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Andreas Markoulidakis

Publications and source records attributed to Andreas Markoulidakis.

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Multi-Parameter Estimation of Prevalence (MPEP): A Bayesian modelling approach to estimate the prevalence of opioid dependence

Estimating the number of the number of people from hidden and/or marginalised populations - such as people dependent on opioids or cocaine - is important to guide policy decisions and provision of harm reduction services. Methods such as capture-recapture are widely used, but rely on assumptions that are often violated and not feasible in specific applications. We describe a Bayesian modelling approach called Multi-Parameter Estimation of Prevalence (MPEP). The MPEP approach leverages routinely collected administrative data, starting from a large baseline cohort of individuals from the population of interest and linked events, to estimate the full size of the target population. When multiple event types are included, the approach enables checking of the consistency of evidence about prevalence from different event types. Additional evidence can be incorporated where inconsistencies are identified. In this article, we summarize the general framework of MPEP, with focus on the most recent version, with improved computational efficiency (implemented in STAN). We also explore several extensions to the model that help us understand the sensitivity of the results to modelling assumptions or identify potential sources of bias. We demonstrate the MPEP approach through a case study estimating the prevalence of opioid dependence in Scotland each year from 2014 to 2022.

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CoBWeb: a user-friendly web application to estimate causal treatment effects from observational data using multiple algorithms

Background/aims: While randomized controlled trials are the gold standard for measuring causal effects, robust conclusions about causal relationships can be obtained using data from observational studies if proper statistical techniques are used to account for the imbalance of pretreatment confounders across groups. Propensity score (PS) and balance weighting are useful techniques that aim to reduce the observed imbalances between treatment groups by weighting the groups to be as similar as possible with respect to observed confounders. Methods: We have created CoBWeb, a free and easy-to-use web application for the estimation of causal treatment effects from observational data, using PS and balancing weights to control for confounding bias. CoBWeb uses multiple algorithms to estimate the PS and balancing weights, to allow for more flexible relations between the treatment indicator and the observed confounders (as different algorithms make different (or no) assumptions about the structural relationship between the treatment covariate and the confounders). The optimal algorithm can be chosen by selecting the one that achieves the best trade-off between balance and effective sample size. Results: CoBWeb follows all the key steps required for robust estimation of the causal treatment effect from observational study data and includes sensitivity analysis of the potential impact of unobserved confounders. We illustrate the practical use of the app using a dataset derived from a study of an intervention for adolescents with substance use disorder, which is available for users within the app environment. Conclusion: CoBWeb is intended to enable non-specialists to understand and apply all the key steps required to perform robust estimation of causal treatment effects using observational data.

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How balance and sample size impact bias in the estimation of causal treatment effects: A simulation study

Observational studies are often used to understand relationships between exposures and outcomes. They do not, however, allow conclusions about causal relationships to be drawn unless statistical techniques are used to account for the imbalance of confounders across exposure groups. Propensity score and balance weighting (PSBW) are useful techniques that aim to reduce the imbalances between exposure groups by weighting the groups to look alike on the observed confounders. Despite the plethora of available methods to estimate PSBW, there is little guidance on what one defines as adequate balance, and unbiased and robust estimation of the causal treatment effect is not guaranteed unless several conditions hold. Accurate inference requires that 1. the treatment allocation mechanism is known, 2. the relationship between the baseline covariates and the outcome is known, 3. adequate balance of baseline covariates is achieved post-weighting, 4. a proper set of covariates to control for confounding bias is known, and 5. a large enough sample size is available. In this article, we use simulated data of various sizes to investigate the influence of these five factors on statistical inference. Our findings provide evidence that the maximum Kolmogorov- Smirnov statistic is the proper statistical measure to assess balance on the baseline covariates, in contrast to the mean standardised mean difference used in many applications, and 0.1 is a suitable threshold to consider as acceptable balance. Finally, we recommend that 60-80 observations, per confounder per treatment group, are required to obtain a reliable and unbiased estimation of the causal treatment effect.

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A tutorial comparing different covariate balancing methods with an application evaluating the causal effects of substance use treatment programs for adolescents

Randomized controlled trials are the gold standard for measuring causal effects. However, they are often not always feasible, and causal treatment effects must be estimated from observational data. Observational studies do not allow robust conclusions about causal relationships unless statistical techniques account for the imbalance of pretreatment confounders across groups while key assumptions hold. Propensity score and balance weighting (PSBW) are useful techniques that aim to reduce the imbalances between treatment groups by weighting the groups to look alike on the observed confounders. There are many methods available to estimate PSBW. However, it is unclear a priori which will achieve the best trade-off between covariate balance and effective sample size. Moreover, it is critical to assess the validity of key assumptions required for robust estimation of the needed treatment effects, including the overlap and no unmeasured confounding assumptions. We present a step-by-step guide to covariate balancing strategies, including how to evaluate overlap, obtain estimates of PSBW, check for covariate balance, and assess sensitivity to unobserved confounding. We compare the performance of several estimation methods using a case study examining the relative effectiveness of substance use treatment programs and provide a user-friendly web application that can implement the proposed steps.

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