Abstract
ImportanceSpecialized research networks are essential to achieve drug approvals for rare pediatric diseases. Such networks help realize the potential of global legislation enacted upon the recognition that most children are treated with drugs whose most beneficial dose and regimen have not been established in pediatric patients. The Pediatric Rheumatology Collaborative Study Group (PRCSG) is a North American clinical trials network that is specialized in the performance of clinical trials of new therapies for pediatric populations with rheumatic diseases. This review provides an overview of the strategies employed by this research network to achieve drug and biologic approvals for children with pediatric rheumatic diseases, particularly juvenile idiopathic arthritis.ObservationsClinical trial conduct in rare pediatric diseases has required global recruitment. Supported or led by the PRCSG, highly responsive, validated, composite measures have been established to assess drug efficacy. For pediatric orphan diseases with high disease burdens, specialized investigative sites and study designs are needed to complete adequately powered trials at the high standard necessary to enable drug labeling by regulatory agencies. Novel trial designs have been utilized for more efficient testing of innovative drug candidates. All these have been developed or co-developed by the PRCSG research network.Conclusions and relevanceSpecialized research networks in pediatric rheumatology, such as the PRCSG, have changed the landscape of available therapies and improved overall disease outcomes for children with pediatric rheumatic diseases.
Highlights
There remains a profound unmet medical need for the effective and safe treatment of pediatric rheumatic diseases
A recent review by the European Medicines Agency (EMA) and Food and Drug Administration (FDA) confirmed that the existence of pediatric research networks, such as the Pediatric Rheumatology Collaborative Study Group (PRCSG), is of utmost importance to realize the potential of this new pediatric legislation [2, 8]
Since the 1970’s, the PRCSG has contributed to the methodology of performing trials in pediatric rheumatic diseases
Summary
There remains a profound unmet medical need for the effective and safe treatment of pediatric rheumatic diseases. In the U.S, medication labeling and drug studies are governed largely by two sections of the FD&C Act: section 505A pertains to The Best Pharmaceuticals for Children Act (BPCA) [3] and section 505B to the Pediatric Research Equity Act (PREA) [4], respectively. Together these two laws encourage and/or require drug companies to study their products in children [5]. Almost all stakeholders involved, including leaders of the PRCSG, asserted that permanence of PREA and BPCA was an important aspect for future drug development for children Together, these laws resulted in the labeling of more than 600 products for pediatric indications, 149 of which occurred since the passage of FDASIA [7]. A recent review by the European Medicines Agency (EMA) and FDA confirmed that the existence of pediatric research networks, such as the PRCSG, is of utmost importance to realize the potential of this new pediatric legislation [2, 8]
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