Do health economic evaluations using observational data provide reliable assessment of treatment effects?

Abstract

Economic evaluation in modern health care systems is seen as a transparent scientific framework that can be used to advance progress towards improvements in population health at the best possible value. Despite the perceived superiority that trial-based studies have in terms of internal validity, economic evaluations often employ observational data. In this review, the interface between econometrics and economic evaluation is explored, with emphasis placed on highlighting methodological issues relating to the evaluation of cost-effectiveness within a bivariate framework. Studies that satisfied the eligibility criteria exemplified the use of matching, regression analysis, propensity scores, instrumental variables, as well as difference-in-differences approaches. All studies were reviewed and critically appraised using a structured template. The findings suggest that although state-of-the-art econometric methods have the potential to provide evidence on the causal effects of clinical and policy interventions, their application in economic evaluation is subject to a number of limitations. These range from no credible assessment of key assumptions and scarce evidence regarding the relative performance of different methods, to lack of reporting of important study elements, such as a summary outcome measure and its associated sampling uncertainty. Further research is required to better understand the ways in which observational data should be analysed in the context of the economic evaluation framework.

The possibilities of day surgery system development within the health policy in Slovakia

“…It is an exact definition of patients who are suitable for treatment in the DS mode with a high emphasis on the DS workplace outside the hospital facility [ 35 - 37 ]. Consequently, there is a necessity of analyses realization that will focus on revelation of risk groups of patients in order to correctly localize a realization of the given procedure type [ 38 ]. This is connected with a determination of clearly defined requirements of personal and technical equipment of given workplaces.…”

Conducting Value for Money Analyses for Non-randomised Interventional Studies Including Service Evaluations: An Educational Review with Recommendations

“…Internationally, few decision makers have the available finances to produce evidence such as the National Institute for Health Research (NIHR) as the largest national funder of clinical research in Europe [22]. As such, there has been much international interest in alternatives to RCTs to produce an evidence base including related to VfM [23].…”

“…When economic evaluations are conducted as part of non-randomised study designs, the need to account for the non-randomised nature of the data is not always recognised [23]. There are suggested statistical methods/guidance to mitigate confounding and bias using observational [90] and 'real-world' data [91].…”

Cost-effectiveness analysis of pneumococcal conjugate vaccines in preventing pneumonia in Peruvian children

“…We used information from the epidemiological surveillance in Uruguay, a country that had a similar pneumococcal serotypes distribution to Peru in the PCV7 pre-vaccination period [43,44]; thus, we extrapolated the direct effect by standardized international methods [24,[46][47][48]. However, observational designs are of high relevance and utmost importance in CEA [59,60]. The second limitation was our use of only pneumonia as the outcome, to the exclusion of pneumococcal meningitis, sepsis, and otitis media; this was because available evidence is still limited and the record systems for these diseases in Peru are scarce.…”