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.

基因组图谱技术使靶向治疗的发展成为可能，这些靶向治疗旨在靶向参与肿瘤起始、转移和耐药性的病理生理学的特定生物标志物和分子途径。近年来，具有创新设计的临床试验侧重于基于特定患者分子改变或其他肿瘤特征的新型药物的开发，并包括具有异质肿瘤类型的患者。具有创新设计的精准肿瘤学研究与新的剂量寻找方法和专注于患者亚组的数据分析是主方案的特征。真实世界的数据、患者报告的结果和 N-of-1 试验增强了证据知识库，从而为患者提供个性化治疗。主方案通过同时进行多个子研究来加速药物开发，这些子研究将患者的肿瘤分子谱与实验治疗臂相匹配。然而，精准肿瘤学试验增加的灵活性通常与患者的小亚群有关，这可能不足以得出统计学上可靠的结论。尽管存在局限性，但创新的临床试验继续将新药的新发现迅速转化为癌症患者前所未有的临床结果，并加速精准肿瘤学的实施。这可能不足以得出统计上可靠的结论。尽管存在局限性，但创新的临床试验继续将新药的新发现迅速转化为癌症患者前所未有的临床结果，并加速精准肿瘤学的实施。这可能不足以得出统计上可靠的结论。尽管存在局限性，但创新的临床试验继续将新药的新发现迅速转化为癌症患者前所未有的临床结果，并加速精准肿瘤学的实施。