CTTI Project: Unmet Need
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ABDD Unmet Need
The Use of Antibacterial Drugs Developed via Streamlined Approaches for Serious Infections Where There is Unmet Need: Understanding Patient and Physician Perspectives and Considerations to Take Forward
MARCH 01, 2016
CTTI Project: Unmet Need
Meeting Objectives
- Present perspectives from patients, caregivers and physicians on antibacterial drugs developed using streamlined approaches
- Identify focus group themes and discuss topics which should be further explored or where draft recommendations could be made
- Obtain feedback to improve labeling, risk communication, public understanding and stewardship
Meeting Location:
Sheraton Silver Spring Hotel, Silver Spring, MD
Meeting Presentations:
- Introduction to the Clinical Trials Transformation Initiative and the ABDD Program by Pamela Tenaerts
- Project Overview and Scope by Jamie Roberts
- Current Landscape and Pipeline of Antibacterial Products by Vance Fowler
- Streamlined Development Approaches by Joseph Toerner
- Findings: Perspectives of Patients, Caregivers and Healthy People by Diane Bloom
- Findings: Perspectives of Providers and Investigators by Thomas Holland
- Focus Group Themes and Questions for Consideration by Stephen Mikita
- Closing by Pamela Tenaerts, Rosemary Tiernan & Jamie Roberts
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.
Statistical Issues Think Tank II
NOVEMBER 19, 2014
CTTI Project: Unmet Need
Meeting Objectives
- To provide an update on the current status of statistical methodologies for the design and analysis of antibacterial drugs
- To discuss ongoing challenges in the development and adoption of innovative methods
- To generate strategies to propel antibacterial drug development forward.
Meeting Location:
Bethesda North Marriott Hotel & Conference Center, Bethesda, MD
Meeting Presentations:
Session 1: Current status of drug development and ongoing challenges
- Introduction to CTTI's Clinical Trials Transformation Initiative: Antibacterial Drug Development Project by Lisa M. LaVange
- Summary of Regulatory Standards and Guidances by Joseph Toerner
- Summary of unmet need guidance and statistical challenges by Daniel B. Rubin
- Application of Pharmacokinetics/Pharmacodynamics in New Anti-Infective Drug Development: Current Challenges and Future Perspectivesby Seong Jang
- Statistical Considerations for Antibiotic Drug Development by Aaron Dane
Session 2: Current research and additional opportunities for the future
- Discordant MIC Analysis: Testing for Superiority within a Non-inferiority Trial by Dean Follmann, Erica BriDain, and John Powers
- Hierarchical nested trial design (HNTD) for new antibacterial drugs in patients with emerging bacterial resistance by Thamban Valappil and Mohammad Huque
- Response Adjusted for Duration of Antibiotic Risk (RADAR) by Scott Evans
- Some Proposals in the Analysis of Antibacterial Drug Trials by Margaret Gamalo-Siebers and Ram C. Tiwari
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.
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 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.
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.
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 Presentations:
- 1.1 Overview of Risk-Benefit within Antibacterial Drug Development by Ed Cox
- 1.2 Antibiotic Resistance by John Bartlett
- 1.3 2012 Update of Antibiotic Resistance in the Multidrug-resistant Organism Repository & Surveillance Network (MRSN) by Emil Lesho
- 1.4Drug Development Challenges: Facilitating Collaboration by Charlene Reed
- 1.5 Clinical Trials for Rare Diseases in CDER by Kathryn O’Connell
- 1.6 Novel Approaches to Further Antibacterial Drug Development: New Approaches to the Clinical Development Program by John Rex
- 1.7 Limitations of PK-PD in Clinical Trials: Focus on Vancomycin MIC and MRSA Infection by Vance Fowler
- 1.8 Possible Clinical Trial Approaches for Areas of Unmet Need by Ed Cox
- 1.9 CTTI Think Tank for Anti-Bacterial Drug Development: Unmet Medical Need and HABP/VABP by Lisa LaVange & Daniel Rubin
- 2.1 Hospital-Acquired Bacterial Pneumonia & Ventilator-Associated Bacterial Pneumonia by Joseph Toerner
- 2.2 Direct and Indirect Measures of Treatment Effects Including Surrogate Endpoints by John Powers
- 2.3 Endpoints in HAP/VAP trials: Mortality, Clinical Endpoints, Biomarkers by Richard Wunderink
- 2.4 Identifying Non-mortality Clinical Endpoints FNIH Approach to CABP and ABSSSI by George Talbot
- 2.5 Design Strategies to Improve the Feasibility of HABP-VABP Registration Studies by P. Prokocimer
- 2.6 Issues in Operational Efficiency of HAP/VAPClinical Trials: Current and Future State by Charles Knirsch
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.
