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.
Building a Growing Network of Clinical Trial-Ready Sites
Because the number of children eligible for clinical trials is much lower than that of adults, up to 50 trial sites can be required to enroll enough children to complete just one pediatric clinical trial. At the same time, multiple drug developers may use the same sites – requiring research centers to perform often duplicative processes for each drug sponsor. These challenges can significantly delay clinical trials, create time-consuming administrative burdens and increase costs across the board.
I-ACT for Children has a growing network of pre-qualified pediatric clinical trial-ready 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 through a four-step process to become an I-ACT network site. Our network sites can access a centralized IRB and are connected through an I-ACT portal where they can share best practices, education and tools. In 2019, we will have a central Data Monitoring Committee in place, as well as master budgeting agreements and other standardized processes designed to shorten trial start-up time and reduce both costs and administrative burden.
Creating Harmonized Pediatric Good Clinical Practice (GCP) Training
While many GCP training modules exist for PIs, research staff, there is no commonly accepted GCP training that focuses on pediatrics. In the past, 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 is another factor that has slowed trial start-up time.
I-ACT is collaborating with the Duke Clinical Research Institute to develop a harmonized, pediatric-focused GCP training module that will be accepted by the pediatric research community and industry sponsors, eliminating the need for multiple trainings and speeding the time to trial enrollment.
Launching a Quality Improvement System to Measure Pediatric Trial Efficiency
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.
Enhancing the Development of Pediatric Devices
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.
We have completed a number of proprietary projects on behalf of our I-ACT members. These include:
Adaptive Trial Design
We reviewed study protocols and worked with our experts to develop an adaptive trial design for a cardiovascular indication, recommending elements that would reduce sample size and shorten the trial duration.
Site Identification and Recruitment
Leveraging our network of pre-qualified trial sites, we have recruited critical sites for our members' ongoing clinical trials. The goal: help members achieve their recruitment targets more quickly and complete their trials ahead of schedule. Therapeutic areas we have been working in include the psychiatric and metabolic spaces.