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.
Are prescribed off-label, without adequate data.
Innovative drug development in pediatrics remains slow and inefficient.