Delivery of meaningful cancer care: a retrospective cohort study assessing cost and benefit with the ASCO and ESMO frameworks
Delivery of meaningful cancer care: a retrospective cohort study assessing cost and benefit with the ASCO and ESMO frameworks
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DOI：
10.1016/s1470-2045(17)30415-1
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发表时间：
2017-07-01
期刊：
影响因子：
51.1
通讯作者：
Booth, Christopher M.
中科院分区：
文献类型：
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作者：
Del Paggio, Joseph C.;Sullivan, Richard;Booth, Christopher M.
美国临床肿瘤学会（ASCO）和欧洲肿瘤医学学会（ESMO）已经制定了框架，根据毒性和生活质量来量化生存期的增加，以评估癌症治疗的益处。我们将这些框架应用于一组当代随机对照试验，以探索两种方法之间的一致性，并评估治疗效益和成本之间的关系。方法：选取2011年1月1日至2015年12月31日期间发表的非小细胞肺癌、乳腺癌、结直肠癌和胰腺癌全身治疗的所有随机对照试验，并对其摘要和方法进行评估。如果在预先指定的主要或次要结局中报告了有利于实验组的显著差异，则试验有资格纳入我们的队列（次要结局仅在主要结局不显著时进行评估）。我们在相隔3个月的两个时间点用ASCO和ESMO框架评估试验终点，以确认组内可靠性。科恩。根据ASCO评分中位数（作为任意获益阈值）和框架推荐的ESMO阈值计算两个框架之间的一致性。各随机对照试验试验组与对照组每月药品成本差异（即增量药品成本）来源于2016年平均批发价。109项随机对照试验符合纳入条件，其中42项（39%）用于非小细胞肺癌，36项（33%）用于乳腺癌，25项（23%）用于结直肠癌，6项（6%）用于胰腺癌。ASCO评分范围为2 ~ 77；中位评分为25分（IQR 16-35）。41项（38%）试验达到了ESMO框架的获益阈值。两个框架之间的一致性尚可（kappa=0.326）。在有药物成本数据的100项随机对照试验中，ASCO获益评分与每月药物成本增量呈负相关，平方=-0.207；p = 0.039)。达到ESMO获益阈值的治疗比未达到获益阈值的治疗有更低的药物增量成本中位数（2981美元[IQR 320-9059] vs 8621美元[1174- 13930]；p=0.018）。这两个主要的价值关怀框架之间仅存在公平的相关性，框架产出与药品成本之间存在负相关。在可持续的卫生系统中提供最佳的癌症治疗将需要未来的肿瘤学家、研究人员和政策制定者协调药物成本和临床效益之间的脱节。
Background The American Society of Clinical Oncology (ASCO) and the European Society for Medical Oncology (ESMO) have developed frameworks that quantify survival gains in light of toxicity and quality of life to assess the benefits of cancer therapies. We applied these frameworks to a cohort of contemporary randomised controlled trials to explore agreement between the two approaches and to assess the relation between treatment benefit and cost.Methods We identified all randomised controlled trials of systemic therapies in non-small-cell lung cancer, breast cancer, colorectal cancer, and pancreatic cancer published between Jan 1, 2011, and Dec 31, 2015, and assessed their abstracts and methods. Trials were eligible for inclusion in our cohort if significant differences favouring the experimental group in a prespecified primary or secondary outcome were reported (secondary outcomes were assessed only if primary outcomes were not significant). We assessed trial endpoints with the ASCO and ESMO frameworks at two timepoints 3 months apart to confirm intra-rater reliability. Cohen's. statistic was calculated to establish agreement between the two frameworks on the basis of the median ASCO score, which was used as an arbitrary threshold of benefit, and the framework-recommended ESMO threshold. Differences in monthly drug cost between the experimental and control groups of each randomised controlled trial (ie, incremental drug cost) were derived from 2016 average wholesale prices.Findings 109 randomised controlled trials were eligible for inclusion, 42 (39%) in non-small-cell lung cancer, 36 (33%) in breast cancer, 25 (23%) in colorectal cancer, and six (6%) in pancreatic cancer. ASCO scores ranged from 2 to 77; median score was 25 (IQR 16-35). 41 (38%) trials met the benefit thresholds in the ESMO framework. Agreement between the two frameworks was fair (kappa=0.326). Among the 100 randomised controlled trials for which drug costing data were available, ASCO benefit score and monthly incremental drug costs were negatively correlated square=-0.207; p=0.039). Treatments that met ESMO benefit thresholds had a lower median incremental drug cost than did those that did not meet benefit thresholds (US$2981 [IQR 320-9059] vs $8621 [1174-13 930]; p=0.018).Interpretation There is only fair correlation between these two major value care frameworks, and negative correlations between framework outputs and drug costs. Delivery of optimal cancer care in a sustainable health system will necessitate future oncologists, investigators, and policy makers to reconcile the disconnect between drug cost and clinical benefit.