Statistical Challenges in Oncology Clinical Trials in the Era of Targeted Therapy

• Activity Number: 664
• Type: Invited
• Date/Time: Thursday, August 13, 2015 : 10:30 AM to 12:20 PM
• Sponsor: Biopharmaceutical Section
• Author(s): Rajeshwari Sridhara*
• Companies: FDA
• Keywords: Molecular signatures ; Targeted therapies ; Biomarker

Abstract

With the advances in technology to identify molecular signatures of diseases, there is increased interest and need for development of targeted therapies. It is expected that targeted therapies offer the best balance between benefit and risk of therapy by treating only patients who will potentially benefit by the therapy. However, it is a challenge to design clinical trials for evaluation of such therapies as the natural history of the disease as defined by a molecular biomarker is generally unknown. Furthermore, the current experience suggests that while patients benefit initially, it is observed that there is increased drug resistance at the time of disease recurrence, posing a challenge in evaluating overall benefit-risk of a targeted therapy. This presentation will include examples of statistical challenges in designing, analyzing, and interpretation of results of oncology clinical trials evaluating targeted therapies.