Introduction: Issues in Multiple outcomes in clinical trails when they are ordered or simultaneous and possibly censored

• Activity Number: 282
• Type: Topic Contributed
• Date/Time: Tuesday, August 5, 2014 : 8:30 AM to 10:20 AM
• Sponsor: Biometrics Section
• Author(s): Dianne M. Finkelstein*+
• Companies: Massachusetts General Hospital/Harvard University
• Keywords: Joint Test ; Censored Data ; Clinical Trial ; Multiple Outcomes ; Progression-free survival ; Chronic Disease

Abstract

Introduction A clinical trial may collect several outcomes that are to be used in deciding whether a new therapy is effective and tolerable for treating a chronic disease such as cancer. Often some of the outcomes (such as mortality) are censored. In oncology, the combined outcome of progression-free survival has been a standard primary outcome for some time, but there can be ambiguous results when survival benefit is not clear. In other diseases such as cardiology and neurology there has been interest in using global tests that combine multiple (possibly hierarchical) outcomes to assess new regimen. This session will discuss considerations for the design and interpretation a trial with multiple outcomes. Proposed non-parametric tests for a combined outcome will be considered. Specific trials will be discussed as illustration of the issues.