Expert Meeting

CTTI Antibacterial Drug Development: Statistical Issues Think Tank Meeting

CTTI Project: Unmet Need

Meeting Location

Bethesda Hyatt Regency, Bethesda, MD

CLICK HERE for a list of meeting attendees.
CLICK HERE for the meeting agenda.


Meeting Background

A CTTI-hosted Statistical Issues Think Tank Meeting was convened to provide an opportunity for leading experts in clinical trial methodologies to discuss alternative approaches to design and analysis that may be useful for antibacterial drug development programs.


Meeting Presentation

CTTI Statistics Think Tank for Anti-Bacterial Drug Development by Lisa LaVange


Resulting Publications

1) Hierarchical nested trial design (HNTD) for demonstrating treatment efficacy of new antibacterial drugs in patient populations with emerging bacterial resistance

On June 23, 2014, Statistics in Medicine published an article by Huque et al. that describes a novel clinical trial model to address the increased prevalence of antibiotic-resistant bacterial infections. This paper resulted from the CTTI-hosted Statistical Issues Think Tank Meeting that author Mohammad Huque attended.


2) The Role of Statistics in Regulatory Decision Making

In February, 2014, this peer-reviewed article was published in Therapeutic Innovation & Regulatory Science. This paper resulted from the CTTI-hosted Statistical Issues Think Tank Meeting where author Lisa M. LaVange, PhD, attended and presented. In this publication, LaVange concludes:

This article presents the author’s view of the important role that statisticians play in regulatory decision making, beginning with a broad overview of current office initiatives, including the development of guidance documents and a recent push for open and transparent collaboration with industry on methods development. Several recent examples are provided to illustrate the impact that statisticians can have on regulatory decisions through the use of strategic quantitative thinking. Also discussed are areas where it is believed that innovative statistical solutions or greater clarity on existing approaches is still needed.

3) Bayesian approach to the design and analysis of non-inferiority trials for anti-infective products

On August 5, 2013, the Journal of Pharmaceutical Statistics published an article by Gamalo et al. that describes a Bayesian approach for the design and analysis of active comparator trials used in the development and market approval of antibacterial drugs. This work was presented in part at the CTTI-hosted Statistical Issues Think Tank Meeting.

“The CTTI Statistics Think Tank, a collaborative initiative with the FDA/CDER Anti-Bacterial Drug Development Task Force, was held in August, 2012 and provided an excellent opportunity for leading statisticians from academia, government, and industry to discuss innovative approaches for accelerating the development of new antibiotics. This article presents a comprehensive Bayesian approach to non-inferiority trials and represents but one of several approaches that were discussed at the Statistics Think Tank meeting. We look forward to additional research being published from the ideas generated at that meeting.” said Lisa LaVange Director of Biostatistics, Food and Drug Administration.

The Bayesian approach may allow for use of information from sources that include observational studies and early phase trials on the effect of the active comparator for study design. This approach may aid in reducing large sample size requirements and decrease study durations for antibacterial agents that enter late-stage clinical development.