Using Registries to Identify Adverse Events in Rheumatic Diseases
Year: 2013
Type: article
Abstract: The proven effectiveness of biologics and other immunomodulatory products in inflammatory rheumatic diseases has resulted in their widespread use as well as reports of potential short- and long-term complications such as infection and malignancy. These complications are especially worrisome in children who often have serial exposures to multiple immunomodulatory products. Post-marketing surveillance of immunomodulatory products in juvenile idiopathic arthritis (JIA) and pediatric systemic lupus erythematosus is currently based on product-specific registries and passive surveillance, which may not accurately reflect the safety risks for children owing to low numbers, poor long-term retention, and inadequate comparators. In collaboration with the US Food and Drug Administration (FDA), patient and family advocacy groups, biopharmaceutical industry representatives and other stakeholders, the Childhood Arthritis and Rheumatology Research Alliance (CARRA) and the Duke Clinical Research Institute (DCRI) have developed a novel pharmacosurveillance model (CARRA Consolidated Safety Registry [CoRe]) based on a multicenter longitudinal pediatric rheumatic diseases registry with over 8000 participants. The existing CARRA infrastructure provides access to much larger numbers of subjects than is feasible in single-product registries. Enrollment regardless of medication exposure allows more accurate detection and evaluation of safety signals. Flexibility built into the model allows the addition of specific data elements and safety outcomes, and designation of appropriate disease comparator groups relevant to each product, fulfilling post-marketing requirements and commitments. The proposed model can be applied to other pediatric and adult diseases, potentially transforming the paradigm of pharmacosurveillance in response to the growing public mandate for rigorous post-marketing safety monitoring.
Source: PEDIATRICS
Authors: Geraldina Lionetti, Yukiko Kimura, Laura E. Schanberg, Timothy Beukelman, Carol A. Wallace, Norman T. Ilowite, Jane Winsor, Kathleen Fox, Marc Natter, John S. Sundy, Eric Brodsky, Jeffrey R. Curtis, Vincent Del Gaizo, Solomon Iyasu, Angelika Jahreis, Ann Meeker-O’Connell, Barbara B. Mittleman, Bernard M. Murphy, Eric D. Peterson, Sandra C. Raymond, Soko Setoguchi, Jeffrey N. Siegel, Rachel E. Sobel, Daniel Solomon, Taunton R. Southwood, Richard Vesely, Patience H. White, Nico M. Wulffraat, Christy I. Sandborg
Institutions: Stanford University, Hackensack University Medical Center, Duke Medical Center, University of Alabama at Birmingham, Seattle Children's Hospital, Yeshiva University, Children's Hospital at Montefiore, Duke University, Boston Children's Hospital, Harvard University, United States Food and Drug Administration, Genentech, National Institutes of Health, Arthritis Foundation, Lupus Foundation of America, Pfizer (United States), Brigham and Women's Hospital, University of Birmingham, European Medicines Agency, University Medical Center Utrecht
Language: English
Subfield: Toxicology
Domain: Life Sciences
Open Access status: green
Funders: Amgen, Bristol-Myers Squibb, Pfizer, Childhood Arthritis and Rheumatology Research Alliance, National Institutes of Health, Novartis Pharma, National Institute of Arthritis and Musculoskeletal and Skin Diseases, National Center for Advancing Translational Sciences, Duke Clinical Research Institute
OpenAlex ID: W2135835232