免疫疗法与癌症治疗价值框架定义的持久生存的关联。
Association of Immunotherapy With Durable Survival as Defined by Value Frameworks for Cancer Care.
机构信息
Department of Management, Health System Management Program, Bar Ilan University, Ramat Gan, Israel.
Coller School of Management, Tel Aviv University, Tel Aviv, Israel.
出版信息
JAMA Oncol. 2018 Mar 1;4(3):326-332. doi: 10.1001/jamaoncol.2017.4445.
IMPORTANCE
Modern immuno-oncology agents have generated great excitement because of their potential to provide durable survival for some patients. However, there is concern regarding the cost of cancer care, and multiple frameworks have been developed to assess value. The American Society of Clinical Oncology (ASCO) framework awards bonus points if substantial durable survival is demonstrated.
OBJECTIVE
To assess whether modern immuno-oncology agents reach defined efficacy thresholds in value frameworks.
DESIGN, SETTING, AND PARTICIPANTS: In this analysis, all US Food and Drug Administration (FDA) approvals for immuno-oncology agents between March 2011 and August 2017 were reviewed. Data required for the ASCO framework were collected, specifically improvement in proportion of patients alive with the test regimen and survival rate with standard treatment.
MAIN OUTCOMES AND MEASURES
Awarding of bonus points for durable survival based on the ASCO criteria.
RESULTS
Twenty-three metastatic indications for 6 immuno-oncology agents (ipilimumab, pembrolizumab, nivolumab, atezolizumab, avelumab, and durvalumab) were approved by the FDA from March 2011 to August 2017. Ten (43%) of the approvals were based on survival end points, while 13 (57%) were based on response rates. Only 3 drug indications fulfilled the threshold defined for the survival rate of patients receiving standard care (minimum 20%). Nine indications achieved the required level of improvement in proportion to patients alive in the test regimen compared with the standard (above 50%). There was overlap between these 2 criteria for 3 drug indications, allowing them to gain the durable survival bonus points awarded by the ASCO framework.
CONCLUSIONS AND RELEVANCE
Durable survival and response rates of modern immuno-oncology agents are rarely recognized as significant by current oncology value frameworks. This may be due to insufficient demonstration of efficacy of such agents or inappropriately calibrated value frameworks.
重要性
由于现代免疫肿瘤药物具有为一些患者提供持久生存的潜力，因此引起了极大的关注。但是，人们对癌症治疗的成本表示担忧，并且已经开发出多种框架来评估价值。如果证明具有实质性的持久生存，美国临床肿瘤学会（ASCO）框架会给予额外的分数。
目的
评估现代免疫肿瘤药物是否在价值框架中达到了定义的疗效阈值。
设计、环境和参与者：在这项分析中，审查了 2011 年 3 月至 2017 年 8 月期间所有美国食品和药物管理局（FDA）批准的免疫肿瘤药物。收集了 ASCO 框架所需的数据，特别是测试方案中存活患者比例和标准治疗生存率的改善。
主要结果和测量指标
根据 ASCO 标准授予持久生存的额外分数。
结果
从 2011 年 3 月至 2017 年 8 月，FDA 批准了 6 种免疫肿瘤药物（依匹单抗、帕博利珠单抗、纳武单抗、阿替利珠单抗、avelumab 和度伐单抗）用于 23 种转移性适应症。其中 10 项（43％）批准是基于生存终点，而 13 项（57％）是基于反应率。只有 3 种药物适应症符合接受标准治疗的患者生存率标准（最低 20％）。9 种适应症与标准相比，在测试方案中存活的患者比例有了所需的改善（超过 50％）。对于 3 种药物适应症，这两个标准之间存在重叠，使它们能够获得 ASCO 框架授予的持久生存奖励积分。
结论和相关性
现代免疫肿瘤药物的持久生存和反应率很少被当前肿瘤学价值框架认为具有重要意义。这可能是由于这些药物的疗效证明不足，或者是由于价值框架校准不当。
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