Probabilistic Sensitivity Analysis in Health Economics

Health economic evaluations have recently built upon more advanced statistical decision-theoretic foundations and nowadays it is officially required that uncertainty about both parameters and observable variables be taken into account thoroughly, increasingly often by means of Bayesian methods. Among these, Probabilistic Sensitivity Analysis (PSA) has assumed a predominant role and Cost Effectiveness Acceptability Curves (CEACs) are established as the most important tool. The objective of this paper is to review the problem of health economic assessment from the standpoint of Bayesian statistical decision theory with particular attention to the philosophy underlying the procedures for sensitivity analysis. We advocate here the use of an integrated vision that is based on the value of information analysis, a procedure that is well grounded in the theory of decision under uncertainty, and criticise the indiscriminate use of other approaches to sensitivity analysis

Essays on Applying Bayesian Data Analysis to Improve Evidence-based Decision-making in Education

This three-article dissertation aims to apply Bayesian data analysis to improve the methodologies that process effectiveness findings, cost information and subjective judgments with the purpose of providing clear, localized guidance for decision makers in educational resource allocation. The first article shows how to use a Bayesian hierarchical model to capture the uncertainty of the effectiveness-cost ratio. The uncertainty information produced by the model may inform the decision makers of the best- and worst-case scenarios of the program efficiency if it is replicated. The second article introduces Bayesian decision theory to address a subset of methodological barriers that hamper the influence of research on educational decision-making, including how to generalize or extrapolate effectiveness and cost information from the evaluation site(s) to a specific context, how to incorporate information from multiple sources, and how to aggregate multiple consequences of an intervention into one framework. The purpose of this article is to generate evidence of program comparison that applies to a specific school facing a decision problem by incorporating the decision-makers' subjective judgements and modeling their specific preference on multiple consequences. The third article proposes a randomized control trial to detect whether principals and practitioners update their beliefs on the effectiveness and cost of educational programs in the light of uncertainty information and localized evidence. Supplemented by a pilot qualitative study that guides decision makers to work on self-defined decision problems, the pilot testing of the experiment provides some evidence on the plausibility of using an experiment to identify the causal impact of research evidence on decision-making

A Bayesian approach to stochastic cost-effectiveness analysis

The aim of this paper is to discuss the use of Bayesian methods in cost-effectiveness analysis (CEA) and the common ground between Bayesian and traditional frequentist approaches. A further aim is to explore the use of the net benefit statistic and its advantages over the incremental cost-effectiveness ratio (ICER) statistic. In particular, the use of cost-effectiveness acceptability curves is examined as a device for presenting the implications of uncertainty in a CEA to decision makers. Although it is argued that the interpretation of such curves as the probability that an intervention is cost-effective given the data requires a Bayesian approach, this should generate no misgivings for the frequentist. Furthermore, cost-effectiveness acceptability curves estimated using the net benefit statistic are exactly equivalent to those estimated from an appropriate analysis of ICERs on the cost-effectiveness plane. The principles examined in this paper are illustrated by application to the cost-effectiveness of blood pressure control in the U.K. Prospective Diabetes Study (UKPDS 40). Due to a lack of good-quality prior information on the cost and effectiveness of blood pressure control in diabetes, a Bayesian analysis assuming an uninformative prior is argued to be most appropriate. This generates exactly the same cost-effectiveness results as a standard frequentist analysis

Analysis of uncertainty in health care cost-effectiveness studies: an introduction to statistical issues and methods

Cost-effectiveness analysis is now an integral part of health technology assessment and addresses the question of whether a new treatment or other health care program offers good value for money. In this paper we introduce the basic framework for decision making with cost-effectiveness data and then review recent developments in statistical methods for analysis of uncertainty when cost-effectiveness estimates are based on observed data from a clinical trial. Although much research has focused on methods for calculating confidence intervals for cost-effectiveness ratios using bootstrapping or Fieller’s method, these calculations can be problematic with a ratio-based statistic where numerator and=or denominator can be zero. We advocate plotting the joint density of cost and effect differences, together with cumulative density plots known as cost-effectiveness acceptability curves (CEACs) to summarize the overall value-for-money of interventions. We also outline the net-benefit formulation of the cost-effectiveness problem and show that it has particular advantages over the standard incremental cost-effectiveness ratio formulation

Thinking outside the box: recent advances in the analysis and presentation of uncertainty in cost-effectiveness studies

