Taylor R S, Elston J
Peninsula Technology Assessment Group (PenTAG), Peninsula Medical School, Universities of Exeter and Plymouth, UK.
Health Technol Assess. 2009 Jan;13(8):iii, ix-xi, 1-50. doi: 10.3310/hta13080.
To explore the use of surrogate outcomes in Health Technology Assessment (HTA) and provide a basis for guidance for their future use, validation and reporting. This report focuses on the role of surrogate outcomes in cost-effectiveness models (CEMs) within UK HTA Programme reports.
Reports published in the UK HTA Programme monograph series in 2005 and 2006 formed the sampling frame for this study.
Reports were selected on the basis that they addressed a treatment effectiveness/efficacy question, that they included a CEM and that the CEM was primarily based on a surrogate outcome. Reports addressing diagnostic, screening, aetiology, prognostic and methodological questions were excluded. Information was extracted from included reports by two reviewers using a standardised proforma. Surrogate outcomes were assessed according to two published validation frameworks [Journal of the American Medical Association (JAMA) criteria and Outcomes Measures in Rheumatology Clinical Trials (OMERACT) scoring schema]. A narrative synthesis of findings is presented in the form of tabular summaries and illustrative qualitative quotations.
A total of 35 UK HTA reports published in 2005 and 2006 addressed an effectiveness/efficacy question and contained a CEM. Of these, four were found to have based their CEM on a surrogate outcome. All four reports sourced treatment-related changes in surrogate outcomes through a systematic review of the literature; however, there was some variability in the consistency and transparency by which these reports provided evidence of the validation for the surrogate-final outcome relationship. Only one of the reports undertook a systematic review to specifically seek the evidence base for the association between surrogate and final outcomes. Furthermore, this was the only report to provide level 1 surrogate-final outcome validation evidence, i.e. RCT data showing a strong association between the change in surrogate outcome (BPAR) and the change in final outcome (graft survival) at an individual patient level. This report met the JAMA criteria for acceptable evidence of a surrogate. Two reports provided level 2 evidence, i.e. observational study data showing the relationship between the surrogate and final outcome, and one report provided level 3 evidence, i.e. a review of disease natural history. None of the four reports achieved a sufficient score on the OMERACT schema to be judged to have acceptable evidence of a surrogate outcome by its authors.
In this survey of UK HTA reports about 10% of the CEMs therein were explicitly based on surrogate outcomes. The strength of evidence for the surrogate-final outcome relationship, transparency of quantification and exploration of uncertainty of this relationship were found to vary considerably. Recommendations are made for the use of surrogate outcomes in future HTA reports.
探讨替代结局在卫生技术评估（HTA）中的应用，并为其未来的使用、验证和报告提供指导依据。本报告重点关注替代结局在英国HTA计划报告中的成本效益模型（CEM）中的作用。
2005年和2006年英国HTA计划专著系列中发表的报告构成了本研究的抽样框架。
选择的报告需满足以下条件：涉及治疗有效性/疗效问题；包含成本效益模型；且该成本效益模型主要基于替代结局。涉及诊断、筛查、病因、预后和方法学问题的报告被排除。两名评审员使用标准化表格从纳入的报告中提取信息。根据两个已发表的验证框架[《美国医学会杂志》（JAMA）标准和风湿病临床试验结局测量（OMERACT）评分方案]对替代结局进行评估。研究结果以表格摘要和说明性定性引语的形式进行叙述性综合呈现。
2005年和2006年共发表了35份英国HTA报告，涉及有效性/疗效问题并包含成本效益模型。其中，发现有4份报告的成本效益模型基于替代结局。所有4份报告均通过对文献的系统综述获取替代结局中与治疗相关的变化；然而，这些报告在提供替代结局与最终结局关系验证证据的一致性和透明度方面存在一些差异。只有1份报告进行了系统综述，专门寻找替代结局与最终结局关联的证据基础。此外，这是唯一一份提供1级替代结局与最终结局验证证据的报告，即随机对照试验（RCT）数据显示在个体患者层面替代结局（BPAR）的变化与最终结局（移植物存活）的变化之间存在强关联。该报告符合JAMA关于可接受替代结局证据的标准。两份报告提供了2级证据，即观察性研究数据显示替代结局与最终结局之间的关系，一份报告提供了3级证据，即疾病自然史综述。这4份报告在OMERACT方案中均未获得足够分数，其作者认为没有可接受的替代结局证据。
在本次对英国HTA报告的调查中，其中约10%的成本效益模型明确基于替代结局。替代结局与最终结局关系的证据强度、量化的透明度以及对这种关系不确定性的探索差异很大。针对未来HTA报告中替代结局的使用提出了建议。
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