ABDD HABP/VABP Studies

Overview

Clinical trials of new antibacterial drugs for hospital-acquired and ventilator-associated bacterial pneumonia (HABP/VABP) are critically needed but challenging to conduct.

Building on our streamlining HABP/VABP trials work, CTTI conducted a prospective, multi-center, observational study of the risk factors for HABP/VABP, with over 6,700 adult patients enrolled at 28 U.S. sites and seven European sites, and 800 pediatric patients enrolled at nine U.S. children’s hospitals.

The study, an analysis of cost drivers and formative research on an early consent approach, are informing resources to help you develop and streamline HABP/VABP clinical trial planning.  For example, patients at high risk for developing pneumonia could be approached and consented early, potentially before pneumonia symptoms develop so that fewer patients are excluded because they have already received 24 hours of effective antibacterial therapy.

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Recent CTTI Publication Investigates HABP/VABP Risk and Antibiotic Trial Eligibility for Hospitalized Patients in Europe
A new CTTI publication investigates the prospective identification of hospital-acquired and ventilator-associated bacterial pneumonia (HABP/VABP) risk factors and antibiotic trial eligibility among hospitalized, critically ill patients at seven European hospitals. ...

Case Study

In Detail

What is the issue?

One of the challenges in planning the HABP/VABP pilot study is determining which patients to approach for early enrollment. A group of patients at risk for pneumonia is required, but selecting criteria based on existing literature will involve a tradeoff between sensitivity and specificity. Approaching only the highest risk patients (e.g., those on a ventilator for a pre-specified length of time) would likely yield a relatively high percentage of patients who go on to develop pneumonia. The drawbacks of this approach include 1) not including a mix of VABP and non-VABP HABP; 2) exclusion of a large group of other patients who go on to develop pneumonia in the hospital in absence of the highest risk factors; and 3) increased likelihood of exclusion due to current use of antibiotics. Using a more inclusive approach, such as enrolling patients with a history of aspiration or with chronic lung disease, would increase the total number of patients who ultimately develop pneumonia, but would decrease the proportion of enrolled patients that develop pneumonia. If the proportion of patients developing pneumonia is too low, it is possible that the time, effort, and money required to conduct a study using an early enrollment strategy would not result in a meaningful increase in the number of patients enrolled who ultimately meet study criteria for antibacterial therapy for HABP/VABP.

A risk factor study was conducted to better define the population at highest risk for developing HABP/VABP. These results, in combination with CTTI’s Recommendations for Streamlining HABP/VABP Clinical Trials, which include a proposal for early consent to increase enrollment, have informed the development of an early enrollment strategy. Resources for those planning to implement an early enrollment strategy are being developed.

Read more about issues in ABDD.

What CTTI project has been initiated to address the issue?

What are the objectives of CTTI's work on the HABP/VABP pilot study?

What is the anticipated impact of CTTI's work on the HABP/VABP pilot study?

How was evidence gathered to support our findings?

What were the findings from the risk factor study activities?

The related CTTI Streamlining HABP/VABP Trials Project included a number of collaborative efforts to better understand and address the challenges of conducting HABP/VABP trials. A data gathering and mapping project conducted in collaboration with the Tufts Center for the Study of Drug Development found that the cost of screen failures, as well as screen failure rates, are the main drivers of cost for a phase 3 HABP/VABP trial. HABP/VABP phase 3 trials are generally non-inferiority trials.

To address the concern of bias toward non-inferiority, FDA Draft Guidance recommends that patients who have received effective antibacterial drug therapy for HABP/VABP for a continuous duration of more than 24 hours during the previous 72 hours be excluded from HABP/VABP clinical trials. In a guided discussion with experienced research coordinators conducted by CTTI, this recommended exclusion criteria was noted as a significant challenge to enrollment as care standards in the intensive care unit often lead to antibacterial drugs being administered as soon as pneumonia is suspected. In addition, patients are often quite ill and unable to provide informed consent themselves, so legally authorized representatives must be identified and contacted for enrollment in a clinical trial. Other necessary enrollment procedures such as laboratory tests and other procedures to meet inclusion/exclusion criteria, randomization, and obtaining study drug from the pharmacy can add to the time between administration of antibacterial drugs and study enrollment. Strategies to address these feasibility concerns while ensuring that trial interpretability will not be limited by a bias toward non-inferiority are needed.

