A Catalyst to Enable Innovation

A Catalyst to Enable Innovation

A Catalyst to Enable Innovation

A Catalyst to Enable Innovation


Connecting advocacy organizations, academia, regulatory and nonprofit organizations, I-ACT for Children develops and leads activities addressing topics aimed at improving drug development for children worldwide.

Since our inception, I-ACT for Children has engaged in a number of pre-competitive projects. Focus areas include inclusion of adolescents in adult clinical trials, COVID-related pediatric research priorities, innovations in pediatric inflammatory bowel disease (IBD) therapies and pediatric clinical trial design and development.

By engaging in pre-competitive projects, our goal is to develop innovative tools and systems that increase efficiency to benefit current and future pediatric clinical trials and researchers.

Pre-competitive Projects

Duchenne Muscular Dystrophy Platform Trial

  • A collaboration between Parent Project Muscular Dystrophy, the Critical Path Institute and I-ACT for Children to develop a platform trial that will increase access to therapeutic trials
  • The trial will study multiple drugs in multiple patient types with one common placebo-controlled arm
  • The goal: faster Duchenne muscular dystrophy (DMD) therapy testing and approval, as well as reduced trial startup and execution time

Closing the Gap in Labeling New Antibiotics for Children

Using Real-World Data to Advance Clinical Trials That Involve Critically Ill Newborns

  • Partnered with Critical Path Institute and International Neonatal Consortium to develop real-world database
  • Organized 20+ sites in the I-ACT for Children site network to contribute data from NICU patients

Addressing the Challenges of Approving and Labeling Innovative Therapies to Treat Children With Inflammatory Bowel Disease

  • Hosted series of webinars that identified challenges faced in the current regulatory pathway for approval and labeling of new therapies to treat children with IBD
  • Recommendations of key stakeholders, including patient advocates, patients, clinicians, academics, regulators and sponsors

Adolescent Participation in Adult Clinical Trials

  • Hosted Pediatric Research Innovation Forum focused on addressing the challenges of including adolescents in Phase 3 registration trials
  • Published consensus on Forum’s recommendations advocating for inclusion of adolescents in early clinical development (Therapeutic Innovation & Regulatory Science. 2021)

Little Patients Have Little Patience

Act Now