Design and Analytic Considerations in Determining the Cost-Effectiveness of Early Intervention in Asthma from a Multinational Clinical Trial

Demand for economic and outcomes data in support of drug formulary listing in private and government-sponsored health programs has led to fundamental changes in drug development. In part as a response to these pressures, the pharmaceutical industry has begun to include economic and quality-of-life endpoints in clinical trials with the hope of providing information to answer health policy questions on the economic value of its products. Here, the design and health economic techniques that will be used to analyze the START (inhaled Steroid Treatment As Regular Therapy in early asthma) study—a multinational (31 countries), randomized, placebo-controlled trial of 7240 patients with mild asthma over 3 years—will be presented. START compares the effect of once-daily administration of an inhaled glucocorticosteroid (Pulmicort Turbuhaler®) to conventional therapy in the management of newly diagnosed asthma, for which the use of this therapy is currently not the standard. The START study will examine both clinical effectiveness (measured as symptom-free days) and asthma-related costs for both treatment arms, aggregated for all patients across all countries. We believe that presenting the analytical plan prior to disseminating the results is an important way of increasing the credibility of economic evaluations. However, using clinical trials for collecting economic data poses several challenges, and the methods for conducting such evaluations are being developed. This paper will present and discuss several methodological options and the current state of the art for conducting economic evaluations alongside multinational clinical trials. Control Clin Trials 2001; 22:420–437