Meltzer Michele, Pizzi Laura T, Jutkowitz Eric
Department of Rheumatology, Jefferson Medical College, Philadelphia, PA 19107, USA.
Evid Based Med. 2012 Aug;17(4):105-8. doi: 10.1136/ebmed-2011-100065. Epub 2012 Feb 18.
The need for comparative effectiveness (CE) data continues to grow, fuelled by market demand as well as health reform. There may be an assumption that new drugs result in improved efficacy compared with the standard of care, therefore warranting premium prices. Gout treatment has recently become controversial, as expensive new drugs enter the market with limited CE data.
The authors reviewed published clinical trials and conducted a cost effectiveness analysis on a new drug (febuxostat) versus the standard (allopurinol) to illustrate the limitations in using these data to inform evidence-based decision-making.
Although febuxostat trials included allopurinol as a comparator, methodological limitations make comparative effectiveness evaluations difficult. However, when available trial data were input to a decision analytic model, the authors found that a significant reduction in febuxostat cost would be required in order for it to dominate allopurinol in cost effectiveness analysis. This case exemplifies the challenges of using clinical trial data in comparative and cost effectiveness analyses.
受市场需求和医疗改革推动，对比较疗效（CE）数据的需求持续增长。可能存在一种假设，即新药与标准治疗相比疗效更佳，因此值得收取更高价格。随着昂贵的新药进入市场且CE数据有限，痛风治疗最近引发了争议。
作者回顾了已发表的临床试验，并对一种新药（非布司他）与标准药物（别嘌醇）进行了成本效益分析，以说明利用这些数据为循证决策提供信息时存在的局限性。
尽管非布司他试验将别嘌醇作为对照，但方法学上的局限性使得比较疗效评估变得困难。然而，当将现有的试验数据输入决策分析模型时，作者发现非布司他要在成本效益分析中优于别嘌醇，就需要大幅降低成本。这个案例体现了在比较和成本效益分析中使用临床试验数据所面临的挑战。
