Cortinovis Diego, Abbate Marida, Bidoli Paolo, Pelizzoni Davide, Canova Stefania
Medical Oncology Unit, S Gerardo Hospital, Monza, Italy.
J Thorac Dis. 2015 Nov;7(11):E541-7. doi: 10.3978/j.issn.2072-1439.2015.11.26.
Lung cancer is the leading cause of cancer death in developed countries. However, in the last few years we observed an important acceleration in drug development due to oncogenic driver tumors discovery. Sharing and putting together preclinical data from benchmark and data from clinical research is the scientific paradigm that allows real breakthrough in clinical practice in this field, but only a few targeted agents are worthy and practice changing. The clinical research and proper use of statistical methodology are the pillars to continue to achieve important goals like improvement of overall survival. A good medical oncologist should be able to critically read a scientific paper and move from the observed outcomes into clinical perspective. Despite clinical improvements, sometimes the union of promising targeted agents and optimistic expectations misrepresent the reality and the value of clinical research. In this article, we try to analyze the meaning of statistical assumptions from clinical trials, especially in lung cancer, through a critical review of the concept of value-based medicine. We also attempt to give the reader some practical tools to weigh scientific value of literature reports.
肺癌是发达国家癌症死亡的主要原因。然而，在过去几年中，由于致癌驱动肿瘤的发现，我们观察到药物开发有了显著加速。将基准临床前数据与临床研究数据共享并整合起来，是实现该领域临床实践真正突破的科学范式，但只有少数靶向药物是有价值且能改变实践的。临床研究和正确使用统计方法是继续实现改善总生存期等重要目标的支柱。优秀的肿瘤内科医生应该能够批判性地阅读科学论文，并从观察到的结果转向临床视角。尽管有临床改善，但有时有前景的靶向药物与乐观期望的结合会歪曲临床研究的现实和价值。在本文中，我们试图通过对基于价值的医学概念进行批判性回顾，分析临床试验中统计假设的意义，尤其是在肺癌方面。我们还试图为读者提供一些实用工具，以权衡文献报告的科学价值。
