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 e ndpoints in clinical trials with the hope of providing information to answe r health policy questions on the economic value of its products. Here, the design and health economic techniques that will be used to analyze the STAR T (inhaled Steroid Treatment As Regular Therapy in early asthma) study-a mu ltinational (31 countries), randomized, placebo-controlled trial of 7240 pa tients with mild asthma over 3 years-will be presented. START compares the effect of once-daily administration of an inhaled glucocorticosteroid (Pulm icort Turbuhaler((R))) to conventional therapy in the management of newly d iagnosed asthma, for which the use of this therapy is currently not the sta ndard. The START study will examine both clinical effectiveness (measured a s symptom-free days) and asthma-related costs for both treatment arms, aggr egated for all patients across all countries. We believe that presenting th e analytical plan prior to disseminating the results is an important way of increasing the credibility of economic evaluations. However, using clinica l trials for collecting economic data poses several challenges, and the met hods for conducting such evaluations are being developed. This paper will p resent and discuss several methodological options and the current state of the art for conducting economic evaluations alongside multinational clinica l trials. (C) 2001 Elsevier Science Inc.