statistical analysis of clinical trial data for resource allocation decisions

Randomised controlled trials (RCTs) are a major source of individual patient level data (IPD) on the clinical outcomes, costs and other measures of health consequences associated with alternative healthcare interventions. These data are typically used to establish the ‘value for money’ of healthcare technologies. While a clear policy framework to guide the above assessment exists, the analysis of RCT data to inform decision making requires the adoption of a specific quantitative methodological framework. This article describes the use of RCTs for cost-effectiveness analysis, discusses the major statistical issues and possible solutions, and outlines recent research developments in this area.