Challenges and Opportunities in Early Oncology Trials

• Activity Number: 380
• Type: Roundtables
• Date/Time: Tuesday, August 11, 2015 : 12:30 PM to 1:50 PM
• Sponsor: Biopharmaceutical Section
• Author(s): Rong Liu*
• Companies: Bayer HealthCare
• Keywords: Oncology ; Early Phase ; Biomarkers ; futility analysis

Abstract

Early-phase oncology development shares common challenges encountered in other therapeutic areas, such as small sample size, rapid dose escalation, etc. It also has some uniqueness; rather than conducted on healthy volunteers, it is almost always conducted in heterogeneous patient population with little or no therapeutic alternative, it is naturally adaptive, and---after defining MTD---expansion cohorts are often part of the FiM study to get a first look at efficacy. It seems the size of expansion cohorts are increasing. In addition, biomarker studies are incorporated into drug trials from very early on and it is complex. Therefore, goals of this roundtable discussion are to share experiences on 1) design and options for appropriate futility analysis for the expansion cohorts and 2) utility of statistics on biomarker evaluation such as scientific rationale, assay criteria, and trial design and analysis.