Emulation of a Comparative Effectiveness Study of Pembrolizumab and Chemotherapy vs. Chemotherapy for the First-line Treatment of Metastatic Non-small Cell Lung Cancer
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- Aetion, Inc.
- 入组人数
- 1,854
- 试验地点
- 1
- 主要终点
- Overall Survival - Mortality Hazard Ratio
研究概览
简要总结
Investigators are building an empirical evidence base supporting the utility of real-world data through the emulation of randomized controlled trials in the oncology setting. The purpose of this work is to demonstrate whether real-world evidence studies can provide reliable conclusions on treatment effectiveness to inform further applications of real-world data in pharmaceutical product label expansion, post-marketing safety, and other purposes that are complementary to RCTs.
详细描述
This is a non-randomized observational study that is being conducted as a part of the Coalition to Advance Real-World Evidence through Randomized Controlled Trial Emulation (CARE) Initiative, a multi-stakeholder collaboration led by Aetion. The study is intended to emulate the trial listed below/above. Although many features of the trial cannot be directly replicated in real-world data sources, key elements of the study design, including outcomes, exposures, and inclusion/exclusion criteria, were selected to proxy those features from the trial as closely as possible. Investigators assume that the trial provides the reference standard treatment effect estimate and that failure to replicate trial findings is indicative of the inadequacy of the real-world data source for replication for a range of possible reasons and does not provide information on the validity of the original trial finding.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Retrospective
入排标准
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Has evidence in electronic health record indicating a 'lung' tumor site, non-squamous morphology (e.g., adenocarcinoma, large cell, etc), and a stage IV or metastatic disease in the form of diagnosis codes or derived variables from physician notes or linked data from a tumor registry. There is no evidence of other lung cancer types (i.e., mesothelioma, small cell, etc.).
- •Does not have evidence of HGVS codes in health record indicating EGFR or ALK mutations.
- •Has no evidence of use of guideline-recommended systemic cancer therapy* for NSCLC on or after first date associated with stage IV (or metastatic) disease and prior to index treatment.
排除标准
- •Has evidence of squamous cell lung cancer morphology in the form of diagnosis codes or derived variables from physician notes or linked information from a tumor registry.
- •Has evidence of systemic cancer therapy utilization on or after first date associated with stage IV (or metastatic) disease and prior to index treatment. Patient has no evidence of biologic therapy at any time prior to the first record indicating an exposure of interest.
- •Has evidence in medical record or tumor registry of primary malignancies beyond the lung tissue, except basal cell carcinoma of the skin, superficial bladder cancer, squamous cell carcinoma of the skin, in situ cervical cancer, or other in situ cancers.
- •Has evidence of central nervous system (CNS) metastases and/or carcinomatous meningitis in the 2 weeks prior to initiation.
- •Has a record of use of one of the following agents in the 2 years prior to the index date: methotrexate, sulfasalazine, hydroxychloroquine, leflunomide, azathioprine, etanercept, adalimumab, infliximab, certolizumab, golimumab, anakinra, tocilizumab, sarilumumab, abatacept, rituximab, tofacitinib, barivitinib, upadacitinib, mycophenolate.
- •Has at least two records 60 days apart indicating use of one of the following agents in the 3 months prior to the index date: prednisone, prednisolone, methylprednisolone, dexamethasone, hydrocortisone.
- •Has evidence of use of one of the following agents prior to the index date: pembrolizumab, nivolumab, cemiplimab, atezolizumab, durvalumab.
研究组 & 干预措施
Pembrolizumab + Chemotherapy
Exposure Group
干预措施: Pembrolizumab + Pemetrexed-Platinum (Drug)
Chemotherapy
Reference Group
干预措施: Pemetrexed-Platinum (Drug)
结局指标
主要结局
Overall Survival - Mortality Hazard Ratio
时间窗: Through study completion (earliest of 640 days or censoring)
Relative hazard of mortality due to any cause
Overall Survival - Mortality Risk
时间窗: 1 year, year 1
12-month survival probability
次要结局
未报告次要终点