Advancing Pediatric Clinical Trials: Examples of Our Work
COVID-19 research is moving quickly, and I-ACT for Children is on the front lines advocating for the research needed to develop agents that are proven safe and effective for children.
The COVID-19 Emergency Access Program allows all biopharmaceutical and other companies who are developing COVID-19 treatments and vaccines to use the organization’s pediatric research network to conduct their pediatric trials. Under the new emergency program, non-member companies can work with I-ACT for Children to plan and execute COVID-19 clinical trials and access a global research network designed to generate regulatory-grade scientific data.
Learn more about our Emergency Access Program by clicking here.
On May 28, I-ACT for Children held a virtual workshop – “Developing Pediatric Treatments for COVID-19” – with regulators, biopharma developers and experts on the front lines of treating children with COVID-19. Speakers shared the latest on the clinical presentation of COVID-19 in children, the state of COVID-19 therapeutic research and innovative approaches to collecting the data required to advance safe and effective COVID-19 therapies for children. More than 300 people participated in the workshop. Recommendations from the workshop have been submitted for publication consideration to a peer-reviewed journal. The workshop agenda, presentations and a recording of the meeting can be found here.
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.
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 for Children has launched the pilot phase of our quality improvement initiative at the first 17 sites in our network. This pilot phase of the Pediatric Improvement Collaborative for Clinical Trials & Research (PICTR®) program will assess the feasibility of collecting QI data across our broader site network.
In 2020, we will work with Dr. Peter Margolis and his team at the James M. Anderson Center for Health Systems Excellence at Cincinnati Children’s Hospital Medical Center to expand the program to 40 I-ACT for Children network sites. PICTR® 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
A stakeholder meeting was held in 2019 to gather input for the protocol from parents, researchers, clinicians and drug developers; complete meeting information can be found here. I-ACT, PPMD and C-Path will meet with FDA in February to seek feedback before finalizing the protocol.
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.
I-ACT for Children has launched a U.S. mentoring program within our site network that pairs seasoned research professionals with those who are newer to the field of pediatric research and want to advance their knowledge and experience. The goal of the program is to support and expand the next generation of pediatric clinical trial staff, so that more children have access to high-quality pediatric trials and to medicines that have been proven safe and effective in this population.
Research staff at I-ACT for Children network sites are eligible for the program. Those with five or fewer years of experience may apply to be a mentee; those with more than five years of experience are eligible to be mentors.
The program launched in January 2021 with its first group of 13 pairs of mentors and mentees, who will participate in the program for one year. (They can choose to continue the relationship informally after the one-year program.) Pairings were prioritized by institutional role and sub-specialty area and each participant was paired with someone from a different institution.
The program will expand to include our Australian sites by Q3 2021. The application process for the 2022 program will launch in Q4 of 2021.
We have completed a number of proprietary projects on behalf of our I-ACT for Children members. These include:
In 2019, the FDA’s Center for Drug Evaluation and Research approved 48 novel medicines. Of the 46,391 study participants in the clinical trials that supported those approvals, only 9% were black/African American and only 18% were Hispanic (FDA: 2019 Drug Trials Snapshots Summary Report).
To address this issue, I-ACT for Children has assembled a group of experts to review clinical trial protocols and provide related counsel to maximize the inclusion of a diverse participant population. This supports our member companies’ diversity goals by helping to ensure that pediatric studies better reflect the populations their medicines may eventually treat.
Panel members can be convened for group discussion and consult or engaged individually as needed. This process is expected to take approximately 2 weeks to complete from the time a trial sponsor requests a consult to the time a final set of recommendations is provided. The preferred timing for this consultation is during the early stages of protocol development, but can be provided at any stage in a trial’s development or conduct.
For more information, contact I-ACT Chief Medical Officer Dr. Gary Noel.