Pharmaceutical Development: Statistical Challenges and Innovative Solutions

• Activity Number: 266
• Type: Invited
• Date/Time: Tuesday, July 31, 2012 : 8:30 AM to 10:20 AM
• Sponsor: International Indian Statistical Association
• Author(s): Steven Michael Snapinn*+
• Companies: Amgen, Inc.
• Keywords: Adaptive designs ; Non-inferiority ; Comparative efficacy ; Benefit-risk

Abstract

As highlighted in the FDA's Critical Path Initiative, the pharmaceutical industry's productivity in terms of innovative new drugs has been declining at a time when the pace of scientific discoveries has been increasing. The result is that the industry is currently undergoing a period of transformation, which has led to a host of new challenges for statisticians. While many of the traditional issues, such as adjusting for multiplicity and handling missing data, remain, many new issues have become more prominent. For example, statisticians have been focusing on new approaches for the evaluation of drug safety, and for the quantitative comparisons of benefit and harm. Adaptive clinical trial designs and non-inferiority designs have become increasingly common, and statisticians have played a critical role in developing valid approaches for these designs. It has also become increasingly important to demonstrate that a new treatment's magnitude of efficacy compares favorably with that of existing therapies. In this presentation I will review many of these issues, and the innovative solutions that have been proposed to address them.