CTTI Publishes Findings on Patient & Physician Perceptions of Streamlined Development for Antibacterial Drugs
Over 2 million people in the United States are estimated to become infected with drug-resistant bacteria each year, so new antibacterial therapies are desperately needed. Streamlined drug development approaches have the potential to accelerate the availability of new antibacterial drugs for patients with serious unmet need who have limited or no treatment options. CTTI has published findings on patient and physician attitudes regarding streamlined development approaches for antibacterial drugs in BMJ Open.
Through a series of interviews and focus group discussions, CTTI found that patients and physicians agreed on the usefulness of streamlined approaches in situations of unmet need, but both groups also emphasized the need for careful oversight, transparency in risk communication, and continuous monitoring and reporting of safety and efficacy post-approval.
These findings, which resulted from CTTI’s Unmet Need in Antibiotic Development Project, can help inform the future use of streamlined drug development approaches and communication with stakeholders. For example, patients described information they would like to know when treatment with a drug developed using a streamlined approach is being considered. They also expressed a preference to have early conversations with their providers around the potential use of these treatments before they become seriously ill, when their decision-making may be impaired.
CTTI gathered input on these findings and their impact in a multi-stakeholder expert meeting; a meeting summary is available for additional context. CTTI would like to thank the interview and focus group participants, expert meeting attendees, and project team members who contributed to this work.
Materials from CTTI’s Unmet Need in Antibiotic Development Meeting Now Available
On March 1, 2016, CTTI hosted an expert meeting entitled, The Use of Antibacterial Drugs Developed via Streamlined Approaches for Serious Infections Where There is Unmet Need: Understanding Patient and Physician Perspectives and Considerations to Take Forward. We are pleased to now share the meeting materials online, including the presentations, agenda, and attendee list.
This meeting was associated with the Unmet Need in Antibiotic Development Project. A variety of stakeholders attended, including academia, clinical experts, patient advocates, regulators, pharmaceutical companies, and others involved in the clinical research enterprise. The meeting objectives were to:
- Present perspectives from patients, caregivers and physicians on antibacterial drugs developed using streamlined approaches
- Identify focus group themes and discuss topics which should be further explored or where draft recommendations could be made
- Obtain feedback to improve labeling, risk communication, public understanding and stewardship
Executive Summary Available for CTTI’s Statistical Issues in Antibiotic Development Think Tank II
Advances in clinical trial design over the past decade have offered new and accelerated pathways for drug development. Despite this, the development of novel antibacterial therapies is lagging due to scientific and economic challenges. To address this critical unmet need, CTTI has hosted meetings 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.
On November 19, 2014, CTTI, in collaboration the FDA’s Antibacterial Statistics Working Group, convened the second meeting of statistical and medical experts in the area of antibacterial drug development. The goals of this meeting were to:
- Provide an update on the current status of statistical methodologies for the design and analysis of antibacterial drugs
- Discuss ongoing challenges in the development and adoption of innovative methods
- Generate strategies to propel antibacterial drug development forward
We are pleased to share an Executive Summary of this meeting, in addition to the presentations and agenda. Click here to view all meeting materials.
*This meeting is part of CTTI’s Unmet Need in Antibiotic Development Project.