Department of Management, Health System Management Program, Bar-Ilan University, Ramat Gan, Israel.
Coller School of Management, Tel Aviv University, Tel Aviv, Israel.
JCO Oncol Pract. 2020 Aug;16(8):e779-e790. doi: 10.1200/JOP.19.00725. Epub 2020 Mar 20.
A unique feature of immuno-oncology agents is the potential for durable survival for a subset of patients; however, this benefit usually cannot not be seen in the early published data used for regulatory approval. Value frameworks developed by ASCO and the European Society for Medical Oncology (ESMO) assess the clinical benefit demonstrated in clinical trials. Proven benefit may change with time as more mature data are available. Our objective was to evaluate the impact of mature data for immuno-oncology agents on ASCO and ESMO scores and to examine the concordance of these frameworks using more mature data.
We reviewed Food and Drug Administration (FDA) approvals for immuno-oncology agents between 2011 and 2017, calculated the ASCO-Net Health Benefit (NHB) score and ESMO-Magnitude of Clinical Benefit Score (MCBS), checked which agents fulfilled the criteria of being rewarded for durable survival, assessed the concordance between models using the Spearman correlation test, and compared the initial results of registration studies with mature follow-up data from the same studies.
The FDA approved 27 solid tumor indications for immuno-oncology agents between 2011 and 2017. The correlation between ASCO-NHB score and ESMO-MCBS was high (0.88). Mature follow-up data were available for 13 of these indications, in which 6 studies were found to have improved in the grade of ASCO and/or ESMO value frameworks, whereas 2 cases were downgraded in the scale.
Despite different approaches, the high concordance between ASCO and ESMO value frameworks indicates that both models reward treatments as beneficial for the same immuno-oncology agents. Mature data with longer follow-up reaffirmed most of the findings found in the evaluation in the initially published registration studies.
免疫肿瘤药物的一个独特特征是，其为一部分患者提供了持久生存的潜力；然而，这种获益通常无法在用于监管批准的早期发布数据中显现。美国临床肿瘤学会（ASCO）和欧洲肿瘤内科学会（ESMO）制定的价值框架评估临床试验中所展示的临床获益。随着更多成熟数据的出现，已证实的获益可能会发生变化。我们的目的是评估免疫肿瘤药物的成熟数据对 ASCO 和 ESMO 评分的影响，并使用更成熟的数据来检验这些框架的一致性。
我们回顾了 2011 年至 2017 年期间 FDA 批准的免疫肿瘤药物，计算了 ASCO-Net 健康获益（NHB）评分和 ESMO 临床获益度量评分（MCBS），检查了哪些药物符合持久生存获益的标准，使用 Spearman 相关检验评估模型之间的一致性，并将注册研究的初始结果与来自同一研究的成熟随访数据进行比较。
2011 年至 2017 年，FDA 批准了 27 种用于实体瘤的免疫肿瘤药物的适应证。ASCO-NHB 评分与 ESMO-MCBS 之间的相关性很高（0.88）。其中 13 个适应证有成熟的随访数据，其中 6 项研究在 ASCO 和/或 ESMO 价值框架中的等级提高，而 2 项研究降级。
尽管方法不同，但 ASCO 和 ESMO 价值框架之间的高度一致性表明，这两个模型都对同样的免疫肿瘤药物的治疗获益进行了奖励。具有更长随访时间的成熟数据证实了最初在已发表的注册研究中评估发现的大多数结果。
