The US Food and Drug Administration has awarded the Institute for Advanced Clinical Trials for Children (I-ACT for Children) a grant to improve clinical trials of new drugs and devices for children. The award establishes an alliance among I-ACT for Children and PEDSnet, the James M. Anderson Center for Health Systems Excellence, Critical Path Institute and the National Capital Consortium for Pediatric Device Innovation, to work together with leading children's institutions and health systems to address key gaps and challenges in pediatric clinical trials. The award provides $1 M in fiscal year 2017 funds with the potential for $1 M each year for an additional 4 years contingent on annual appropriations and the availability of funding to support the development of the scientific infrastructure needed to plan and execute pediatric clinical trials through collaboration with stakeholders from academia, industry, parent/patient advocacy groups, and regulatory agencies.
"This is a first-in-kind opportunity to improve the use of medicines in children. I-ACT for Children will bring together stakeholders from academia, industry, patient/parent advocacy groups and government agencies to facilitate pediatric clinical trials. The development of biomarkers, outcome assessments, data standards, and training in regulatory ready studies will improve the efficiency of pediatric clinical trials with the potential to provide safe and effective therapies for children," said Janet Woodcock, M.D., Director of the Center for Drug Evaluation and Research at the U.S. Food and Drug Administration.
The I-ACT for Children network will emphasize innovative trials design, improved operational efficiency and trial feasibility, shared best practices, early stakeholder and community engagement, and integrative approaches to pediatric studies. I-ACT for Children, an independent 501(c)3 non-profit, with expertise in pediatric medical product development and public-private collaboration serves as the strategic lead for the award. The Institute will continue the public-private collaboration that began in 2016 through the Pediatric Trials Consortium which launched the effort to build a sustainable clinical trials infrastructure focused on children's need for innovative medical products. The work of this consortium is described in its Advisory Report which was cited in the request for proposal as a foundational tenet for applicants.
"Through this cooperative agreement we and our alliance partners will work to establish a sustainable infrastructure for pediatric clinical trials that is collaborative, child-centered and promotes regulatory science and innovative methodologies to improve research on the safety and effectiveness of new medicines and devices for children," said Edward Connor, MD, MBE, FAAP Chairman and President of I-ACT for Children, Emeritus Professor of Pediatrics, Microbiology, Immunology, and Tropical Medicine, George Washington School of Medicine and Children's National Health System and principal investigator for the grant. "We are honored by FDA's confidence in our approach for a new paradigm in pediatric clinical trials."
I-ACT for Children works to optimize and accelerate biomedical innovation and catalyze improvements in the quality and timely completion of global pediatric studies to address the gap in evidence for therapeutics in children. Strategic partners in this grant include PEDSnet and the Anderson Center for Health Systems Excellence that serve as the network's Data and Learning Core. PEDSnet institutions share common data elements for >5.3 million children, and the Data and Learning Core includes learning health networks in >450 centers at 225 medical institutions, representing families and children from diverse socio-demographic and clinical backgrounds. The Critical Path Institute will serve as the Regulatory Science Core and the National Capital Consortium for Pediatric Device Innovation, an FDA-funded initiative, will act as the Device Core.
"This award brings together leaders in pediatric trials innovation from across the public and private sectors in an exciting new clinical trials network and creates an environment of continuous learning that leverages real world data to inform trial design and feasibility," said Dr. Christopher Forrest, MD, PhD, FAAP Professor of Pediatrics, Health Care Management, and Biomedical and Health Care Informatics, Perelman School of Medicine, University of Pennsylvania and Children's Hospital of Philadelphia, principal investigator, PEDSnet, and co-investigator for the grant.
About the Institute for Advanced Clinical Trials for Children (I-ACT for Children)
The Institute for Advanced Clinical Trials for Children is a new independent, nonprofit organization that works with public and private stakeholders to improve the planning and completion of pediatric clinical trials. It facilitates advanced research and education directed at improving the timeliness, quality and medical impact of clinical trials of innovative therapeutics on child health. The Institute was established by Critical Path Institute with advice and guidance from its Pediatric Trials Consortium that includes more than 30 diverse organizations, including patients/parents, government agencies, professional organizations, academia, the biopharmaceutical industry and international experts. I-ACT for Children is headquartered in Rockville, MD. For more information, visit https:/
PEDSnet is a large, national community of hospitals and healthcare organizations, researchers, clinicians, patients and families dedicated to improving the health and lives of children by identifying the most important research questions that can reduce children's suffering and support their healthy development. They have formed a multi-specialty network that conducts observational research and clinical trials across multiple children's hospital health systems governed by parents and senior leaders. Through their work, PEDSnet has produced reusable and expandable governance, logistical, informatics, regulatory, scientific, and training resources. Furthermore, across its 8 founding institutions, PEDSnet created a longitudinal data resource that dates back to 2009, cuts across all pediatric diseases and includes all pediatric specialties. For more information, visit https:/
About the Anderson Center for Health Systems Excellence
To elevate Cincinnati Children's focus on quality improvement, and spread the impact of their improvements in children's health, the James M. Anderson Center for Health Systems Excellence was created in 2010 and is led by co-directors Peter Margolis, M.D., PhD, and Steve Muething, M.D. The Anderson Center challenges conventional thinking in children's health care by identifying best practices, connecting research in one area with its practical application in another and collaborating and partnering with outside organizations to bring new knowledge to healthcare. For more information, visit https:/
About Critical Path Institute (C-Path)
Critical Path Institute is an independent, nonprofit organization established in 2005 with public and private philanthropic support from the Arizona community, Science Foundation Arizona, and the US Food and Drug Administration (FDA). C-Path's mission is to catalyze the development of new approaches that advance medical innovation and regulatory science, accelerating the path to a healthier world. An international leader in forming collaborations, C-Path has established 12 global, public-private partnerships that currently include over 1,450 scientists from government and regulatory agencies, academia, patient advocacy organizations, and dozens of major pharmaceutical companies. C-Path is headquartered in Tucson, Arizona. For more information, visit https:/
About the National Capital Consortium for Pediatric Device Innovation (NCC-PDI)
The National Capital Consortium for Pediatric Device Innovation (NCC-PDI) is an FDA funded consortium, led by the Sheikh Zayed Institute for Pediatric Surgical Innovation at Children's National Health System and the A. James Clark School of Engineering at the University of Maryland. It was formed in September 2013 through the FDA's Pediatric Device Consortia Grant Program to provide infrastructure support and expert consultation on pediatric medical device development throughout the development lifecycle -- concept formation, prototyping, preclinical, clinical, manufacturing, marketing, and commercialization - to help in bringing devices to children faster. Since inception, NCC-PDI has supported 67 pediatric devices, and the companies and research labs owning these devices have collectively raised over $55 million in follow-on early-stage funding.
-Funding for this work was made possible, in part, by the Food and Drug Administration through grant (1 U18 FD 006297). Views expressed in written materials or publications and by speakers and moderators do not necessarily reflect the official policies of the Department of Health and Human Services; nor does any mention of trade names, commercial practices, or organization imply endorsement by the United States Government.