Medidata's Cloud and Mobile Technology Selected to Drive Collaborative, 10-Year Research in Pediatric Rheumatology February 23, 2015 08:30 AM Eastern Standard Time NEW YORK--(BUSINESS WIRE)--Medidata (NASDAQ:MDSO), the leading global provider of cloud-based solutions for clinical research in life sciences, today announced that it has been selected to support the Childhood Arthritis and Rheumatology Research Alliance (CARRA) Registry, a 10-year project to collect comprehensive data on pediatric rheumatic diseases and their treatments. "This is a critical next step in understanding how to best apply available therapies and improve the systems for studying the safety and efficacy of treatment options for childhood-onset arthritis." "While we've made huge progress in the last decade, 300,000 children and families in the US alone are affected by rheumatic diseases," said Laura Schanberg, MD, professor of pediatrics at Duke University and CARRA president and the principal investigator for the CARRA Registry, an observational study of children and adolescents with major rheumatic diseases. By powering data capture for the scalable informatics infrastructure of the CARRA Registry, the Medidata Clinical Cloud® will help CARRA develop a resource for patients, physicians and researchers seeking to learn more about pediatric rheumatic diseases, and, ultimately, drive better diagnostic and treatment approaches. In support of its long-term research efforts, the registry is leveraging Medidata's cloud-based technology for electronic data capture and management (Medidata Rave®) and mobile solution for patient-direct data capture (Medidata Patient Cloud®). A mobile app unified with Medidata's platform, Patient Cloud brings new efficiencies to the administration of electronic patient-reported outcomes (ePROs) in clinical trials and registries. The mobile app provides electronic patient questionnaires and diaries in a model that simplifies the process for both patients and researchers. "Using Medidata's platform in combination with the open-source, i2b2 federated clinical research data warehouse platform already in use for the CARRA Registry, this post-marketing surveillance network will collect at least 10 years of comprehensive information, including detailed safety and treatment data, on pediatric rheumatic disorders in the US and Canada," said Marc Natter, MD, the CARRA Registry's director of informatics development and instructor in the Boston Children's Hospital Informatics Program. "This is a critical next step in understanding how to best apply available therapies and improve the systems for studying the safety and efficacy of treatment options for childhood-onset arthritis." Dedicated to advancing the health and quality of life of children living with rheumatic disease and arthritis, CARRA was formed by pediatric rheumatologists seeking to answer critical clinical research questions. "Juvenile idiopathic arthritis and other pediatric inflammatory disorders result in persistent joint pain, swelling and stiffness of joints, decreased activity, and potentially growth and eye problems," Schanberg added. "Children lose days from school, parents lose days from work and quality of life suffers greatly. We believe that simplifying data capture and patient participation processes will catapult the field of pediatric rheumatology research to the level of performance necessary to realize dramatic improvements in outcomes and quality of life for all children with rheumatic disease." Glen de Vries, Medidata's president, said: "We're excited to be partnering with CARRA on this observational study and proud that our technology platform is helping to further the alliance's important mission to prevent, treat and cure arthritis and other rheumatic diseases in children and adolescents. All of us at Medidata share CARRA's commitment to fostering, facilitating and conducting high-quality research that advances the development of new, enhanced diagnostic and treatment approaches."
About CARRA CARRA is a North American non-profit research organization of more than 400 pediatric rheumatologists, researchers and research coordinators at more than 100 sites (95% of all pediatric rheumatologists/sites in North America) who are working together to find treatments for juvenile idiopathic arthritis and other pediatric rheumatic diseases in children. The CARRA Registry is a cornerstone of CARRA and provides disease and treatment data on children with a variety of rheumatic diseases. CARRA aims to make it possible for all affected children in North America to have the opportunity to participate in meaningful and high quality clinical and translational research. CARRA researchers have been awarded over $40 million in research funding over the last 10 years. In addition to Dr. Schanberg, the CARRA Executive team includes Yukiko Kimura, MD, Hackensack University Medical Center, Norman Ilowite, MD, Montefiore Medical Center, and Robert Fuhlbrigge, MD, PhD, Boston Children's Hospital.
About Medidata Solutions Medidata is the leading global provider of cloud-based solutions for clinical research in life sciences, transforming clinical development through its advanced applications and intelligent data analytics. The Medidata Clinical Cloud® brings new levels of productivity and quality to the clinical testing of promising medical treatments, from study design and planning through execution, management and reporting. We are committed to advancing the competitive and scientific goals of global customers, which include over 90% of the top 25 global pharmaceutical companies; innovative biotech, diagnostic and device firms; leading academic medical centers; and contract research organizations.
Juvenile rheumatoid arthritis (JRA) Etiology and Pathophysiology The etiology and pathogenesis of JIA are not completely understood. Genetic susceptibility plays a major role, but there is significant overlap between loci associated with JIA and those associated with other autoimmune diseases. JIA is a genetically complex disorder in which multiple genes are important for disease onset and manifestations. The IL2RA/CD25 gene has been implicated as a JIA susceptibility locus, as has the VTCN1 gene.[8] Associations have been found between specific HLA alleles and clinical subtypes of JIA (eg, HLA-A(*)02:06 with susceptibility to JIA accompanied by uveitis, and HLA-DRB1(*)04:05 with polyarticular JIA, in a Japanese cohort). Humoral and cell-mediated immunity are involved in the pathogenesis of JIA. T lymphocytes have a central role, releasing proinflammatory cytokines (eg, tumor necrosis factor–alpha [TNF-α], interleukin [IL]-6, IL-1) and favoring a type-1 helper T-lymphocyte response. A disordered interaction between type 1 and type 2 T-helper cells has been postulated. Studies of T-cell receptor expression confirm recruitment of T-lymphocytes specific for synovial nonself antigens. Evidence for abnormalities in the humoral immune system include the increased presence of autoantibodies (especially antinuclear antibodies), increased serum immunoglobulins, the presence of circulating immune complexes, and complement activation. Chronic inflammation of synovium is characterized by B-lymphocyte infiltration and expansion. Macrophages and T-cell invasion are associated with the release of cytokines, which evoke synoviocyte proliferation. A study by Scola et al found synovium to contain messenger ribonucleic acid (mRNA) for vascular endothelial growth factor and angiopoietin 1, as well as for their receptors, suggesting that induction of angiogenesis by products of lymphocytic infiltration may be involved in persistence of disease. Some pediatric rheumatologists view systemic-onset JIA as an autoinflammatory disorder, such as familial Mediterranean fever (FMF) or cryopyrin-associated periodic fever syndromes, rather than a subtype of JIA. This theory is supported by work demonstrating similar expression patterns of a phagocytic protein (S100A12) in systemic-onset JIA and FMF, as well as the same marked responsiveness to IL-1 receptor antagonists. FMF is associated with mutations in the MEFV gene; these mutations are associated with activation of the IL-1b pathway, resulting in inflammation. A study by Ayaz et al found an increased frequency of MEFV mutations in Turkish children who were diagnosed with systemic JIA[12] ; this study has not been replicated in other populations.
Source: Medscape