Statistical Evaluation of Surrogate Endpoints: Validity, Efficiency, and Sensitivity

• Activity Number: 491
• Type: Topic Contributed
• Date/Time: Wednesday, August 1, 2012 : 10:30 AM to 12:20 PM
• Sponsor: Biopharmaceutical Section
• Author(s): Pandurang Kulkarni*+ and Yongming Qu and Wen Li
• Companies: Eli Lilly and Company and Eli Lilly and Company and Singapore Clinical Research Institute
• Keywords: Clinical outcome ; Surrogate endpoint

Abstract

Surrogate endpoints are important in drug development as they may reduce the development cost and cycle dramatically compared to some clinical outcomes. Statistical evaluation of surrogate endpoints can be dated back to thirty years ago. So far, there has been little progression in identifying new surrogate endpoints. Therefore, clinical outcomes still remain mandatory requirement for clinical drug development for many disease areas. For example, "the FDA approved Avastin for advanced breast cancer in February 2008, after one clinical trial showed that combining Avastin with another drug, paclitaxel, delayed the median time before tumors worsened by 5.5 months, compared with using paclitaxel alone. But the women who received Avastin did not live significantly longer than those who got only paclitaxel But the women who received Avastin did not live significantly longer than those who got only paclitaxel, which is also known by the brand name Taxol" (http://www.nytimes.com/2011/06/27/health/27drug.html). In this research, we will discuss the validity, efficiency & sensitivity in statistical evaluation of surrogate endpoints. New definitions with simulation & examples will be provided