Mission, Vision & Values


Our mission is to expedite the development and approval of safe, effective pediatric therapeutics to ensure every child has access to the treatment they need with the same urgency afforded to adults. Our work encompasses research, advocacy, education, and collaboration, all targeted towards improving pediatric treatment availability and safety standards.


Our vision is a world where every child has access to safe and effective treatments that address their needs.  Through our efforts, we envision an ecosystem that fully recognizes and meets the unique treatment needs of children, fostering a healthier future for all our children.


  • Children’s unmet medical needs drive our agenda,and all of our work supports the development of innovative medicines and devices for children that are most needed to improve and preserve children’s health and wellness.
  • We are an independent voice in innovative treatment development, focused on optimal impact on child health.
  • We are collaborative and synergistic with other elements of the clinical research ecosystem– finding common ground and aligning common values, with children at the center.
  • Our standards are grounded in regulatory requirements, providing the basis upon which we work with others to deliver scientifically strong, clinically relevant and ethically sound advice and manage high-quality, timely conduct of clinical trials.
  • Because children are waiting, we must shorten the time it takes to bring innovative treatments to them. Streamlining operations, reducing burdensome processes, researching innovative approaches, and optimizing the use of resources are fundamental principles that guide us.

Little Patients Have Little Patience

Act Now