As many more clinical trials collect economic information within their study design, so health economics analysts are increasingly working with patient-level data on both costs and effects. In this paper, we review recent advances in the use of statistical methods for economic analysis of information collected alongside clinical trials. In particular, we focus on the handling and presentation of uncertainty, including the importance of estimation rather than hypothesis testing, the use of the net-benefit statistic, and the presentation of cost-effectiveness acceptability curves. We also discuss the appropriate sample size calculations for cost-effectiveness analysis at the design stage of a study. Finally, we outline some of the challenges for future research in this area—particularly in relation to the appropriate use of Bayesian methods and methods for analyzing costs that are typically skewed and often incomplete

Probabilistic analysis of cost-effectiveness models: choosing between treatment strategies for gastroesophageal reflux disease

When choosing between mutually exclusive treatment options, it is common to construct a cost-effectiveness frontier on the cost-effectiveness plane that represents efficient points from among the treatment choices. Treatment options internal to the frontier are considered inefficient and are excluded either by strict dominance or by appealing to the principle of extended dominance. However, when uncertainty is considered, options excluded under the baseline analysis may form part of the cost-effectiveness frontier. By adopting a Bayesian approach, where distributions for model parameters are specified, uncertainty in the decision concerning which treatment option should be implemented is addressed directly. The approach is illustrated using an example from a recently published cost-effectiveness analysis of different possible treatment strategies for gastroesophageal reflux disease.It is argued that probabilistic analyses should be encouraged because they have potential to quantify the strength of evidence in favor of particular treatment choices

Bayesian approaches to technology assessment and decision making

Until the mid-1980s, most economic analyses of healthcare technologies were based on decision theory and used decision-analytic models. The goal was to synthesize all relevant clinical and economic evidence for the purpose of assisting decision makers to efficiently allocate society's scarce resources. This was true of virtually all the early cost-effectiveness evaluations sponsored and/or published by the U.S. Congressional Office of Technology Assessment (OTA) (15), Centers of Disease Control and Prevention (CDC), the National Cancer Institute, other elements of the U.S. Public Health Service, and of healthcare technology assessors in Europe and elsewhere around the world. Methodologists routinely espoused, or at minimum assumed, that these economic analyses were based on decision theory (8;24;25). Since decision theory is rooted in—in fact, an informal application of—Bayesian statistical theory, these analysts were conducting studies to assist healthcare decision making by appealing to a Bayesian rather than a classical, or frequentist, inference approach. But their efforts were not so labeled. Oddly, the statistical training of these decision analysts was invariably classical, not Bayesian. Many were not—and still are not—conversant with Bayesian statistical approaches

Incorporation of genuine prior information in cost-effectiveness analysis of clinical trial data

The Bayesian approach to statistics has been growing rapidly in popularity as an alternative to the frequentist approach in the appraisal of heathcare technologies in clinical trials. Bayesian methods have significant advantages over classical frequentist statistical methods and the presentation of evidence to decision makers. A fundamental feature of a Bayesian analysis is the use of prior information as well as the clinical trial data in the final analysis. However, the incorporation of prior information remains a controversial subject that provides a potential barrier to the acceptance of practical uses of Bayesian methods. The pur pose of this paper is to stimulate a debate on the use of prior information in evidence submitted to decision makers. We discuss the advantages of incorporating genuine prior information in cost-effectiveness analyses of clinical trial data and explore mechanisms to safeguard scientific rigor in the use of such prior information

Estimating the Expected Value of Partial Perfect Information in Health Economic Evaluations using Integrated Nested Laplace Approximation

The Expected Value of Perfect Partial Information (EVPPI) is a decision-theoretic measure of the "cost" of parametric uncertainty in decision making used principally in health economic decision making. Despite this decision-theoretic grounding, the uptake of EVPPI calculations in practice has been slow. This is in part due to the prohibitive computational time required to estimate the EVPPI via Monte Carlo simulations. However, recent developments have demonstrated that the EVPPI can be estimated by non-parametric regression methods, which have significantly decreased the computation time required to approximate the EVPPI. Under certain circumstances, high-dimensional Gaussian Process regression is suggested, but this can still be prohibitively expensive. Applying fast computation methods developed in spatial statistics using Integrated Nested Laplace Approximations (INLA) and projecting from a high-dimensional into a low-dimensional input space allows us to decrease the computation time for fitting these high-dimensional Gaussian Processes, often substantially. We demonstrate that the EVPPI calculated using our method for Gaussian Process regression is in line with the standard Gaussian Process regression method and that despite the apparent methodological complexity of this new method, R functions are available in the package BCEA to implement it simply and efficiently