Using registries to identify adverse events in rheumatic diseases

Geraldina Lionetti, Yukiko Kimura, Laura E. Schanberg, Timothy Beukelman, Carol A. Wallace, Norman Todd 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. RaymondSoko Setoguchi, Jeffrey N. Siegel, Rachel E. Sobel, Daniel Solomon, Taunton R. Southwood, Richard Vesely, Patience H. White, Nico M. Wulffraat, Christy I. Sandborg

Research output: Contribution to journalArticle

12 Citations (Scopus)

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 longterm 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. Pediatrics 2013;132:e1384-e1394.

Original languageEnglish (US)
JournalPediatrics
Volume132
Issue number5
DOIs
StatePublished - Nov 1 2013

Fingerprint

Rheumatic Diseases
Registries
Safety
Rheumatology
Pediatrics
Arthritis
Marketing
Postmarketing Product Surveillance
Research
Patient Advocacy
Juvenile Arthritis
United States Food and Drug Administration
Biological Products
Systemic Lupus Erythematosus
Industry
Infection
Neoplasms
Childhood
Alliances

Keywords

  • Biologic products
  • Immunomodulatory therapy
  • Juvenile rheumatic disease
  • Medication safety

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Arts and Humanities (miscellaneous)
  • Medicine(all)

Cite this

Lionetti, G., Kimura, Y., Schanberg, L. E., Beukelman, T., Wallace, C. A., Ilowite, N. T., ... Sandborg, C. I. (2013). Using registries to identify adverse events in rheumatic diseases. Pediatrics, 132(5). https://doi.org/10.1542/peds.2013-0755

Using registries to identify adverse events in rheumatic diseases. / Lionetti, Geraldina; Kimura, Yukiko; Schanberg, Laura E.; Beukelman, Timothy; Wallace, Carol A.; Ilowite, Norman Todd; Winsor, Jane; Fox, Kathleen; Natter, Marc; Sundy, John S.; Brodsky, Eric; Curtis, Jeffrey R.; Gaizo, Vincent Del; Iyasu, Solomon; Jahreis, Angelika; Meeker-O'connell, Ann; Mittleman, Barbara B.; Murphy, Bernard M.; Peterson, Eric D.; Raymond, Sandra C.; Setoguchi, Soko; Siegel, Jeffrey N.; Sobel, Rachel E.; Solomon, Daniel; Southwood, Taunton R.; Vesely, Richard; White, Patience H.; Wulffraat, Nico M.; Sandborg, Christy I.

In: Pediatrics, Vol. 132, No. 5, 01.11.2013.

Research output: Contribution to journalArticle

Lionetti, G, Kimura, Y, Schanberg, LE, Beukelman, T, Wallace, CA, Ilowite, NT, Winsor, J, Fox, K, Natter, M, Sundy, JS, Brodsky, E, Curtis, JR, Gaizo, VD, Iyasu, S, Jahreis, A, Meeker-O'connell, A, Mittleman, BB, Murphy, BM, Peterson, ED, Raymond, SC, Setoguchi, S, Siegel, JN, Sobel, RE, Solomon, D, Southwood, TR, Vesely, R, White, PH, Wulffraat, NM & Sandborg, CI 2013, 'Using registries to identify adverse events in rheumatic diseases', Pediatrics, vol. 132, no. 5. https://doi.org/10.1542/peds.2013-0755
Lionetti G, Kimura Y, Schanberg LE, Beukelman T, Wallace CA, Ilowite NT et al. Using registries to identify adverse events in rheumatic diseases. Pediatrics. 2013 Nov 1;132(5). https://doi.org/10.1542/peds.2013-0755
Lionetti, Geraldina ; Kimura, Yukiko ; Schanberg, Laura E. ; Beukelman, Timothy ; Wallace, Carol A. ; Ilowite, Norman Todd ; Winsor, Jane ; Fox, Kathleen ; Natter, Marc ; Sundy, John S. ; Brodsky, Eric ; Curtis, Jeffrey R. ; Gaizo, Vincent Del ; Iyasu, Solomon ; Jahreis, Angelika ; Meeker-O'connell, Ann ; Mittleman, Barbara B. ; Murphy, Bernard M. ; Peterson, Eric D. ; Raymond, Sandra C. ; Setoguchi, Soko ; Siegel, Jeffrey N. ; Sobel, Rachel E. ; Solomon, Daniel ; Southwood, Taunton R. ; Vesely, Richard ; White, Patience H. ; Wulffraat, Nico M. ; Sandborg, Christy I. / Using registries to identify adverse events in rheumatic diseases. In: Pediatrics. 2013 ; Vol. 132, No. 5.
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