CTTI

Meeting Presentations:

CLICK HERE to view the presentation slides from this meeting.

The views and opinions expressed in this presentation are those of the individual presenter and should not be attributed to the Clinical Trials Transformation Initiative, or any organization with which the presenter is employed or affiliated.

Embedding Trials in Clinical Practice Expert Meetings

May 11, 2022 & September 21, 2022

CTTI Project: Trials in Clinical Practice

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MEETING OBJECTIVES:

Meeting #1 Objectives:

Present findings from project’s evidence generation: in-depth interviews with study designers and implementers
Refine operational recommendations
Begin to strategize implementation of the operational recommendations

Meeting #2 Objectives:

Develop strategies for implementing at least 2 of CTTI’s new recommendations into the planning of trials intended for regulatory review
Identify 3 implementation barriers that trial designers and health systems have the power to mitigate
Brainstorm relevant metrics to monitor and evaluate implementation of the selected recommendations

Meeting #1 Location: Virtual Meeting

Full Presentation Set

Meeting #1 Materials:

Session 1: Introduction to Embedding Trials in Health Care Settings
- Introduction to the Clinical Trails Transformation Initiative (CTTI)
- Trials in Health Care Settings Project Overview and Meeting Objectives
- Review of Project’s In-depth Interview Results
Session 2: Leveraging Examples to Refine Recommendations
- Review of Project’s Draft Recommendations
- Case Examples
Session 3: Strategizing Implementation of Recommendations
- Opportunities to Implement CTTI Recommendations

Meeting #2 Location: Hotel Washington, 515 15th Street, NW Washington, D.C.

Full Presentation Set

Meeting #2 Materials:

Session 1: Paving the Way
- Integrating Clinical Research and Care: Perspectives from Groups Paving the Way
- TQ&A
- Trials in Clinical Practice Project Overview
Session 2: Implementation Workshop
Session 3: Developing Metrics of Implementation
- Level setting on what is meant by metrics and implementation
- Metrics Development: Breakout Groups
- Gaining Momentum

The views and opinions expressed in this presentation are those of the individual presenter and should not be attributed to the Clinical Trials Transformation Initiative, or any organization with which the presenter is employed or affiliated.

Recent Webinars

2026 CTTI Patient Summit May 29, 2026
Assessing U.S. Clinical Trials Site Capacity and Readiness for Public Health Emergencies August 13, 2025
The State of Clinical Trials: Charting the Path to 2030 June 17, 2025
Optimizing Data Quality and Flexibility in Clinical Trials Expert Meeting February 28, 2025
Use of Central IRBs for Multi-center Clinical Trials October 10, 2024
Workshop on Quality Risk Management: Making Clinical Trials Fit for Purpose October 10, 2024
Workshop on Quality Risk Management: Understanding What Matters October 10, 2024

CTTI Antibacterial Drug Development: Statistical Issues Think Tank Meeting

AUGUST 20, 2012

CTTI Project: Unmet Need

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 Location:

Bethesda Hyatt Regency, Bethesda, MD

Meeting Materials:

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

Meeting Presentation:

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

Resulting Publications:

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.

The views and opinions expressed in this presentation are those of the individual presenter and should not be attributed to the Clinical Trials Transformation Initiative, or any organization with which the presenter is employed or affiliated.

Developing Effective Quality Systems in Clinical Trials: An Enlightened Approach

OCTOBER 13, 2010 TO OCTOBER 14, 2010

CTTI Project: Quality by Design

Meeting Background:

The Clinical Trials Transformation Initiative (CTTI) has been conducting a project to identify effective and efficient methods to monitor clinical trials. In seeking to identify how best to ensure the reliability of study results and the protection of trial participants, the project team has recognized that quality cannot be “inspected into” a trial but rather must be incorporated from the outset in the trial’s protocol design and operational conduct. To be effective, monitoring should be one component of an overall quality framework that allows potential issues to be identified and addressed as early as possible.

