Real-World Evidence: A Comparison of the Australian Herceptin Program and Clinical Trials of Trastuzumab for HER2-Positive Metastatic Breast Cancer
Real-World Evidence: A Comparison of the Australian Herceptin Program and Clinical Trials of Trastuzumab for HER2-Positive Metastatic Breast Cancer
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真实世界证据：澳大利亚赫赛汀计划与曲妥珠单抗治疗 HER2 阳性转移性乳腺癌的临床试验的比较
DOI：
10.1007/s40273-016-0411-2
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发表时间：
2016
影响因子：
4.4
通讯作者：
Sallie
中科院分区：
文献类型：
--
作者：
B. Parkinson;R. Viney;M. Haas;S. Goodall;P. Srasuebkul;Sallie
摘要引言估计新药的实际成本效益依赖于了解临床试验数据之间的差异（报销前）
临床实践（报销后）。这对于决策者在审查报销决定、价格和考虑相同条件下的其他药物时非常重要。差异可能来自患者特征的差异，也可能来自新证据的可用性和不断发展的治疗实践。本文探讨这些问题使用的案例study.MethodsIn 2001年，澳大利亚政府资助曲妥珠单抗治疗HER2+转移性乳腺癌通过赫赛汀计划。该项目的管理安排产生了丰富的观察性数据，收集了2001年至2010年期间接受曲妥珠单抗治疗的患者的信息（n = 3830）。该数据集包括患者的特点，分发的药物，医疗服务的使用和死亡日期。结果相比，在临床试验的参与者，患者年龄较大，接受更多的化疗和更广泛的联合化疗。治疗实践与临床试验不同，但也随着时间的推移而变化。例如，越来越多地使用原位杂交检测而不是免疫组织化学检测，以及每周三次而不是每周一次的给药方案。与临床试验相比，接受曲妥珠单抗联合化疗的患者的总生存期通常较长（151.3周，95%CI：142.6，163.4），而接受曲妥珠单抗单药治疗的患者的总生存期较短（94.4周，95%CI：86.4，102.9）。这些研究结果可能是由于不同的相对治疗效果在临床实践中，但也可能是由于一系列其他factor.ConclusionThis分析表明，决策者的挑战，因为新的证据和不断发展的治疗实践中产生的临床试验数据和现实世界的临床实践和结果之间的差距。
AbstractIntroductionEstimating the real-world cost-effectiveness of a new drug relies on understanding the differences between clinical trial data (pre-reimbursement)
and clinical practice (post-reimbursement). This is important for decision makers when reviewing reimbursement decisions, prices, and considering other drugs for the same condition. Differences can arise from differences in patient characteristics, but also from the availability of new evidence and evolving treatment practices. This paper examines these issues using a case study.MethodsIn 2001, the Australian Government funded trastuzumab for the treatment of HER2+ metastatic breast cancer through the Herceptin Program. The administrative arrangements of the Program resulted in rich observational data that captured information about patients treated with trastuzumab between 2001 and 2010 (n = 3830). The dataset included patient characteristics, dispensed medicines, medical service use and date of death.ResultsCompared to participants in the clinical trials, patients were older, received more prior chemotherapies and a broader range of co-administered chemotherapies. Treatment practices differed from the clinical trials, but also changed over time. For example, in situ hybridization testing, rather than immunohistochemistry testing, and a three weekly administration schedule, rather than one weekly, were increasingly used. Compared to the clinical trials, patients administered trastuzumab with a concomitant chemotherapy generally experienced longer overall survival (151.3 weeks, 95 % CI: 142.6, 163.4), while those who received trastuzumab as a monotherapy experienced shorter overall survival (94.4 weeks, 95%CI: 86.4, 102.9). These findings may be due to a differing relative treatment effect in clinical practice, but may also be due to a range of other factors.ConclusionThis analysis demonstrates the challenges for decision makers that arise because new evidence and evolving treatment practices create a gap between clinical trial data and real-world clinical practice and outcomes.
影响因子：
3.9
作者：
Olson, Erin M.;Najita, Julie S.;Sohl, Jessica;Arnaout, Amal;Burstein, Harold J.;Winer, Eric P.;Lin, Nancy U.
通讯作者：
Lin, Nancy U.
影响因子：
56.9
作者：
SLAMON, DJ;CLARK, GM;MCGUIRE, WL
通讯作者：
MCGUIRE, WL