Methods Development for Health Technology Assessment

Abstract

Most health care systems internationally have to face the problem of how to allocate limited resources across interventions and programs. This is true for any collectively funded system, whether financed from general taxation, private or social insurance, or a mix of sources. In part this relates to decisions about which medical technologies to fund and, in particular, whether to devote the system’s resources to the numerous new pharmaceuticals, devices, and procedures that become available each year. Health technology assessment (HTA) offers a broad set of tools to support these types of decisions including systematic review, analysis of clinical studies and routine administrative data, health outcome measurement and valuation, and economic evaluation. Jurisdictions differ in the way they use these tools. Some systems, such as those in Germany and the public sector systems in the US, use largely clinical evidence from trials, perhaps augmented with information on the impact of technology adoption on the system’s budget. In contrast, an increasing number of systems, including those in the UK, Canada, and Australia, inform decisions by synthesizing clinical evidence more formally with data on costs and patients’ health outcomes using cost-effectiveness analysis. Despite this variety of ways in which HTA is used to support decision making, there are common issues that the HTA research community has sought to inform. The most appropriate ways in which HTA is used to inform decisions is one such issue. For example, in this issue of Medical Decision Making, Stevens and Longson describe the process by which the National Institute for Health and Clinical Excellence (NICE) uses HTA to support decision making on new pharmaceuticals and other technologies in England and Wales and the challenges faced in using HTA for this purpose. Taking an international perspective, Neumann and others have reported on the extent to which 14 organizations using HTA to support decisions adhere to a series of key principles suggested by the International Working Group for HTA Advancement and organized into 4 broad areas: the structure of the program, methods, processes, and use of HTA in decision making. The review demonstrated considerable variation across organizations in the extent to which these principles were evident in the process and methods used. Another important issue relating to the use of HTA to support decisions across a range of health systems is methodological uncertainty: Is the range of available HTA methods appropriate and fit for the purpose of informing decisions? Because many health systems have sought to use evidence and analysis to support resource allocation, questions have arisen about the adequacy of existing practices and the need for methodologists to sharpen the tools in the HTA toolbox. A key challenge, however, is to characterize the major gaps in existing methods and to set priorities for methods research. To a large extent the scientific community has determined these priorities through the work it undertakes. Indeed, there has been a flow of methods research for HTA, much of it published in this journal. For example, this issue sees contributions on the methods of systematic review with the purpose of speeding up the process of identifying new studies; various methods to estimate parameters relevant to HTA decisions, including the derivation of preference-based measures of health from disease-specific, patient-reported outcome From the University of York, York, UK (MS).