Use of real-world evidence in meta-analyses and cost-effectiveness models

Abstract

Real-world evidence (RWE) provides external validity, supplementing and enhancing the randomized controlled trial data with valuable information on patient behaviors and outcomes, turning efficacy and safety results into real-world effectiveness and risks, but the use of RWE is associated with challenges. The objectives of this communication were to (1) summarize all guidance on how to conduct an RWE meta-analysis (MA) and how to develop an RWE cost-effectiveness model, (2) to describe our experience, challenges faced and solutions identified, (3) to provide recommendations on how to conduct such analyses. No formal guidelines on how to conduct an RWE MA or to develop an RWE cost-effectiveness model were identified. Using the context of non-vitamin K antagonist oral anticoagulants in stroke prevention in atrial fibrillation, we conducted an RWE MA, after having identified sources of uncertainty. We then implemented the results in an RWE cost-effectiveness model, defined as a model where all inputs come from RWE, including all parameters related to treatment effect. Based on challenges faced, our first recommendation relates to the necessity of conducting sensitivity analyses, either based on clinical or methodological considerations. Our second recommendation is the need for extensive collaboration with a wide range of experts, during the development of the analyses protocols, the implementation of the analyses and the interpretation of the results. RWE may address a number of gaps related to the treatment effect, and RWE economic evaluations for the treatment effect can provide extremely valuable insights into real-world economic value of interventions. As the increased recognition of the value of RWE could influence health technology assessment decision, and current practices, this communication supports the urgent need of more formal guidelines.     

Richard Whately and the Gospel of Transparency

Whately is a difficult thinker, partly because he is competent in so many disciplines. Joseph Schumpeter, who struggled with Whatley's "elusive" greatness, saw a systematic core in Whately: the force behind Nassau Senior's axiomatics. Whately's contemporaries did not talk of axiomatics, but they did point out that his work depended upon an unusually small number of authorities, that is, Aristotle, Bacon, and Smith. In our interpretation, these foundational sources gave Whately three guiding principles to characterize all human activity: innate sociability, innate self-love, and costly mental activity. Self-love includes a desire to know and a desire to share knowledge. These principles, coupled with a normative principle of fairness, constitute the basis for his science of reciprocal exchange, or catallactics. Violations of fairness motivate his multidimensional reform proposals. For Whately, fairness requires transparency, and the demands of transparency for tractions is literally Gospel.     

Stochastic games with information lag

Two-player zero-sum stochastic games with finite state and action spaces are known to have undiscounted values. We study such games under the assumption that one or both players observe the actions of their opponent after some time-dependent delay. We develop criteria for the rate of growth of the delay such that a player subject to such an information lag can still guarantee himself in the undiscounted game as much as he could have with perfect monitoring. We also demonstrate that the player in the Big Match with the absorbing action subject to information lags that grows too rapidly will not be able to guarantee as much as he could have in the game with perfect monitoring.     

The Geopolitics of Neighbourhood: Jerusalem’s Colonial Space Revisited

This article will focus on an ongoing process of Jerusalem’s contested urban space during the last decade namely the immigration of Palestinians, mostly Israeli citizens, to “satellite neighbourhoods”, i.e. Jerusalem’s colonial neighbourhoods that were constructed after 1967. Theoretically, this paper attempts to discuss neighbourhood planning in contested cities within the framework of geopolitics. In more details, we will focus on the relevance of geopolitics to the study of neighbourhood planning, by which we mean not merely a discussion of international relations and conflict or of the roles of military acts and wars in producing space. Rather, geopolitics refers to the emergence of discourses and forces connected with the technologies of control, patterns of internal migrations by individuals and communities, and the flow of cultures and capital.     

Envy and Altruism: Contrasting Bivariate and Univariate Prospect Preferences

We compare prospect ordering with and without envy and altruism. We find that envy can induce a violation of the univariate first‐degree stochastic dominance (FSD), and thus a violation of the classic expected utility monotonicity axiom. Surprisingly, altruism can also violate FSD preferences. The intuitive explanation of the result in the case of altruism hinges on the sign of the mixed derivative of the bivariate preference: the individual might prefer a certain correlation between her wealth and her peer group's wealth, and is therefore willing to violate FSD as long as the outcomes of the two parties are ordered according to her preferences. When investments are considered, envy and altruism can distort not only preferences but also actual choices.     

When destructive deviance in the workplace becomes a liability: a decisional behavioral model

In recent years destructive deviant behavior in organizations has drawn increasing attention. However, surprisingly little work has been devoted to assessing when the economic value of deviant behavior is tolerable (i.e., the benefits from employing the deviant worker exceed the financial costs of his/her deviance) and when it becomes unbearable (i.e., the financial cost of the deviance exceeds the benefits accrued from his/her employment). The purpose of the present paper is to introduce a mathematical model which may resolve this lacuna. In addition, a simulation was conducted and its results were reported in order to illustrate the usefulness of the proposed mathematical model. In conclusion it is suggested that the present model be further examined empirically in the real workplace.     

Mean-Gini analysis of stochastic externalities: The case of groundwater contamination

The mean-Gini approach is used to analyze stochastic externalities generated by agricultural production. The model addresses the problem of groundwater pollution caused by excessive fertilizer application. Inherent in the mean-Gini approach to expected utility maximization is a two-fold value: the simplicity of the two-parameter mean-variance model and satisfaction of necessary and sufficient conditions for stochastic dominance. Price and quantity policy recommendations to control externalities are formulated based upon the relative assessment of uncertainty by the regulatory authority and the farmers. Using the Gini as a measure of risk allows for the quantification of control policy measures under differentiated risk aversion and multiple sources of pollution. The model shows that when producers underestimate uncertainty, quota policies restricting fertilizer are more efficient than tax policies in reducing groundwater contamination.Work on this paper was carried out when visiting the University of Maryland. Financial aid for the work was provided by the USDA ERS-NRED under a cooperative agreement between the Department of Agricultural and Resource Economics, University of Maryland, and the USDA — Economic Research Service — Natural Resource Economics Division, I am grateful to John Miranowski and Darrell Hueth for that support. I am indebted as well to Lana Shalit, who helped me revise the paper.