This methodology extends EVSI when future evidence is expected to be observational and affected by confounding, enabling value-of-information analysis in more realistic and feasible data collection scenarios and provides a principled approach to sample size planning when observational evidence is anticipated.
Abstract
Background: The Expected Value of Sample Information (EVSI) quantifies the value of collecting additional evidence to inform a health economic model. Existing EVSI methods typically assume idealized data collection mechanisms, most commonly randomized controlled trials (RCTs). However, in many realistic contexts, additional evidence comes from observational studies, which are subject to confounding or other bias. In this work, we define a methodology to calculate EVSI when additional data are observational and confounded. Methods: First, we define a simulation-based framework in which confounded observational data are generated through Inverse Target Trial Emulation (ITTE), a methodology that generates observational data with controlled levels of confounding starting from initial level data or prior information on population structure. Then, we apply inverse probability weighting (IPW) to obtain an adjusted summary statistic of the data targeting the corresponding randomized estimand. EVSI is finally computed using a regression based approach. Moreover, we propose a computationally efficient method to determine the sample size required to recover the EVSI achievable under an idealized randomized design. Results: We apply the methodology to two health economic models: a Normal Normal conjugate model and a chemotherapy treatment model combining a decision tree and Markov structure. We show that EVSI computed from observational data, even after adjustment, is lower than EVSI based on randomized data. The loss in EVSI increases with the level of induced confounding. Conclusions: This methodology extends EVSI when future evidence is expected to be observational and affected by confounding, enabling value-of-information analysis in more realistic and feasible data collection scenarios. It also provides a principled approach to sample size planning when observational evidence is anticipated.
Importance. Observational studies dominate evidence in cardiovascular outcomes research, yet no widely adopted reporting standard requires authors to disclose how much of an observed association is attributable to systematic error. The negative-control calibration framework of the Observational Health Data Sciences and...
Z. Zeng, J. Wang, H. Zuo et al.· medRxiv· 0 citations
A decision-analytic framework is developed that extends value-of-information methods by incorporating an explicit bias parameter to capture structural distortion in RWE treatment effect estimates and quantifies the probability of RCT-based CED dominance.
Bo-Shen Jiao, Yi-Zhi Liang, Li-Yuan Tao et al.· PharmacoEconomics (Auckland)· 0 citations
Randomized controlled trials (RCTs) identify treatment effects without confounding but are often small, whereas observational studies (OBS) are large but may be confounded. Many estimators combining a small RCT with a large OBS have been developed for the average treatment effect (ATE) and the conditional ATE (CATE). H...
Background: The increasing use of real-world data in economic evaluation raises concerns about the potential for confounding bias. Methods to control for this bias have been developed, but there is still much to be understood about how confounding influences economic evaluations. Objectives: To illustrate the impact of...
Simon LaRue, M. Paulden, Denis Talbot et al.· Medical decision making· 0 citations
Observational data can be used to answer descriptive, predictive, and causal questions. Analysis of observational data can be challenging and may require complex analytical methods to produce meaningful, unbiased results; for example, when addressing confounding in causal studies. In recent years, there has been a grow...
B. Dyer, Mari D. Takashima, Robert S. Ware· Journal of Hospital Medicine· 0 citations
INTRODUCTION
Randomized controlled trials are the gold standard for evaluating interventions but are resource-intensive and impose considerable burden on participants. Historical control designs, which incorporate data from previous trials into new analyses, could reduce sample size requirements, but their practical ap...
Tim J. Schouten, A. Gabrio, Jochen W L Cals et al.· Clinical Trials· 0 citations
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