As part of the Streamlining HABP/VABP Trials Project, CTTI identified a network of U.S. and European sites for conducting HABP/VABP studies. A working group was convened and included key stakeholders, such as infectious diseases and pulmonary/critical care medicine experts, those experienced in designing clinical studies, and patient representatives. Site contracts were negotiated to minimize potential future delays. The network is being used to conduct a prospective observational study that examines the risk factors associated with HABP/VABP. Results from this study will inform the development of a randomized interventional pilot trial to test an early enrollment strategy that could make HABP/VABP trials more feasible. An expert meeting was held to gather input on design of the planned pilot study.

CTTI, together with the Tufts Center for the Study of Drug Development, conducted an analysis of cost drivers for phase 3 HABP/VABP trials to identify ways to make these trials more feasible. Key findings included:

The cost of a phase 3 HABP/VABP clinical trial averages $89.6 million, which is over double the average cost of a phase 3 oncology trial
Per patient, phase 3 HABP/VABP clinical trials were $2,300 more expensive than oncology clinical trials and $31,900 more expensive than endocrine trials
Screening failure rates and the cost of screening failures were the biggest drivers of cost, suggesting sponsors and research sites should consider ways to increase the number of potentially eligible patients

Additional Resources

Project Team Members

Role	Name	Affiliation
Principal Investigator	Vance Fowler	Duke University
Project Manager	Sara Calvert	Clinical Trials Transformation Initiative
Social Science Lead	Amy Corneli	Clinical Trials Transformation Initiative
Team Member	Elizabeth Alexander	The Medicines Company
Team Member	Stephen Bergin	Duke University
Team Member	Deborah Collyar	Individual Patient/Caregiver
Team Member	Heather Cross	Duke University
Team Member	Carisa DeAnda	Merck & Co, Inc.
Team Member	Helen Donnelly	Northwestern University
Team Member	Beth Evans	Duke University
Team Member	John Farley	Food and Drug Administration
Team Member	Karen Fusaro	Melinta Therapeutics
Team Member	Peidi Gu	Duke University
Team Member	Thomas Holland	Duke University
Team Member	Sebastiaan Hullegie	University Medical Center Utrecht
Team Member	Kristen Miller	Food and Drug Administration
Team Member	John Powers	National Institutes of Health
Team Member	Daniel Rubin	Food and Drug Administration
Team Member	Jonas Santiago	Food and Drug Administration
Team Member	Simone Shurland	Food and Drug Administration
Team Member	Pamela Tenaerts	Clinical Trials Transformation Initiative
Team Member	Joshua Thaden	Duke University
Team Member	Rose Tiernan	Food and Drug Administration
Team Member	Joseph Toerner	Food and Drug Administration
Team Member	Henri van Werkhoven	University Medical Center Utrecht

Organization affiliations are listed as active affiliations during the project.

*Indicates former project manager, team leader, or team member.

ABDD Peds Trials

Overview

Children often respond differently to medications approved for adults, and therefore pediatric trials are required and critically needed for new antibiotics in order to inform dosing, efficacy, and safety.

Use CTTI’s recommendations to design pediatric antibacterial trials that ensure adequate enrollment, decrease the burden on sites and families, understand special considerations for neonates, develop appropriate informed consent procedures, and increase engagement with healthcare providers.

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Recommendation Summary

Recommendations

Recommendations for Improving Pediatric Trials in Antibacterial Drug Development
Recommendations for Improving Pediatric Trials in Antibacterial Drug Development

Resources

News

CTTI Study Shows More Efforts Are Needed to Stimulate Pediatric Antibacterial and Antifungal Drug Trials
In an article recently published in Pediatrics, CTTI researchers assess the impact of the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA) on pediatric antibacterial and antifungal drug trials. The ...

Case Study

DCRI’s PTN Builds Efficiencies to Improve the Enrollment, Design, and Conduct of its Studies
DCRI’s PTN Builds Efficiencies to Improve the Enrollment, Design, and Conduct of its Studies

In Detail

What is the issue?

The Pediatric Research Equity Act (PREA) requires pediatric trials to be conducted with a drug for the same use as it was approved in adults, unless an FDA waiver is obtained. To comply, sponsors are required to conduct pediatric trials with most antibacterial drugs to determine dosing, efficacy, and safety in children. Designing trials and establishing antibacterial drug dosage in pediatric populations, including neonates, is challenging for the following reasons:

Estimating a dose regimen for children based solely on the pharmacokinetics of a drug in adults can result in inappropriate dosing. Clinical trials must be conducted to define the pharmacokinetics of a drug in children before a new drug can be used with confidence in the pediatric population.
Typically, fewer children than adults have a given disease, and fewer children than adults are eligible for inclusion in a clinical trial. As a result, trials involving children typically have smaller sample sizes, which raise concern about the robustness of the conclusions.
Pediatric studies are more difficult to conduct due to ethical considerations and challenges in obtaining parental consent. Parents may not wish to consent to a perceived additional risk for their child to participate in a clinical trial with a drug that is considered to be experimental in children but safe and effective in adults.

Read more about issues in ABDD.

What CTTI project has been initiated to address the issue?

What are the objectives of CTTI's work on ABDD pediatric trials?

What is the anticipated impact of CTTI's work on ABDD pediatric trials?

How was evidence gathered to support our findings?

What are the findings from ABDD pediatric trials?

CTTI’s recommendations resulted from the evidence described below, as well as suggestions from research sponsors, parents, investigators, clinicians, and regulators on ways to improve the timing of pediatric trials, streamline trial design, better facilitate informed consent, and foster global and community partnerships that will promote the conduct of trials to improve children’s health. These recommendations can be used to improve the quality and efficiency of pediatric antibacterial trials; additionally, many of the suggested strategies can be applied to streamline pediatric clinical trials in other therapeutic areas.

A CTTI review of the ClinicalTrials.gov AACT Database revealed far fewer antibacterial drug trials than other types of trials in the pediatric population. From 2007 to 2010, there were 110 registered pediatric antibacterial trials, representing <1% of all registered pediatric studies. An online survey with community providers and investigators revealed that many perceive barriers to participating in or conducting these trials. Parental concerns, such as concerns about side effects or invasive procedures, were among the factors most consistently rated as barriers. These findings suggested that further engagement with parents is needed, which is addressed in the CTTI recommendations.

Interviews were conducted with parents and caregivers of children who were approached to participate in clinical trials, as well as with industry representatives who had experience in pediatric antibacterial drug development. The results informed a number of strategies that could help increase recruitment for these trials, such as improving communications with parents, broadening eligibility criteria, and minimizing the burden of participation. A multi-stakeholder expert meeting was held to discuss the findings of the interview and survey data and provide input on recommendations.

The survey and interview results from this project are summarized in this 2016 poster.

Project Team Members

Role	Name	Affiliation
Project Manager	Annemarie Forrest	Clinical Trials Transformation Initiative
Social Science Lead	Amy Corneli	Clinical Trials Transformation Initiative
Team Leader	Daniel Benjamin	Duke University
Team Leader	Sumathi Nambiar	Food and Drug Administration
Team Leader	Gary Noel	Johnson & Johnson Pharmaceutical Research and Development
Team Member	John Bradley	University of California, San Diego
Team Member	John Farley	Food and Drug Administration
Team Member	Breck Gamel	Individual Patient/Caregiver
Team Member	Ethan Hausman	Food and Drug Administration
Team Member	Hasan Jafri	Medimmune
Team Member	Brian Smith	Duke University
Team Member	Edward Spindler	The Medicines Company
Team Member	Pamela Tenaerts	Clinical Trials Transformation Initiative
Team Member	Rose Tiernan	Food and Drug Administration
Team Member	Chris Wheeler	Food and Drug Administration
Team Member	Kunyi Wu	Food and Drug Administration

Organization affiliations are listed as active affiliations during the project.

*Indicates former project manager, team leader, or team member.

ABDD Streamlining HABP/VABP Trials

Overview

Hospital-acquired and ventilator-associated bacterial pneumonia (HABP/VABP) occur in seriously ill, hospitalized patients, where resistance to antibiotics is rising. This makes clinical trials of antibiotics in this population not only urgent but also especially challenging.

Use CTTI’s recommendations to streamline protocol elements and data collection in HABP/VABP trials to increase enrollment, reduce trial complexity, and optimize operational efficiency and, ultimately, help combat the current public health crisis in antibiotic development.

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Webinar Recording Available: Global Expert Panel on Antibacterial Drug Development
On August 24, 2016, CTTI hosted a webinar on innovative approaches to transforming antibacterial drug development as recently featured in a Clinical Infectious Diseases (CID) supplement. The presentation highlighted the important role public-private ...

Case Study

In Detail

What is the issue?

HABP/VABP Antibacterial Drug Development (ABDD) trials are particularly challenging to conduct for many reasons, including the length and complexity of the protocol and the challenges associate with the patient population, trial design, data recording, differences in international regulations, and operational concerns. These complications and obstacles have increased HABP/VABP trial costs and slowed ABDD.

Read more about issues in ABDD.

What CTTI project has been initiated to address the issue?

What are the objectives of CTTI's work on streamlining HABP/VABP trials?

What is the anticipated impact of CTTI's work on streamlining HABP/VABP trials?

The ABDD program will facilitate the development of new antibacterial drugs. Specifically, the results from the Streamlining HABP/VABP Trials project will inform better processes and practices to employ in a HABP/VABP study protocol to be tested at participating clinical sites.

A study protocol using the recommendations developed from the workstreams will show that an innovative approach will work, leading to more ABDD clinical trials, which will eventually lead to more available antibiotics.
Additionally, streamlining data collection and recording will help accelerate study conduct and reduce the cost associated with excess data collection.

How was evidence gathered to support our findings?

CTTI convened experts and held a series of meetings and workshops to address the following:

Identify inefficiencies of current protocol elements and data collection processes in ABDD trial conduct.
Explore novel ideas and issue recommendations on designing scientifically valid, feasible, and clinically meaningful trials to establish the efficacy of antibacterial drugs for the treatment of HABP/VABP.
Build a pilot network of 45 sites globally (30 U.S. and 15 OUS) with a capability of conducting and implementing the HABP/VABP Studies.

Click on the meeting titles below to view associated materials, including agendas, participant lists, presentations, and summaries:

What are the findings from streamlining HABP/VABP trials?

Stakeholders from industry, academia, government, and patient groups met to identify areas in need of improvement in HABP/VABP protocols: the protocol design, the informed consent process (to address decisional impairment), use of a central IRB, and trial outcomes and endpoints. At expert meetings, stakeholders discussed best practices for defining the enrollment criteria, testing new diagnostics, obtaining informed consent from seriously ill patients, selecting an IRB, and pre-specifying trial endpoints in the protocol. Stakeholders also agreed that safety data collection in the HABP/VABP patient population is challenging and burdensome due to the large number of medical procedures, adverse events, and concomitant medications involved. Additional challenges include protocol complexity and the lack of harmonization in U.S. and EU regulations regarding the conduct of HABP/VABP trials. Expert meeting attendees agreed that utilizing a QbD approach to protocol elements and trial planning is critical to facilitate streamlining of data collection and recording.

Based on this work, CTTI released recommendations for Streamlining Protocol Elements and for Optimizing Operational Efficiency for Safety Data Collection HABP VABP Trials. The recommendations appeared as part of a peer-reviewed supplement in Clinical Infectious Diseases that featured collaborative and innovative approaches by CTTI and others to address challenges associated with HABP/VABP trials.

The recommendations are also being applied to a proposed HABP/VABP Pragmatic Clinical Study.

A part of the Streamlining HABP/VABP project, a working group including key stakeholders, such as infectious diseases and pulmonary/critical care medicine experts, those experienced in designing clinical studies, and patient representatives, was convened to identify a network of sites for future studies. Master site contracts were negotiated in advance to minimize start-up delays. There has also been outreach to the Innovative Medicines Initiative’s Combacte and New Drugs for Bad Bugs projects and their CLIN-net site to expand the site list. The U.S. and European sites were used for the Risk Factors for HABP/VABP Study.

Additional Resources

Project Team Members

Role	Name	Affiliation
Project Manager	Gerrit Hamre*	Clinical Trials Transformation Initiative
Project Manager	Jen Goldsack*	Clinical Trials Transformation Initiative
Team Leader	Gary Noel	Johnson & Johnson Pharmaceutical Research and Development
Team Leader	Rosemary Tiernan	Food and Drug Administration
Team Leader	Vance Fowler	Duke University
Team Member	Barry Eisenstein	Merck & Co, Inc
Team Member	Ben Lorenz	Food and Drug Administration
Team Member	Charles Knirsch	Pfizer Inc
Team Member	Christina Reith	University of Oxford, CTSU
Team Member	Daniel Rubin	Food and Drug Administration
Team Member	David Friedland	Cerexa
Team Member	Demissie Alemayehu	Pfizer Inc
Team Member	Eric Pelfrene	European Medicines Agency
Team Member	Helen Donnelly	Northwestern University
Team Member	Jonas Santiago	Food and Drug Administration
Team Member	Joseph Toerner	Food and Drug Administration
Team Member	Joshua Thaden	Duke University
Team Member	Kunal Merchant	RRD International LLC
Team Member	Radu Botgros	European Medicines Agency
Team Member	Richard Wunderink	Northwestern University
Team Member	Sabrina Comic-Savic	The Medicines Company
Team Member	Thomas Holland	Duke University

Organization affiliations are listed as active affiliations during the project.

*Indicates former project manager, team leader, or team member.

New CTTI Paper Outlines Risk Factors to Aid in HABP/VABP Pediatric Trial Enrollment

Posted on March 17, 2020August 11, 2021 by kristi.geercken@duke.edu

A CTTI paper recently published in the Pediatric Infectious Disease Journal sheds light on risk factors that make children more likely to contract hospital-acquired and ventilator-associated bacterial pneumonia (HABP/VABP).

These risk factors are important because they could aid in earlier and more accurate diagnosis of HABP/VABP, alleviating roadblocks to enrollment in HABP/VABP clinical trials. Improved enrollment could help speed the development of new antibiotics for HABP/VABP, which can often be deadly for children.

The paper details a study conducted by the Pediatric Trials Network in collaboration CTTI, which used pediatric patients’ electronic health records to identify which patients received qualifying respiratory support or antibiotics for either a lower respiratory infection or undifferentiated sepsis. The study then followed the selected patients until they were either diagnosed with HABP/VABP or discharged from the intensive care unit.

Of 862 newborns, infants, and children younger than 18 who were evaluated, 10 percent of patients receiving respiratory support and 12 percent overall developed HABP/VABP. While risk factors varied by age group, patients showed increased odds of developing HABP/VABP if they:

Were older
Were shorter
Spent a longer time in the intensive care unit
Were at risk of aspiration
Had received a blood product transfusion in the prior seven days
Had frequent suctioning

Increased attention to patients with these high-risk features could lead to improved identification of eligible participants in HABP/VABP antibiotic trials.

Learn more about CTTI’s work to improve the feasibility of HABP/VABP clinical trials.

ABDD Unmet Need

Overview

The development of novel antibacterial therapies is lagging behind the rise of resistance to antibiotics and threatens to become a critical public health emergency.

To help address the unmet need for new antibiotic therapies, CTTI conducted research and issued findings on patient and physician perspectives and considerations as it relates to using antibacterial drugs developed through streamlined development processes.

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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 ...

Case Study

In Detail

What is the issue?

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).

Read more about issues in ABDD.

What CTTI project has been initiated to address the issue?

What are the objectives of CTTI's work on ABDD unmet need?

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 and provider opinions regarding the tradeoffs between precision and uncertainty in the context of non-traditional pathways to antibiotic development

What is the anticipated impact of CTTI's work on ABDD unmet need?

How was evidence gathered to support our findings?

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:

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
November 19, 2014 Think Tank, Statistical Issues Think Tank II
October 11-12, 2012 Expert Meeting, Antibacterial Drug Development: Issues in the Design of Trials in Patients with Unmet Need and in Patients with Hospital-Acquired and Ventilator-Associated Bacterial Pneumonia
August 20, 2012 Think Tank, CTTI Antibacterial Drug Development: Statistical Issues Think Tank Meeting

What were the findings from this project?

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 post-marketing surveillance, collaboration among stakeholders, and the efforts of those involved in the clinical trials ecosystem 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 (e.g., 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

Role	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

Organization affiliations are listed as active affiliations during the project.

*Indicates former project manager, team leader, or team member.

CTTI Article Finds that Patients and IRBs are Amenable to Early Enrollment Strategy

Posted on December 14, 2018August 12, 2021 by Lorri Bingham

A CTTI article recently published in JAMA Network Open shows that an early enrollment strategy for research on healthcare-associated pneumonia is acceptable to patients, investigators, and institutional review boards (IRBs). This strategy has the potential to speed enrollment in trials for critical new antibiotic therapies by allowing patients to be approached and consented to before being diagnosed with pneumonia.

Through its ABDD HABP/VABP Studies work, CTTI conducted qualitative interviews with 52 stakeholders—including patients at risk for pneumonia, caregivers, study investigators and coordinators, and IRB representatives—as part of formative research to assess the acceptability of the approach.

The study found that patients and caregivers had no concerns about patients being approached early and having their records monitored before they developed pneumonia. They believed that patients would be able to understand consent information before diagnosis, and shared their preferences for opt-out procedures.

IRB representatives were also supportive of an early enrollment strategy, and investigators and study coordinators indicated that the approach would not be burdensome.

CTTI Study Shows More Efforts Are Needed to Stimulate Pediatric Antibacterial and Antifungal Drug Trials

Posted on September 5, 2018August 12, 2021 by Lorri Bingham

In an article recently published in Pediatrics, CTTI researchers assess the impact of the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA) on pediatric antibacterial and antifungal drug trials.

The study, which evaluated pediatric trials conducted between 2007 and 2017, found that nearly two-thirds of pediatric antibacterial and antifungal drug trials were conducted under BPCA or PREA. These trials were more likely to collect pharmacokinetic data and report results than non-BPCA/PREA trials.

However, the overall number of pediatric antibacterial and antifungal drug trials was low, representing less than 1 percent of pediatric trials overall. These trials also rarely enrolled infants up to 30 days old.

The findings show that, while federal legislation is likely having an impact on pediatric antibacterial and antifungal drug trials, more efforts are needed to stimulate these trials and improve the reporting of results.

This study was conducted as part of CTTI’s ABDD Peds Trials Project, which focuses on creating efficient, evidence-based processes to accelerate the development of safe and effective pediatric antibacterial drugs.

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