Advancing Pediatric Clinical Trials: Examples of Our Work
One of our major goals is to build infrastructure through pre-competitive projects that will develop tools and systems to benefit all pediatric clinical trials.
I-ACT for Children has a growing network of pre-qualified pediatric clinical sites that can be activated quickly for our members. A map of the current site network can be found here: https://www.iactc.org/i-act-for-children-site-network/. Each site has been vigorously vetted to become an I-ACT for Children network site. Our network sites can access a centralized IRB and are connected through a site-network portal where they can share best practices, education and tools.
While many GCP training programs exist for PIs and research staff, they typically have little information specific to pediatric research. This has forced sponsors to spend time and money developing their own training material – resulting in study sites having to complete multiple programs. In addition to wasting resources and increasing costs, this repetitive training contributes to delays in trial start-up.
I-ACT for Children is collaborating with the Duke Clinical Research Institute to develop a pediatric-focused GCP training supplement that will be accepted by the pediatric research community and industry sponsors, with the goal of eliminating the need for multiple trainings and speeding the time to trial enrollment.
Although successfully used in pediatric hospital operations and clinical subspecialty networks for years, quality improvement approaches have not been applied within a multi-specialty pediatric research network. Yet the use of QI measures has significant potential to enhance clinical trials in children by speeding start-up times, increasing enrollment and improving patient retention rates.
I-ACT is collaborating with the University of Cincinnati’s James M. Anderson Center for Health Systems Excellence to implement a QI system within our site network. This system will capture clinical trial operational data from each site that can be analyzed to determine where best practices are happening and where processes could improve. These data then will be shared and applied across the network to create a continuous learning environment designed to maximize trial speed, quality and efficiency.
Only 20 percent of devices are approved for use in pediatric patients. The pediatric device space is smaller than the pharmaceutical field, so it faces even greater challenges in patient recruitment and study completion.
I-ACT is working with device experts at Children’s National Health System to develop guidance documents for industry and research centers that will provide best practices for designing, reviewing and completing pediatric device trials. The goal: accelerate the development of pediatric medical devices so that the devices used in children have actually been designed for them.
- More rapid completion of testing and approval of new DMD therapies
- Reduced clinical trial start-up and execution time
- Enhanced patient experience, reduced number on placebo
- Potential for rapidly testing combination therapies
I-ACT for Children co-hosted an FDA workshop on May 15, 2019, with the Duke Clinical Research Institute to understand the gaps in knowledge related to e-cigarette use among youth and seek recommendations on where future FDA resources should be directed to fill those gaps. Click here for more information.
I-ACT for Children hosted its 1st annual Pediatric Research Innovation Forum on Oct. 15-16, 2019, which focused on the inclusion of adolescents in adult clinical trials. The goal of the meeting was to drive innovation in this area by bringing key stakeholders together to agree on what’s known, identify continuing challenges and propose solutions to address those challenges.
There was robust engagement throughout the meeting and participation from multiple stakeholder groups – including leading scientific researchers, FDA representatives, patient advocates and pharma company research leaders. The meeting drew High-level participants from across the United States as well as from Spain, England, Canada and Germany.
I-ACT for Children now will work with meeting participants to develop a recommendations document for publication and dissemination, which will be informed by the Working Group sessions held during the meeting. Click here for meeting materials.
We have completed a number of proprietary projects on behalf of our I-ACT for Children members. These include: