Sacristán J A, Day S J, Navarro O, Ramos J, Hernández J M
Clinical Research Department, Lilly SA, Madrid, Spain.
Ann Pharmacother. 1995 Jul-Aug;29(7-8):719-25. doi: 10.1177/106002809502907-815.
To discuss the calculation and application of confidence intervals in pharmacoeconomic studies.
The increasing frequency with which pharmacoeconomic evaluations are made within clinical trials makes it possible to obtain information on the outputs and costs of an intervention in each patient of a sample under study. This allows the same statistical principles commonly used in clinical trials to be applied to cost or cost-effectiveness data. The methodology described in this article would allow expression of cost-effectiveness ratios in the form of confidence intervals. The calculation of the cost-effectiveness ratio by means of a confidence interval may have important practical consequences, both in decision-making on the choice of 1 intervention versus another and in calculating the size of the sample necessary to identify statistically significant differences, from both clinical and economic points of view.
The complementary use of confidence intervals and sensitivity analysis makes it possible to measure uncertainty related and unrelated to variability in sample data, allowing the decision to adopt 1 technology or another to be based on the most objective information available. Although several ethical and methodologic concerns remain to be addressed, this methodology may contribute to improving the more rational and efficient use of drugs.
探讨药物经济学研究中置信区间的计算与应用。
在临床试验中进行药物经济学评估的频率不断增加，使得获取关于研究样本中每位患者干预措施的产出和成本信息成为可能。这使得通常用于临床试验的相同统计原则能够应用于成本或成本效益数据。本文所述方法能够以置信区间的形式表达成本效益比。通过置信区间计算成本效益比可能会产生重要的实际影响，无论是在选择一种干预措施而非另一种干预措施的决策中，还是在从临床和经济角度计算识别统计学显著差异所需的样本量时。
置信区间和敏感性分析的互补使用能够衡量与样本数据变异性相关和不相关的不确定性，从而使采用一种技术或另一种技术的决策基于可得的最客观信息。尽管仍有若干伦理和方法学问题有待解决，但该方法可能有助于改善药物的更合理和有效使用。
