Performing cost-effectiveness analysis by integrating randomized trial data with a comprehensive decision model: Application to treatment of acute ischemic stroke

A recent national panel on cost-effectiveness in health and medicine has re commended that cost-effectiveness analysis (CEA) of randomized controlled t rials (RCTs) should reflect the effect of treatments on long-term outcomes. Because the follow-up period of RCTs tends to be relatively short, long-te rm implications of treatments must be assessed using other sources. We used a comprehensive simulation model of the natural history of stroke to estim ate long-term outcomes after a hypothetical RCT of an acute stroke treatmen t. The RCT generates estimates of short-term quality-adjusted survival and cost and also the pattern of disability at the conclusion of follow-up. The simulation model incorporates the effect of disability on long-term outcom es, thus supporting a comprehensive CEA. Treatments that produce relatively modest improvements in the pattern of outcomes after ischemic stroke are l ikely to be cost-effective. This conclusion was robust to modifying the ass umptions underlying the analysis. More effective treatments in the acute ph ase immediately following stroke would generate significant public health b enefits, even if these treatments have a high price and result in relativel y small reductions in disability. Simulation-based modeling can provide the critical link between a treatment's short-term effects and its long-term i mplications and can thus support comprehensive CEA. J CLIN EPIDEMIOL 52;3:2 59-271, 1999. (C) 1999 Elsevier Science Inc.