Statistical Considerations When Developing Antibacterial Treatments

Track:
Statistical Science and Quantitative Thinking

Day & Time:
June 25, 10:15AM - 11:45AM

Session Number:
237

Room Number:
157AB

Level:
Intermediate

Type:
Session

Title:
Statistical Considerations When Developing Antibacterial Treatments

Chair(s):
Rima Izem, PhD
Mathematical Statistician, Office of Translational Science, CDER
FDA, United States

Description:
The traditional regulatory requirement when assessing a new agent is often for two adequate and well controlled Phase III trials which control the type I error rate at 2.5% (one-sided) per trial. Such a requirement is a challenge for some antibacterial agents due to issues of feasibility, particularly for infections due to uncommon pathogens or treatments with a narrow spectrum of activity. As a result, there has been discussion regarding the use of differing amounts of clinical data to support approval in areas with large unmet medical need and limited feasibility.

This session will cover experiences working in this challenging area and will consider how to use the totality of evidence from a range of sources, the methodological considerations when interpreting such data, and the application of methods improving the precision of estimates of efficacy in the setting of uncommon pathogens along with design features to enable a more feasible development program while controlling the false-positive risk and ensuring there is sufficient evidence of drug effect.

Learning Objective(s):
Explain the challenges of performing a traditional development program for uncommon pathogens; Describe alternative approaches using the totality of data; Identify statistical challenges and innovative approaches to interpret data from smaller clinical programs, with strengths and limitations.

Presentation(s) & Speaker(s):
Statistical Considerations When Feasibility of Traditionally Sized Trials Is an Issue
Aaron L. Dane, MS
Biometrics & Information Sciences Infection Head
AstraZeneca, United Kingdom

FDA Perspective
Daniel B. Rubin
Statistician, Office of Translational Science, CDER
FDA, United States

Challenges in Design and Analyses of Antibacterial Trials in the Face of Resistance
Scott Evans, PhD
Senior Research Scientist
Harvard University School of Public Health, United States