Comparative Effectiveness Research in Pediatric Rheumatic Diseases: Leveraging CA

DESCRIPTION (provided by applicant): The study of childhood rheumatic conditions has been challenged by disease heterogeneity, poor understanding of underlying pathophysiology, small sample sizes, and limited resources. Development of the Childhood Arthritis and Rheumatology Research Alliance (CARRA) has created the opportunities multi-center retrospective and prospective studies of high impact. The initiatives described below will develop the platform for comparative effectiveness research, creating the evidence base for better care, which will translate into reduced health care costs, enabling American businesses to be more competitive globally. These initiatives will stimulate the American economy through employment of clinical coordinators and other research personnel protecting investigators'time and enable employment of clinical pediatric rheumatologists, both centrally and at peripheral sites. The long-term goal is to improve the health related outcomes of children with rheumatic diseases by entering children into treatment protocols and registries which will enable in and of itself improve care, as well as enable the performance of ongoing comparative effectiveness studies of therapies for Pediatric Rheumatic Diseases. The short- term goal of the current application is to create the platform to perform these studies by leveraging the prior work of CARRA and its unique network structure. This proposal will create a sustainable pediatric rheumatology registry. This will be an ongoing resource for comparative effectiveness studies. Data elements will inform the following domains and will allow varying levels of data collection (demographics, core disease features, treatment, co-morbidities, laboratory and radiology usage and data, serious adverse events, patient reported outcomes and important medical events). The following strategies will be developed to efficiently and rapidly develop registries for each major Pediatric Rheumatic Disease: a system for pooling existing data from distinct data sets;efficient and incentivized mechanisms to collect data from patients screened for clinical trials, but not enrolled and patients followed for routine clinical care. Public Private Partnerships and mechanisms for conducting cost effectiveness studies in pediatric rheumatology will be explored. Best care protocols for the treatment of Pediatric Rheumatic Diseases will be developed. Protocols will be initially developed in four disease areas: Juvenile Idiopathic Arthritis [JIA], Systemic Lupus Erythematosus [SLE], Juvenile Dermatomyositis [JDM] and Localized Scleroderma. Once developed, these protocols will be disseminated, and implemented by the physicians who treat children and adolescents with rheumatic diseases, ensuring that all children with these diseases have the opportunity to enroll in a treatment protocol and enabling uniform data-collection and interventions. Consensus methodology (Delphi Technique and Nominal Group Technique) will be used to identify data elements to be used in novel methods of analysis for comparative effectiveness research, specification of clinical outcome measures and treatment for each disease protocol developed. Each protocol will have associated IRB and informed consent documents, case report forms and other regulatory documents for immediate implementation and data collection at multiple sites. Development of this rich resource will enable analysis of various treatments to determine which were most effective and safe, and drive future iterations of the protocols. In this application we propose to 1) to develop the platform for studies of comparative effectiveness utilizing data collected from existing registries, ongoing clinical trials and newly developed treatment protocols and 2) develop best care protocols based on currently available literature, experience of CARRA practitioners and consensus techniques. With completion of the project CARRA will be able to: 1) Perform comparative effectiveness research to define optimal care in Pediatric Rheumatic Diseases (PRD), 2) Conduct research that will translate to prevention, better treatment, and cure of PRD, 3) Investigate methods to improve quality of life in children with PRD, 4) Promote a culture where ultimately all children with PRD will be able to participate in research protocols. With 95% of members working at academic centers, knowledge gained from research investigations will be rapidly disseminated and translated into practice.