Safe and Effective Medicines for Children Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act (2012) / Chapter Skim
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and to consider the incentives for pediatric studies of biologics. A committee appointed by the IOM reviewed and assessed a representative sample of labeling changes and other FDA actions related to requested or required studies for the period from July 1, 1998, through December 31, 2010.
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Many drugs commonly used with premature and sick neonates are older drugs that have not been adequately evaluated in studies with this vulnerable age group. • ongress has significantly expanded public access to infor C mation from recent pediatric studies conducted under BPCA and PREA and has thereby enhanced the value of these stud
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c I n the late 1990s, the federal government took steps to increase the study of drugs in children and thereby reduce a serious deficit in the data on drug safety and efficacy for young patients. One step was to offer economic incentives for the conduct of requested pediatric studies.
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Studies may also guide dosing adjustments that are often more complicated than simply scaling down doses recommended for adults on the basis of a child's age or weight. The shortage of pediatric drug studies that prompted passage of BPCA and PREA (and their predecessor policies)
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STUDY ORIGINS AND FOCUS Consistent with provisions of the 2007 law reauthorizing BPCA and PREA and with provisions of the Biologics Price Competition and Innovation Act (BPCIA) enacted in 2010, FDA asked the IOM to examine pediatric studies requested under BPCA or required under PREA.
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The committee also reviewed additional FDA actions involving written requests, studies with neonates, and, to the extent possible, required pediatric studies of biologics. FDA's list of labeling changes excludes labeling changes for biologics (including vaccines)
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• Congress has significantly expanded public access to information from recent pediatric studies conducted under BPCA and PREA and has thereby enhanced the value of these studies. Limitations still exist, however, particularly for older pediatric studies and la beling changes.
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Best Pharmaceuticals for Children Act Among other provisions, the Food and Drug Modernization and Accountability Act of 1997 offered companies pediatric exclusivity -- a period of marketing protection from competitor (generic) drugs -- when they undertook pediatric studies of a drug based on a written request from FDA.
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One concern for companies is variation between the United States and Europe in requirements for pediatric drug studies. Oversimplified, the European Medicines Agency requires submission of a pediatric study plan early during the clinical investigation of a drug in adults, whereas the United States requires the plan late in the drug approval process.
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ETHICAL ISSUES IN PEDIATRIC DRUG STUDIES One broad ethical principle for the conduct of pediatric drug studies is that children should not be subjected to research that is not necessary to advance knowledge that is relevant to child health. Another is that children should not participate in studies that are designed or conducted in ways that predictably undermine the potential of the research to generate valid and useful information.
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Certainly, it is appropriate that written requests be accompanied by clear expectations that the requested studies are necessary, soundly designed and executed, and public in their results. SAFETY AND EFFICACY IN STUDIES CONDUCTED UNDER BPCA AND PREA IOM was asked to assess the number and type of pediatric adverse events in a sample of studies conducted under PREA or precursor regulations.
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The 1-year reviews mandated by Congress provide useful opportunities for FDA to examine safety information after labeling changes based on pediatric studies have been made and, in some cases, to recommend further analyses or inclusion of additional safety findings in product labeling. Given the limitations of the short-term studies typically used to support labeling changes and the limitations of the 1-year reviews, FDA might consider more frequent use of its authority to require sponsors to undertake longterm postmarket, follow-up studies of serious or potentially serious risks to patient safety.
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FDA provided the committee with a list of products for which information from studies with neonates had resulted in labeling changes or awards of exclusivity without labeling changes. From 1998 through 2010, only 23 of the more than 350 labeling changes resulting from new pediatric studies included information from studies with neonates.
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To promote more studies of drugs widely used but not adequately evaluated in neonates, one option is for Congress to provide additional resources for short- and long-term neonatal drug studies through the BPCA program at NIH. OUTCOMES OF WRITTEN REQUESTS AND PREA REQUIREMENTS Overall, from July 1998 through October 2011, FDA approved more than 420 labeling changes associated with studies requested under BPCA or required under PREA (or their predecessor policies)
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Just as most studies requested under BPCA or required under PREA yielded useful information, most labeling changes reflected this result. However, labeling changes have sometimes excluded or downplayed important information, for example, information about certain adverse events.
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Barring surprises in their implementation, the incentives of BPCIA can be expected to encourage further pediatric studies of both older and newer biologics. Nonetheless, it seems unlikely that the law will lead to a surge of written requests for pediatric studies of biologics similar to the surge in requests for pediatric drug studies that followed the creation of the pediatric exclusivity incentive in 1997.
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The committee's finding that most biologics have been studied with children does not mean that no further opportunities or needs for pediatric studies of these medications exist. Such opportunities could include studies that pursue promising findings in early-phase studies of specific biologics or studies of biologics for treatment of conditions that are now recognized to occur more frequently in children than previously thought.
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