Program: Antibacterial Drug Development (ABDD)

About This Program


Unmet Need

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Over the past decade, regulatory authorities have accepted streamlined approaches for drug development in general and antibacterial drugs specifically. Despite this, the development of novel antibacterial therapies is still lagging due to scientific and economic challenges. Meanwhile, resistance to currently available antibiotics is on the rise, and the absence of new antibacterial drugs has become a critical public health need. These new, non-traditional streamlined development approaches are urgently needed to address this unmet need but the perception of these approaches is poorly understood.  Therefore, there needs to be a better understanding of patient and provider perspectives on drugs developed via these streamlined approaches. This Feedback can help inform labeling and risk communication regarding these development pathways, as well as public understanding of drugs developed via these pathways and stewardship.

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Respiratory Tract Infection Clinical Trials, 2007-2012: A Systematic Review of, published in Annals of the American Thoracic Society

Expert Meeting Materials

Materials, including the meeting summary and presentations, from the think tank hosted on November 19, 2014 titled, Statistical Issues Think Tank II

Expert Meeting Materials

Materials, including the meeting summary and presentations, from the expert meeting hosted on October 11-12, 2012 titled, Anti-Bacterial Drug Development: Issues in the Design of Trials in Patients with Unmet Need and in Patients with HABP/VABP

Expert Meeting Materials

Materials, including the presentation and resulting publications, from the expert meeting hosted on August 20, 2012 titled, CTTI Antibacterial Drug Development: Statistical Issues Think Tank Meeting

Former FDA Commissioner, Margaret A. Hamburg, cited CTTI as a contributor to the FDA’s effort to address antibiotic resistance.

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The increase in multi-drug resistant bacteria paired with the current environment of ABDD, including the economic and regulatory disincentives to the development of new antibacterial drugs and difficult and complex trials, have created an urgent need to develop more treatment options for patients with unmet need (e.g., those who have multi-drug resistant bacterial infections, and those who have limited or no options for treatment).

CLICK HERE to read more about issues in ABDD.

ABDD Program: Unmet Need in Antibiotic Development (2011-ongoing)

CLICK HERE to view other projects within CTTI’s ABDD Program.

  • Identify challenges and explore solutions to address serious bacterial disease for which effective antibacterial drugs are limited for patients who have an emerging bacterial pathogen with multi-drug resistance
  • Facilitate discussion among stakeholders to explore non-traditional streamlined development pathways to restructure development of antibacterial drugs addressing unmet medical need
  • To explore statistical solutions to antibacterial drug development
  • To characterize patient, provider opinions regarding the tradeoffs between precision and uncertainty in the context of non-traditional pathways to antibiotic development

The ABDD program will facilitate the development of new AB drugs.

  • There will be a better understanding of potential alternative statistical approaches to ABDD.
  • There will be objective qualitative data about patients’ and providers’ perspectives on tradeoffs between precision and uncertainty in the context of streamlined pathways to antibiotic development.
  • Conduct a series of think tanks to explore statistical solutions to ABDD
  • Conduct focus groups to solicit patients’ views concerning the balance of perceived risk and perceived benefit
  • Conduct structured interviews and focus groups of providers, including internal medicine (community and academic), critical care, pulmonary, Pharmacy and Therapeutics committee members and others to solicit their views on the use of antibacterial drugs approved with limited data sets
  • Discuss suggestions and views during the think tanks and focus groups

You can find additional information about each of the think tanks and expert meetings by clicking on the links below:

  • CLICK HERE to view materials from the March 1, 2016 Expert Meeting, 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
  • CLICK HERE to view materials from the November 19, 2014 Think Tank, Statistical Issues Think Tank II
  • CLICK HERE to view materials from the October 11 - 12, 2012 Expert Meeting, 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
  • CLICK HERE to view materials from the August 20, 2012 Think Tank, CTTI Antibacterial Drug Development: Statistical Issues Think Tank Meeting

An expert meeting was held in 2012 to define potential pathways and explore new paradigms to accelerate the development of new antibacterial drugs that would address unmet medical need and to discuss issues in ABDD 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). Participants agreed that it is critical for all stakeholders to work together to characterize novel research models and seek alternatives to traditional superiority trials in ABDD. The following paragraphs summarize suggestions made by the experts to address particular trial issues in ABDD.

To address challenges with enrollment into ABDD trials, experts suggested the following: performing studies in geographic areas where the pathogen is endemic, or where there is a greater likelihood of encountering serious infections; improving rapid diagnostics; and creating large networks of research sites to ensure consistency and efficiency in the execution of innovative trials. The latter may also facilitate collaboration and improve coordination for a more rapid response to newly identified outbreaks of resistant pathogens. Issues with clinical trial design and data analyses may be simplified by including a development/approval pathway using a smaller clinical trial database, using well-defined and reliable efficacy endpoints, and identifying suitable control groups for various study design scenarios. Potential solutions in ABDD to meet the unmet need for antibacterial drugs may rely on accelerated approval, heightened postmarketing surveillance, collaboration among stakeholders, and the efforts of those involved in the clinical trial enterprise to establish a new model of ABDD.

At the expert meeting, attendees also discussed unmet need in HABP/VABP. They explored topics such as the feasibility of endpoints, biomarkers, and other surrogates in HABP/VABP trials; collaborative efforts for data sharing/mining across the available clinical databases; and the need for additional education and potential acceptance of both accelerated approval and use of alternative endpoints in ABDD. Experts also discussed potential solutions to improving the operational efficiency and feasibility of HABP/VABP trials. Briefly, streamlining strategies were discussed as well as plain-language informed consent forms, site pre-approval for enrollment of future studies, simplified data collection, promotion of FDA guidances, clearer public communication, and use of organized research groups (eg, AIDS Clinical Trial Group) as models. HABP/VABP trial-specific results are presented and discussed in more detail in the Streamlining HABP/VABP Trials Project.

Project Team Members

Rolesort descending Name Affiliation
Project Manager Annemarie Forrest Clinical Trials Transformation Initiative
Team Leader Deborah Collyar Individual Patient/Caregiver
Team Leader Edward Cox Food and Drug Administration
Team Leader Vance Fowler Duke University
Team Leader Thomas Holland Duke University
Team Leader Stephen Mikita Individual Patient/Caregiver
Team Leader Rosemary Tiernan Food and Drug Administration
Team Leader Joseph Toerner Food and Drug Administration
Team Member Helen Donnelly Northwestern University
Team Member Jonas Santiago Food and Drug Administration