Bansback Nick, Ara Roberta, Karnon Jonathan, Anis Aslam
Centre for Health Evaluation and Outcome Sciences, St Paul's Hospital, Vancouver, British Columbia, Canada.
Pharmacoeconomics. 2008;26(5):395-408. doi: 10.2165/00019053-200826050-00004.
We reviewed the clinical measures used in rheumatoid arthritis (RA) economic evaluations with respect to their relevance and sensitivity to changes in survival, health-related quality of life (HR-QOL) and costs. We compared the measures from the economic perspective and discussed the validity of methods used to extrapolate beyond the trial data. Cost-effectiveness evaluations of disease-modifying antirheumatic drugs in RA were identified by searching MEDLINE, EMBASE, Econlit and NHS EED databases. Studies were retained if they extrapolated beyond randomized controlled trial evidence using relationships between clinical measures, costs and utilities. In the 22 studies identified, clinical severity was measured using the Health Assessment Questionnaire (HAQ) Disability Index, the American College of Rheumatology (ACR) response criteria, the Disease Activity Score (DAS) or a combination of the HAQ and DAS. The HAQ is correlated with mortality, costs and HR-QOL instruments, and several studies used linear relationships to model these associations. However, a polynomial relationship or discrete states may be more appropriate for patients at the extremes of the disease spectrum, and numerous HAQ health states may be required to capture differences in mortality risk. While the ACR response criteria is a more comprehensive measure than the HAQ, it is a relative measure, which creates difficulties when estimating absolute changes in HR-QOL, costs and mortality risk. The evidence base linking DAS scores with HR-QOL instruments, costs and mortality is less robust, possibly due to the comparatively recent development of the measure and the limited number of possible scores (mild/moderate/severe). While there is some evidence of a relationship between DAS scores and costs, the DAS does not capture all aspects of HR-QOL, and no significant relationship has been established with mortality risk. Evidence suggests the HAQ to be the primary clinical measure for use in economic evaluations as it is measured in almost all clinical studies, and is closely correlated to health utilities, mortality and costs. While new developments suggest the sensitivity of health states may be improved by combining the HAQ with measures such as the DAS, further research is required in this area. Further research is also required to explore the advantages in using either continuous or discrete health states.
我们回顾了类风湿关节炎（RA）经济评估中使用的临床指标，考量其与生存、健康相关生活质量（HR-QOL）及成本变化的相关性与敏感性。我们从经济学角度比较了这些指标，并讨论了用于试验数据外推方法的有效性。通过检索MEDLINE、EMBASE、Econlit和NHS EED数据库，确定了RA中改善病情抗风湿药物的成本效益评估。若研究使用临床指标、成本和效用之间的关系进行随机对照试验证据外推，则予以保留。在确定的22项研究中，使用健康评估问卷（HAQ）残疾指数、美国风湿病学会（ACR）反应标准、疾病活动评分（DAS）或HAQ与DAS的组合来衡量临床严重程度。HAQ与死亡率、成本和HR-QOL工具相关，多项研究使用线性关系对这些关联进行建模。然而，对于疾病谱两端的患者，多项式关系或离散状态可能更合适，可能需要大量的HAQ健康状态来捕捉死亡风险差异。虽然ACR反应标准比HAQ更全面，但它是一种相对指标，在估计HR-QOL、成本和死亡风险的绝对变化时会产生困难。将DAS评分与HR-QOL工具、成本和死亡率联系起来的证据基础较薄弱，这可能是由于该指标发展相对较新且可能的评分数量有限（轻度/中度/重度）。虽然有一些证据表明DAS评分与成本之间存在关系，但DAS并未涵盖HR-QOL的所有方面，且与死亡风险未建立显著关系。有证据表明HAQ是经济评估中使用的主要临床指标，因为几乎所有临床研究都对其进行了测量，且与健康效用、死亡率和成本密切相关。虽然新进展表明，将HAQ与DAS等指标结合可能会提高健康状态的敏感性，但该领域仍需进一步研究。还需要进一步研究，以探索使用连续或离散健康状态的优势。
