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  • Title: From Diagnostic-Therapeutic Pathways to Real-World Data: A Multicenter Prospective Study on Upfront Treatment for EGFR-Positive Non-Small Cell Lung Cancer (MOST Study).
    Author: Pasello G, Vicario G, Zustovich F, Oniga F, Gori S, Rosetti F, Bonetti A, Favaretto A, Toso S, Redelotti R, Santo A, Bernardi D, Giovanis P, Oliani C, Calvetti L, Gatti C, Palazzolo G, Baretta Z, Bortolami A, Bonanno L, Basso M, Menis J, Corte DD, Frega S, Guarneri V, Conte P, Veneto Oncology Network.
    Journal: Oncologist; 2019 Jun; 24(6):e318-e326. PubMed ID: 30846513.
    Abstract:
    INTRODUCTION: Gefitinib, erlotinib, and afatinib represent the approved first-line options for epidermal growth factor receptor (EGFR)-mutant non-small cell lung cancer (NSCLC). Because pivotal trials frequently lack external validity, real-world data may help to depict the diagnostic-therapeutic pathway and treatment outcome in clinical practice. METHODS: MOST is a multicenter observational study promoted by the Veneto Oncology Network, aiming at monitoring the diagnostic-therapeutic pathway of patients with nonsquamous EGFR-mutant NSCLC. We reported treatment outcome in terms of median time to treatment failure (mTTF) and assessed the impact of each agent on the expense of the regional health system, comparing it with a prediction based on the pivotal trials. RESULTS: An EGFR mutation test was performed in 447 enrolled patients, of whom 124 had EGFR mutation and who received gefitinib (n = 69, 55%), erlotinib (n = 33, 27%), or afatinib (n = 22, 18%) as first-line treatment. Because erlotinib was administered within a clinical trial to 15 patients, final analysis was limited to 109 patients. mTTF was 15.3 months, regardless of the type of tyrosine kinase inhibitor (TKI) used. In the MOST study, the budget impact analysis showed a total expense of €3,238,602.17, whereas the cost estimation according to median progression-free survival from pivotal phase III trials was €1,813,557.88. CONCLUSION: Good regional adherence and compliance to the diagnostic-therapeutic pathway defined for patients with nonsquamous NSCLC was shown. mTTF did not significantly differ among the three targeted TKIs. Our budget impact analysis suggests the potential application of real-world data in the process of drug price negotiation. IMPLICATIONS FOR PRACTICE: The MOST study is a real-world data collection reporting a multicenter adherence and compliance to diagnostic-therapeutic pathways defined for patients with epidermal growth factor receptor-mutant non-small cell lung cancer. This represents an essential element of evidence-based medicine, providing information on patients and situations that may be challenging to assess using only data from randomized controlled trials, e.g., turn-around time of diagnostic tests, treatment compliance and persistence, guideline adherence, challenging-to-treat populations, drug safety, comparative effectiveness, and cost effectiveness. This study may be of interest to various stakeholders (patients, clinicians, and payers), providing a meaningful picture of the value of a given therapy in routine clinical practice. 摘要 介绍。吉非替尼、厄洛替尼和阿法替尼是表皮生长因子受体 (EGFR) ‐ 突变非小细胞肺癌 (NSCLC) 的获批一线药选择。由于关键性临床试验经常缺乏外部有效性，因此真实世界数据可能有助于描述临床实践中的诊疗途径和治疗效果。 方法。MOST 是威尼托肿瘤学网络 (Veneto Oncology Network) 推动的一项多中心观察研究，旨在监测非鳞状EGFR突变 NSCLC 患者的诊疗途径。我们根据中位至治疗失败时间 (mTTF) 报告了治疗效果，并评估了每种药剂对区域性卫生系统费用的影响，并将其与基于关键性临床试验的预测进行了对比。 结果。对 447 例入组患者进行 EGFR突变检测，其中 124 例发生EGFR突变，这些患者的一线治疗药物为吉非替尼(n = 69, 55%)、厄洛替尼(n = 33, 27%)或阿法替尼(n = 22, 18%)。由于厄洛替尼在 15 名患者的临床试验中使用，最终分析仅限于 109 名患者。 无论使用何种类型的酪氨酸激酶抑制剂 (TKI)，mTTF 均为 15.3 个月。在 MOST 研究中，预算影响分析显示总费用为 € 3 238 602.17，而根据关键性 III 期试验的中位无进展生存期进行的成本估算为 €1 813 557.88。 结论。结果显示，非鳞状 NSCLC 患者具有良好的区域依从性和对诊疗途径的顺从性。mTTF 在三种目标 TKI 中无显著差异。我们的预算影响分析表明了真实世界数据在药品价格谈判过程中的潜在应用。 实践意义:MOST 研究是一种真实世界数据收集研究，报告了对表皮生长因子受体突变型非小细胞肺癌患者定义的诊疗途径的多中心依从性和顺从性。这是循证医学的一个基本要素，提供关于患者和病情的信息，这些信息可能很难仅使用随机对照试验的数据进行评估，例如诊断性测试的周转时间、治疗依从性和持久性、指南遵从性、治疗人群的挑战性、药物安全性、比较效果和成本效益。该研究可能会引起各种利益相关者(患者、临床医生和支付方)的兴趣，为常规临床实践中给定治疗的价值提供了有意义的图景。
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