We propose to create a national, pediatric-specific learning health system (LHS) composed of a newly formed multi-institutional clinical data research network (CDRN) (called PEDSnet); three condition-specific patient-powered research networks (PPRNs) focusing on pediatric inflammatory bowel syndrome, hypoplastic left heart syndrome, and childhood obesity; and two national data partners. An LHS is organized around communities of patients, families, clinicians, and researchers who work together to purposefully integrate knowledge generation (research) with knowledge dissemination and implementation at the point of care (quality improvement). Its goal is to provide information about available, reasonable alternatives that will allow patients and families to make informed, shared decisions with their healthcare team in ways that lead to improvements in outcomes. The PEDSnet CDRN includes eight of the nation's largest children's hospital health systems:
- Children's Hospital of Philadelphia,
- Cincinnati Children's Hospital Medical Center,
- Children's Hospital Colorado,
- Nemours Children's Health System,
- Nationwide Children's Hospital,
- St. Louis Children's Hospital,
- Seattle Children's Hospital, and
- Boston Children's Hospital.
These institutions provide services to 2.1 million children per year (2.8% of the nation's children), providing a large and diverse population for conducting pediatric research. The PPRNs include two established networks—ImproveCareNow (16,000 pediatric inflammatory bowel disease patients), and the National Pediatric Cardiology Quality Improvement Collaborative (1,000 hypoplastic left heart syndrome patients—and a newly established network for childhood obesity. The national data partners are Express Scripts (a pharmacy benefit management company) and IMS Health (a data analytics company). The goal of the proposed project is to transform pediatric health care and children's health in the nation by developing an extensible and efficient infrastructure that enables all participants to collaborate in the work of producing new knowledge and improving health and care delivery. Our specific aims are to:
- engage patients/families, clinicians, researchers, and system executives in the creation of the pediatric LHS;
- form governance structures that provide operational and strategic oversight of the LHS;
- establish an interoperable digital infrastructure to support learning (research, dissemination, and implementation), recruitment, and engagement of stakeholders;
- create a recruitment infrastructure to support prospective research studies; and
- develop policies and procedures (e.g., a central Institutional Review Board [IRB]) that facilitate collaboration.
Our application benefits from robust pre-existing resources and a unique history of collaboration by children's hospitals that has fundamentally reshaped outcomes for previously fatal diseases, such as cystic fibrosis and many childhood cancers. Building on this history, we created, with the Agency for Healthcare Research and Quality and the National Institutes of Health (NIH) sponsorship, an operational prototype of the LHS for pediatric inflammatory bowel disease, which over the past 4 years has increased rates of remission (no disease activity) from 55% to 77% of patients in the network. The pediatric institutions participating in the newly formed CDRN have already collaborated on electronic health record (EHR)-based data sharing studies. With NIH funding, we have launched a national Pediatric Terminology Project, which is harmonizing clinical concepts across conditions and specialties. These successes demonstrate both the will and capacity of participating institutions to develop a national pediatric LHS that addresses fundamental questions of clinical effectiveness for children and their families, particularly for individuals affected by serious, and generally rare, illness that persists into adulthood. The individuals and organizations collaborating in this effort have the expertise needed to address questions of interoperability, patient engagement, governance, and other factors critical to developing a capable national infrastructure that will enable learning across pediatric conditions and stakeholders, harness the motivation and expertise of engaged participants to contribute to advancing knowledge and improving child health, embed research within healthcare delivery, and efficiently implement and disseminate new approaches to care based on research results, ultimately leading to transformations in children's health outcomes.