Pre-competitive Pediatric Projects

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

Identified common obstacles that delay completion of regulatory requirements for recently approved antibacterials
Convened key stakeholders to prioritize critical needs of infants and children related to antibiotic drug resistance
Multi-stakeholder webinar in early/mid 2022 to focus on actionable solutions to challenges
Learn more by reading the article: Advancing Pediatric Antibacterial Drug Development: A Critical Need to Reinvent our Approach

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)