title: An overview of the design and conduct of the BATTLE trials.
abstract: Our increasing knowledge of biomedicine and genomics for human malignancies has placed us within reach of achieving personalized cancer medicine. The Biomarker-integrated Approaches of Targeted Therapy for Lung Cancer Elimination (BATTLE)-1 trial was the first completed, prospective biopsy-mandated, biomarker-based, adaptive randomized clinical trial for patients with advanced non-small cell lung cancer (NSCLC). The ongoing BATTLE-2 trial continues to search for effective targeted therapies by further refining the clinical trial design. The BATTLE program has demonstrated the feasibility and promise of novel biomarker-based clinical trial platforms, which has moved us one step closer to personalized medicine. In this paper, we describe the design and conduct of the BATTLE trials, summarize the main findings, and report the experiences and lessons learned from our pursuit of developing targeted therapies in cancer.
authors.affiliation: Department of Biostatistics, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
authors.forename: Suyu
authors.initials: S
authors.lastname: Liu
authors.affiliation: Department of Biostatistics, The University of Texas MD Anderson Cancer Center, 1400 Pressler Street, Houston, Texas 77030, USA. jjlee@mdanderson.org.
authors.forename: J Jack
authors.initials: JJ
authors.lastname: Lee
mesh_terms: D014408:Biomarkers, Tumor / Q000032:analysis; D002289:Carcinoma, Non-Small-Cell Lung / Q000188:drug therapy*; D017322:Clinical Trials, Phase II as Topic / Q000379:methods; D006801:Humans; D008175:Lung Neoplasms / Q000188:drug therapy*; D058990:Molecular Targeted Therapy / Q000379:methods; D057285:Precision Medicine / Q000379:methods; D047428:Protein Kinase Inhibitors / Q000627:therapeutic use*; D016032:Randomized Controlled Trials as Topic / Q000379:methods*; D012107:Research Design
chemical_list: D014408:Biomarkers, Tumor; D047428:Protein Kinase Inhibitors
keywords: Bayesian adaptive designs; personalized medicine; predictive biomarkers; targeted therapies