IOM Review of FDA-Approved Biologics Labeled or Studied for Pediatric Use
Institute of Medicine, Washington, District of Columbia PEDIATRICS
(Impact Factor: 5.47).
01/2013; 131(2). DOI: 10.1542/peds.2012-2412
Studies have examined the extent to which public policies such as the Best Pharmaceuticals for Children Act have increased pediatric information in drug labeling. Little attention has focused on pediatric labeling of biologics. This analysis examines the extent to which biologics are labeled for pediatric use or have been studied in children.
The analysis covers the 96 biologics (excluding vaccines) that were first licensed by the Food and Drug Administration between 1997 and 2010 and were still marketed as of 2010. Product labeling was consulted for information on approved pediatric uses, pediatric studies, or pediatric safety warnings based on analyses of adverse events. The online database ClinicalTrials.gov was searched for registered pediatric studies of these biologics. A separate analysis examined labeling and studies for 55 vaccines.
For ∼60% of the 96 biologics, labeling shows approved pediatric use or pediatric study information or both. Approximately 85% of the biologics have ≥1 registered pediatric trial completed, underway, or planned. Overall, ∼90% are labeled for pediatric use, have pediatric information in the label, have a registered pediatric study, or have some combination of these characteristics. For the 55 analyzed vaccines, the corresponding figure is 95%.
A majority of biologics approved in the past 15 years include some pediatric information in their labeling, and pediatric trials have been registered for a substantial majority of these products.
Available from: Soko Setoguchi
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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.
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Despite considerable disincentives for conducting drug studies in children, 15 years ago the Food and Drug Administration, pediatric health advocates and congressional sponsors created a carrot-and-stick policy approach of voluntary and mandatory programs to encourage the pharmaceutical industry to include children in the drug development process. After several rounds of reauthorization of the laws on a temporary basis, the enabling statutes have been made permanent.
The purpose of this analysis is to review the advances that resulted from the law and the areas where further progress is needed.
A brief review of the history and results of the pediatric studies initiative was conducted by the authors and a determination made about the accomplishments of the law and remaining challenges.
Indicators of the changes that resulted from this pediatric studies initiative are both indirect, such as the increase in the number of indication supplements for new populations, and direct, such as the decrease in the percentage of medicines used off-label in children. Although the pediatric studies initiative has significantly improved therapeutic options for children, concern still exists that drug companies are reluctant to include children in drug development unless continuously incentivized, whether positively or negatively. Two challenges are particularly problematic: neonatal studies and child-friendly formulations.
Although the latest round of legislation should provide opportunities to address these problems, significantly more effort will be needed to achieve real culture change. Ultimately, the solution will require full program implementation by the Food and Drug Administration and close collaboration by many key stakeholders to ensure that pediatric studies become a routine part of the drug development process.
Available from: Han C G Kemper
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ABSTRACT: In children, levels of play, physical activity, and fitness are key indicators of health and disease and closely tied to optimal growth and development. Cardiopulmonary exercise testing (CPET) provides clinicians with biomarkers of disease and effectiveness of therapy, and researchers with novel insights into fundamental biological mechanisms reflecting an integrated physiological response that is hidden when the child is at rest. Yet the growth of clinical trials utilizing CPET in pediatrics remains stunted despite the current emphasis on preventative medicine and the growing recognition that therapies used in children should be clinically tested in children. There exists a translational gap between basic discovery and clinical application in this essential component of child health. To address this gap, the NIH provided funding through the Clinical and Translational Science Award (CTSA) program to convene a panel of experts. This report summarizes our major findings and outlines next steps necessary to enhance child health exercise medicine translational research. We present specific plans to bolster data interoperability, improve child health CPET reference values, stimulate formal training in exercise medicine for child health care professionals, and outline innovative approaches through which exercise medicine can become more accessible and advance therapeutics across the broad spectrum of child health. Clin Trans Sci 2014; Volume #: 1–10
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