Meeting Objectives:

Describe, discuss, and evaluate novel approaches to clinical trial oversight
Propose an integrated model of quality management that will promote more efficient approaches to design, conduct and oversight of clinical trial
Identify the critical aspects of clinical trials that should be the focus of risk-based approaches to creating quality systems

Meeting Location:

Bethesda Marriott Suites, Bethesda, MD

Meeting Materials:

Meeting Presentations:

CTTI Background by Martin Landray

Defining the Underlying Principles of Quality in Clinical Trials

Quality Design of Trials by Rory Collins
Quality Risk Management in Clinical Trials by Janet Woodcock
Statistical Monitoring Applied to Research Trials by Tomasz Burzykowski

Approaches to Risk-Based Quality Management

Quality by Design/Quality Designs by Beat Widler
Clinical Data Mining as a Basis for an Information-Based Clinical QA Program by Grant Simmons
Quality System Approaches Using Existing Data and Systems by Eileen Magruder
Quality Risk Management Tools by Raffael Jovine
The NIH/NCI Data Quality Initiative by Edward Helton
An Academic Approach: Combining Quality by Design with Central Monitoring by Martin Landray
FDA's Risk-Based Inspection by Leslie Ball
Risk-Based Quality Management of Clinical Trials - A European Regulator's View by Gabriele Schwarz

The views and opinions expressed in this presentation are those of the individual presenter and should not be attributed to the Clinical Trials Transformation Initiative, or any organization with which the presenter is employed or affiliated.

Anti-Bacterial Drug Development: Issues in the Design of Trials in Patients with Unmet Need and in Patients with Hospital-Acquired and Ventilator Associated Bacterial Pneumonia

OCTOBER 11, 2012 TO OCTOBER 12, 2012

CTTI Project: ABDD Unmet Need

Meeting Background:

With rising antibiotic resistance becoming an issue of global concern, the Clinical Trials Transformation Initiative (CTTI) convened a 2-day Antibacterial Drug Development Workshop on October 11-12, 2012 in Crystal City, VA. The workshop is part of a series of expert meetings to explore approaches for accelerating the development of new antibacterial drugs. The two topics of focus for this meeting were exploring a new paradigm for antibacterial drug development in areas of unmet need, and for the treatment of patients with pneumonia that develops in the hospital or while on a ventilator. This workshop followed a think tank on statistical issues that was held on August 20, 2012, in Bethesda, MD, also hosted by CTTI.

There is an urgent need to develop new antibacterial drugs to treat patients with infections caused by bacteria that are resistant to available antibiotics. Antibiotic resistance has been increasing, creating an unmet public health need. This workshop was held in collaboration with FDA’s newly formed Antibacterial Drug Development Task Force within the Center for Drug Evaluation and Research (CDER).

The purpose of the workshop was to understand the challenges in the development of new antibacterial drugs for the treatment of patients with unmet medical need (e.g., patients with multi-drug resistant bacterial infections, who have limited or no options for treatment) and to identify potential solutions that would accelerate the drug development process. Novel endpoints and possible solutions to challenges in trial design for the development of new antibacterial drugs that treat patients with hospital-acquired or ventilator-associated bacterial pneumonia (HABP and VABP) were also explored. A broad range of stakeholders participated in the meeting, including experts from academic institutions, hospitals, pharmaceutical industry, government, and patient advocacy groups.

“The workshop provided an excellent opportunity to discuss new development pathways for antibiotic drugs targeting infections for which we urgently need new antibiotic therapies. Our collaboration with CTTI on this work is part of the CDER Antibacterial Drug Development Task Force’s efforts to accelerate the development of new antibiotics to address critical patient needs”, said Edward Cox, MD, Director of the CDER’s Office of Antimicrobial Products.

Meeting Objectives:

The goal of the first day of the workshop was to define potential pathways and explore new paradigms to accelerate the development of new antibacterial drugs that would address unmet medical need, including discussing acceptable levels of uncertainty related to the risks and benefits of such treatments. The second day focused on issues in clinical trial design including endpoints and operational efficiencies specific to the development of antibacterial drugs for treating patients with hospital-acquired or ventilator-associated bacterial pneumonia (HABP/VABP).

Meeting Location:

Sheraton Crystal City Hotel, Arlington, VA

Meeting Materials: