Program: Antibacterial Drug Development (ABDD)

About This Program

Project:

Peds Trials

overview icon Overview

Addressing challenges in pediatric clinical trials for antibacterial drugs

Developing new antibacterial drugs is challenging, and conducting antibacterial trials in pediatric populations is even more complex. Despite the fact that pediatric trials are required for new drugs, these trials can be especially difficult to design, enroll, and complete. Children respond differently to medications due to their developing organs and body systems, and these trials are urgently needed to inform dosing, efficacy, and safety.

CTTI has conducted research to identify the challenges of conducting pediatric antibacterial trials, and with the input of diverse stakeholders, has developed actionable recommendations to address these challenges. Our recommendations offer practical, evidence-based strategies that can be applied by research sponsors, investigators, and site staff to improve the quality and efficiency of pediatric antibacterial trials. These include tips on determining the timing of pediatric trials, methods for streamlining trial design to decrease burden on sites and families, special considerations for conducting trials with neonates, approaches for improving the informed consent process, and ways to increase engagement with healthcare providers. Although developed in the context of antibacterial drug development, many of the recommendations can be applied to improve pediatric clinical trials in multiple therapeutic areas.

quick links icon Quick Links to Top Deliverables

Discover Additional Deliverables Generated From This Project

Pediatrics

Pediatric Antibacterial and Antifungal Trials From 2007 to 2017

Contemporary Clinical Trials Communications

Facilitators and barriers to the successful implementation of pediatric antibacterial drug trials: Findings from CTTI's survey of investigators

Journal of the Pediatric Infectious Diseases Society

Advancing Pediatric Antibacterial Drug Development: A Critical Need to Reinvent our Approach, published in Contemporary Clinical Trials

Press Release

CTTI Releases New Recommendations to Improve Studies of Antibacterial Drugs for Children

Webinar

CTTI Recommendations from the Antibacterial Drug Development (ABDD) Peds Trial Project

Expert Meeting Materials

Click to view materials, including presentations & summary, for the CTTI-hosted expert meeting, Improving Pediatric Trials in Antibacterial Drug Development

Poster

Pediatric Trials in Antibacterial Drug Development: Findings from the Clinical Trials Transformation Project, presented at the National Cystic Fibrosis Foundation Meeting

Poster

Pediatric Trials in Antibacterial Drug Development: Findings from the Clinical Trials Transformation Project, presented at the Pediatric Academic Societies (PAS) 2016 Meeting

in detail icon In Detail

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.

CLICK HERE to read more about issues in ABDD.

ABDD Program: Addressing scientific and operational challenges in pediatric clinical trials for antibacterial drugs (2011-ongoing)

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

  • Identify scientific and operational issues in pediatric antibacterial drug trial recruitment
  • Develop recommendations to address scientific and operational challenges in the design and conduct of antibacterial drug clinical trials in children.

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

Sponsors, investigators, and sites will be better able to conduct pediatric antibacterial drug trials, leading to evidence that can improve care for children.

  • Stakeholder interviews*
  • Electronic survey*
  • Review of antibacterial pediatric drugs trials in ClinicalTrials.gov (utilizing using the AACT Database) to determine the state of these trials*
  • Expert meeting

*For a summary, check out this poster that was presented at the Pediatric Academic Societies 2016 Conference.

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 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

Rolesort descending 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