J Clin Oncol. 1996 Feb;14(2):671-9. doi: 10.1200/JCO.1996.14.2.671.
In 1993, the Health Services Research Committee of the American Society of Clinical Oncology (ASCO) charged an Outcomes Working Group with defining the outcomes of adult and pediatric cancer treatment to be used for technology assessment and development of cancer treatment guidelines. The Working Group defined by consensus outcomes for technology assessment and guideline development, focusing on cancer treatments. The Working Group considered a variety of perspectives on outcomes, including those of patients, physicians, clinical investigators, ASCO, and policy makers. Because ASCO's guidelines will define what constitutes the best treatment and not whether that treatment should be paid for, the Working Group gave higher priority to the clinical and clinical research perspectives than to the health policy perspective. Survival is the most important outcome of cancer treatment. An improvement in at least disease-free survival is a prerequisite for recommending adjuvant therapy. In the case of metastatic cancer, treatment can be recommended even without an improvement in survival, if it improves quality of life. Quality of life includes global quality of life, as well as its physical, psychologic, and social dimensions. To be an outcome of cancer treatment, quality-of-life measures must be sensitive to clinically meaningful changes produced by treatment; evaluations must control for placebo effects and determinants of quality of life not related to cancer or its treatment. Toxicity, both short and long term, is vitally important, with the latter being particularly critical in children. The value of cancer outcomes like tumor response (eg, complete or partial response) and biomarkers (eg, CA-125) for technology assessment and guideline development depends on their ability to predict patient outcomes (survival and quality of life) or to influence decisions about treatment. Complete response is an important outcome when it predicts survival. Progression is important because it signals the need to change or stop treatment. Cost-effectiveness is an especially important outcome to consider when the benefits of treatment are modest or the costs are high. Patient outcomes (eg, survival and quality of life) should receive higher priority than cancer outcomes (eg, response rate), but both types of outcomes are important in technology assessment and guideline development. Multiple outcomes should be considered because no single outcome adequately describes the results of cancer treatment. In general, there is no minimum benefit above which treatments are justified; rather, benefits should be balanced against toxicity and cost.
1993年，美国临床肿瘤学会（ASCO）的卫生服务研究委员会责成一个结果工作组确定用于技术评估和制定癌症治疗指南的成人及儿童癌症治疗结果。该工作组经协商一致确定了用于技术评估和指南制定的结果，重点是癌症治疗。工作组考虑了关于结果的各种观点，包括患者、医生、临床研究人员、ASCO和政策制定者的观点。由于ASCO的指南将界定什么构成最佳治疗，而不是该治疗是否应得到支付，因此工作组将临床和临床研究观点置于比卫生政策观点更高的优先地位。生存是癌症治疗最重要的结果。至少无病生存的改善是推荐辅助治疗的先决条件。对于转移性癌症，如果治疗能改善生活质量，即使生存没有改善也可推荐治疗。生活质量包括总体生活质量及其身体、心理和社会层面。要成为癌症治疗的一个结果，生活质量衡量指标必须对治疗产生的具有临床意义的变化敏感；评估必须控制安慰剂效应以及与癌症或其治疗无关的生活质量决定因素。短期和长期毒性都至关重要，长期毒性在儿童中尤为关键。肿瘤反应（如完全或部分缓解）和生物标志物（如CA - 125）等癌症结果对于技术评估和指南制定的价值取决于它们预测患者结果（生存和生活质量）或影响治疗决策的能力。当完全缓解能预测生存时，它是一个重要结果。疾病进展很重要，因为它表明需要改变或停止治疗。当治疗益处不大或成本很高时，成本效益是一个尤其需要考虑的重要结果。患者结果（如生存和生活质量）应比癌症结果（如缓解率）得到更高的优先考虑，但这两种结果在技术评估和指南制定中都很重要。应考虑多种结果，因为没有单一结果能充分描述癌症治疗的结果。一般来说，不存在一个最低益处水平，超过该水平治疗才合理；相反，应在益处与毒性和成本之间进行权衡。
