Clinical Trial Design in Juvenile Idiopathic Arthritis
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Randomized clinical trials provide the gold standard evidence base to guide clinical practice. Despite major advances in trial design, pediatric clinical trials are still difficult to perform and pose unique challenges, including the need to consider the impact of developmental changes in trial design. Advances within pediatric rheumatology combined with the need to comply with legislative requirements have driven new approaches to performing pediatric clinical trials such as utilization of large research networks, incorporation of patient and family stakeholders in the planning and implementation of clinical trials, and the development of novel trial designs. The expansion of available biological therapeutics that now includes biosimilar drugs highlights the important and difficult balance of providing new and cost-effective drugs to children while ensuring safety in a vulnerable population. Future advances in juvenile idiopathic arthritis (JIA) clinical trials will likely be the application of precision medicine based on biologic, rather than phenotypic, classification of JIA, with improved understanding of pediatric clinical pharmacology. Clinical trial simulations and comparative effectiveness studies are important supplements to traditional clinical trials, permitting efficient studies and results that are more generalizable.
Michael Cohen-Wolkowiez receives support for research from the National Institutes of Health (1R01-HD076676-01A1), the National Institute of Allergy and Infectious Disease (HHSN272201500006I and HHSN272201300017I), the National Institute of Child Health and Human Development (HHSN275201000003I), the Biomedical Advanced Research and Development Authority (HHSO100201300009C), and Industry for drug development in adults and children.
Compliance with Ethical Standards
Stephen J. Balevic receives salary and research support from the National Institute of General Medical Sciences and the National Institute of Child Health and Human Development (2T32GM086330-06). The National Institutes of Health sponsor Open Access. Laura E. Schanberg receives research support from the National Institutes of Health (5R01-AR063890-02), Patient-Centered Outcomes Research Institute (CER-1408-20534 and PPRN-1306-04601), the National Institute of Arthritis and Musculoskeletal and Skin Diseases (1U19AR069522-01), and participates in the Data Safety and Monitoring Board for Sanofi, and the Swedish Orphan Biovitrum AB. Mara L. Becker and Michael Cohen-Wolkowiez have no conflicts of interest that are directly relevant to this article. This article did not involve the use of human participants or animals.
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