Real-World Evidence Prediction of a Phase IV Oncology Trial: Comparative Degarelix vs Leuprolide Safety.
Real-World Evidence Prediction of a Phase IV Oncology Trial: Comparative Degarelix vs Leuprolide Safety.
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DOI：
10.1093/jncics/pkac049
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发表时间：
2022-08-10
影响因子：
4.4
通讯作者：
中科院分区：
文献类型：
--
作者：
医学界和监管界对使用真实世界证据(RWE)回答与药物安全性和有效性有关的问题越来越感兴趣，但对有效性的担忧依然存在。进行莱茵研究的原则性方法可能会缓解人们的担忧，并增加对研究结果的信心。这项研究试图使用一种原则性的方法来预测发音试验的结果，以产生RWE。这项倾向性评分匹配的观察性队列研究使用了3个索赔数据库，以比较去加瑞利与亮丙瑞林启动者之间主要不良心血管事件的发生情况。如果患者有前列腺癌和动脉粥样硬化性心血管疾病的病史，就包括在内。如果患者在治疗开始前一年没有连续的数据库登记，在治疗开始前30 天内接受过雄激素剥夺治疗或经历过急性心血管事件，或有QT延长的病史或危险因素，则被排除在外。配对前，符合 研究条件的患者有12例，分别为448例和1969年例，配对后每组患者各1,887例。观察性分析中比较地加瑞利和亮丙瑞林的主要心血管不良事件的结果(危险比=1.35，95%可信区间 = 为0.94~1.93)与随后公布的结果(危险比 = 为1.28，95%可信区间 = 为0.59~2.79)是一致的。这项研究成功地预测了在肿瘤学环境中进行的心血管安全性比较试验的结果。尽管研究结果令人鼓舞，但测量癌症分期和肿瘤进展的局限性代表了在尝试推广基于索赔的RWE是否可以用作可诉讼证据方面的挑战。
Medical and regulatory communities are increasingly interested in the utility of real-world evidence (RWE) for answering questions pertaining to drug safety and effectiveness, but concerns about validity remain. A principled approach to conducting RWE studies may alleviate concerns and increase confidence in findings. This study sought to predict the findings from the PRONOUNCE trial using a principled approach to generating RWE. This propensity score–matched observational cohort study used 3 claims databases to compare the occurrence of major adverse cardiovascular events among initiators of degarelix vs leuprolide. Patients were included if they had a history of prostate cancer and atherosclerotic cardiovascular disease. Patients were excluded if they did not have continuous database enrollment in the year before treatment initiation, were exposed to androgen deprivation therapy or experienced an acute cardiovascular event within 30 days before treatment initiation, or had a history or risk factors of QT prolongation. There were 12 448 leuprolide and 1969 degarelix study-eligible patients before matching, with 1887 in each arm after propensity score matching. The results for major adverse cardiovascular events comparing degarelix with leuprolide in the observational analysis (hazard ratio = 1.35, 95% confidence interval = 0.94 to 1.93) was consistent with the subsequently released PRONOUNCE result (hazard ratio = 1.28, 95% confidence interval = 0.59 to 2.79). This study successfully predicted the result of a comparative cardiovascular safety trial in the oncology setting. Although the findings are encouraging, limitations of measuring cancer stage and tumor progression are representative of challenges in attempting to generalize whether claims-based RWE can be used as actionable evidence.
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DOI：
10.1056/nejmoa1503747
发表时间：
2015-08-20
期刊：
The New England journal of medicine
影响因子：
--
作者：
Sweeney CJ;Chen YH;Carducci M;Liu G;Jarrard DF;Eisenberger M;Wong YN;Hahn N;Kohli M;Cooney MM;Dreicer R;Vogelzang NJ;Picus J;Shevrin D;Hussain M;Garcia JA;DiPaola RS
通讯作者：
DiPaola RS
影响因子：
13.3
作者：
Kim, Seoyoung C.;Solomon, Daniel H.;Schneeweiss, Sebastian
通讯作者：
Schneeweiss, Sebastian
影响因子：
4.8
作者：
Crawford ED;Heidenreich A;Lawrentschuk N;Tombal B;Pompeo ACL;Mendoza-Valdes A;Miller K;Debruyne FMJ;Klotz L
通讯作者：
Klotz L
影响因子：
6.7
作者：
Franklin, Jessica M.;Glynn, Robert J.;Schneeweiss, Sebastian
通讯作者：
Schneeweiss, Sebastian
影响因子：
39
作者：
Fralick, Michael;Kesselheim, Aaron S.;Schneeweiss, Sebastian
通讯作者：
Schneeweiss, Sebastian