精确肿瘤学
临床试验
精密医学
医学
药物开发
灵活性(工程)
肿瘤科
仿形(计算机编程)
临床研究设计
个性化医疗
临床肿瘤学
抗癌药
生物信息学
癌症
内科学
医学物理学
药品
计算机科学
药理学
病理
生物
操作系统
统计
数学
作者
Apostolia M. Tsimberidou,Peter Müller,Yuan Ji
标识
DOI:10.1016/j.semcancer.2020.09.006
摘要
Genomic profiling technologies have enabled the development of targeted therapies designed to target specific biomarkers and molecular pathways involved in the pathophysiology of tumor initiation, metastasis, and drug resistance. In recent years, clinical trials with innovative design focus on the development of novel agents based on specific patient molecular alterations or other tumor characteristics and include patients with heterogenous tumor types. Precision oncology studies with innovative design associated with novel dose-finding approaches and data analysis focusing on subgroups of patients are characteristic of master protocols. Real-world data, patient-reported outcomes, and N-of-1 trials enhance the knowledge base of evidence to deliver personalized treatment to patients. Master protocols accelerate drug development by enabling simultaneous multiple sub-studies that match the patient's tumor molecular profile with experimental treatment arms. However, the increased flexibility of precision oncology trials is often associated with small subpopulations of patients, which may be underpowered to draw statistically robust conclusions. Despite their limitations, innovative clinical trials continue to rapidly translate the emerging discoveries of novel drugs into unprecedented clinical outcomes in patients with cancer and to accelerate the implementation of precision oncology.
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