Antimicrobial Products: Statistical Challenges and Opportunities

Abstract

According to a 2013 report of the Centers for Disease Control and Prevention, more than 2 million people in the United States develop illness from antibiotic-resistant infections yearly and at least 23,000 die as a direct result of antibiotic-resistant infections. Despite an alarming number of antibiotic-resistant infections, antibacterial drug development has been slow. The consequences of the lack of new effective antibiotics may be dire to our global public health.

There are scientific, economic, and regulatory challenges that underpin the sparse antibiotic pipeline. In this article, we outline critical design and analysis elements that impact clinical trials of new antibiotics and include some of the complex scientific challenges unique to antibacterial products. The challenges also present opportunities for statisticians to provide innovative strategies to the design and analysis of these trials.

Key Words:

• Bayesian analysis
• Clinical trial
• Hierarchical design
• Master protocol
• Noninferiority trial

Additional information

Notes on contributors

Dionne L. Price

Dionne L. Price (E-mail: [email protected]), Daniel B. Rubin (E-mail: [email protected]), and Thamban Valappil (E-mail: [email protected]), Division of Biometrics IV, Office of Biostatistics, Office of Translational Sciences, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Avenue, Silver Spring, MD 20993.

Daniel B. Rubin

Dionne L. Price (E-mail: [email protected]), Daniel B. Rubin (E-mail: [email protected]), and Thamban Valappil (E-mail: [email protected]), Division of Biometrics IV, Office of Biostatistics, Office of Translational Sciences, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Avenue, Silver Spring, MD 20993.

Thamban Valappil

Dionne L. Price (E-mail: [email protected]), Daniel B. Rubin (E-mail: [email protected]), and Thamban Valappil (E-mail: [email protected]), Division of Biometrics IV, Office of Biostatistics, Office of Translational Sciences, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Avenue, Silver Spring, MD 20993.