1186/s12874-025-02540-2
Primary Topic
Advanced Causal Inference Techniques
Type
article
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article

Estimating treatment effects using parametric models as counter-factual evidence

Richard Jackson, Daniel H. Palmer, David Hughes, Philip J. Johnson et al.
BMC Medical Research Methodology
Advanced Causal Inference Techniques
article

Estimating treatment effects using parametric models as counter-factual evidence

Richard Jackson, Daniel H. Palmer, David Hughes, Philip J. Johnson, Susanna Dodd, John P. Neoptolemos, Trevor F. Cox, Sarah Berhane, Ruwanthi Kolamunnage‐Dona
article en

Abstract

Randomisation controlled trial are the gold standard for causal inference, however the rapidly increasing development of new treatments and the movement towards personalised medicine mean there is a need to measure efficacy outside of the costly and time-consuming RCT.Here we propose a method of estimating treatment effects using parametric models to act as control against which to compare data from an experimental arm. This allows for treatment effects to be estimated where data are only available from an experimental arm and can be a tool useful in the analysis of observational cohorts or for the design and analysis of RCTs.We develop this approach using Bayesian estimation procedures and compare this approach against other casual inference tools. We then demonstrate how this may be used to estimate the efficacy between two treatment in different RCTs for the analysis of Pancreatic Cancer.It is proposed that with reasonable assumptions, this approach can produce a reliable estimate of efficacy and can have applications in both evaluating currently available data and in the efficient design of future trials.

BMC Medical Research MethodologyVol. 25(1)
University Hospitals Birmingham NHS Foundation Trust (GB), University of Liverpool (GB), Heidelberg University (DE), NIHR Birmingham Biomedical Research Centre (GB), University of Birmingham (GB)
Good health and well-being
Openalex Percentile: Top 93%
Advanced Causal Inference Techniques
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Estimating treatment effects using parametric models as counter-factual evidence — Richard Jackson, Daniel H. Palmer, et al. · BMC Medical Research Methodology (2025) | TGRS Research Map | TGRS