Long-Term Cost-Effectiveness of Pioglitazone versus Placebo in Addition to Existing Diabetes Treatment: A US Analysis Based on PROactive

Objective: To estimate the long-term cost-effectiveness of adding pioglitazone versus placebo to standard treatment in high-risk patients with type 2 diabetes. Methods: The validated CORE Diabetes Model was modified to project long-term clinical and cost outcomes associated with pioglitazone versus placebo, based on results from PROactive. The model retained basic structure and functionality, with interdependent Markov submodels, Monte Carlo simulation and user interface. Adjustments to submodels were made to accommodate the PROactive primary end points. The analysis was from the perspective of a third party US health-care payer perspective, projected over a lifetime horizon using a 3% annual discount. Results: Over a lifetime horizon, addition of pioglitazone was associated with increased life expectancy (0.237 life-years) and quality-adjusted life expectancy (QALE) [0.166 quality-adjusted life-years (QALYs)] versus placebo. Estimated long-term complication rates showed that pioglitazone reduced the number of events versus placebo for most outcomes. Lifetime total direct costs were marginally higher with pioglitazone versus placebo ($272,694 vs. $265,390, difference $7,305). The incremental cost-effectiveness ratio for pioglitazone versus placebo was $44,105 per QALY gained. Probabilistic sensitivity analysis indicated a 55% likelihood that pioglitazone would be considered cost-effective in the United States, with a willingness to pay of $50,000 per QALY gained. Conclusions: The addition of pioglitazone to existing therapy in high-risk patients with type 2 diabetes was projected to improve life expectancy, QALE and complication rates compared with placebo. Addition of pioglitazone was in the range generally considered acceptable. © 2008, International Society for Pharmacoeconomics and Outcomes Research (ISPOR).