The Mission of I-ACT for Children is
To serve as a neutral and independent organization on behalf of children everywhere, bringing a dedicated voice to the advancement of new medicines and devices needed now and in the future. Our work is to engage public and private stakeholders through research and education to ensure that healthcare for children is continually improved by enhancing the awareness, quality and support for pediatric clinical trials.
Duchenne Platform Trial Stakeholder Meeting
I-ACT for Children and Parent Project Muscular Dystrophy co-hosted a stakeholder meeting on Sept. 9, 2019, to seek input on a draft platform trial protocol that is designed to allow multiple investigational treatments for Duchenne muscular dystrophy to be tested at one time – which could lead to more rapid completion of testing and approval of new therapies and minimize patients’ exposure to placebo. FDA’s Dr. Janet Woodcock and Dr. Billy Dunn were featured speakers at the meeting, which also was attended by researchers, parents, advocacy groups, industry representatives and other FDA scientists.
Pediatric Research Innovation Forum
I-ACT for Children hosted its 1st annual Pediatric Research Innovation Forum on Oct. 15-16, 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.
Pediatric Trials face many hurdles that adult trials do not. Because of this, pediatric trials often take longer and fail more frequently.
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Are prescribed off-label, without adequate data.
Innovative drug development in pediatrics remains slow and inefficient.
We act because every child with a medical need deserves the best chance possible. That chance depends on a commitment to innovation, quality and urgency in advancing medical therapies specifically for children.
How I-ACT for Children is Improving Pediatric Clinical Trials
Strategy & Planning
Strategy & Planning work ranges from innovative trial design (to help reduce sample size, exposure to placebo etc.) to use of real-world data (to assess feasibility & identify optimal research sites), in an effort to enhance the trial process from the very beginning.
Infrastructure & Trial Execution
The I-ACT for Children Site Network is an infrastructure of pediatric research sites that are expert in the conduct of innovative and efficient pediatric trials.
Tools, Capabilities, Best Practices
By implementing standard processes and practices such as budgeting templates, our network can shorten its study startup time when approached by a sponsor to conduct a clinical trial. Learn more about our site network.
Thought Leadership
We engaging stakeholders to generate discussion and launch groundbreaking initiatives that raise awareness of the need to advance medical therapies specifically for children and promoting a sense of urgency to achieve this goal.
