Pediatric Rheumatology Collaborative Study Group – over four decades of pivotal clinical drug research in pediatric rheumatology

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.

Safety and Effectiveness of Adalimumab in Patients With Polyarticular Course of Juvenile Idiopathic Arthritis: STRIVE Registry Seven‐Year Interim Results

“…STRIVE is a 10‐year, ongoing, multicenter, noninterventional, observational registry of children with active polyarticular‐course JIA treated with MTX or ADA ± MTX. Patients were enrolled in 16 countries at 92 centers of the Paediatric Rheumatology International Trials Organisation (18) and the Pediatric Rheumatology Collaborative Study Group (19). Children entered the MTX arm (i.e., patients treated with MTX alone or in combination with other synthetic DMARDs according to the local product labeling) or the ADA ± MTX arm (i.e., patients treated with ADA alone [ADA – MTX] or ADA in combination with MTX [ADA + MTX]).…”

Long-term outcomes in patients with polyarticular juvenile idiopathic arthritis receiving adalimumab with or without methotrexate

“…Children were enrolled from 31 centres in 8 countries, belonging to the Pediatric Rheumatology Collaborative Study Group 15 and the Pediatric Rheumatology International Trials Organization (PRINTO). 16…”

Maintenance of antibody response to diphtheria/tetanus vaccine in patients aged 2–5 years with polyarticular-course juvenile idiopathic arthritis receiving subcutaneous abatacept

“…This substudy of a 24-month, Phase III trial (NCT01844518) of SC abatacept in patients (cohort 1: 173 patients aged 6-17 years; cohort 2: 46 patients aged 2-5 years) with active pJIA and inadequate response/intolerance to ≥1 DMARD [6] was conducted across 48 centers worldwide by members of the Paediatric Rheumatology International Trials Organisation [10] and the Pediatric Rheumatology Collaborative Study Group [11]. Patients received weight-tiered weekly SC abatacept (10-< 25 kg [50 mg], 25-< 50 kg [87.5 mg]) for 4 months.…”

“…10 Center for Pediatric and Adolescent Medicine/Pediatric Rheumatology, University Hospital Heidelberg, Heidelberg, Germany. 11 Paris University, IMAGINE Institute, RAISE reference centre, Necker-Enfants Malades Hospital, Assistance Publique-Hopitaux de Paris, Paris, France. 12 Bristol-Myers Squibb, Princeton, NJ, USA.…”