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Peer-reviewed publication of clinical trials completed for pediatric exclusivity.

Publication ,  Journal Article
Benjamin, DK; Smith, PB; Murphy, MD; Roberts, R; Mathis, L; Avant, D; Califf, RM; Li, JS
Published in: JAMA
September 13, 2006

CONTEXT: Much of pediatric drug use is off-label because appropriate pediatric studies have not been conducted and the drugs have not been labeled by the US Food and Drug Administration (FDA) for use in children. In 1997, Congress authorized the FDA to grant extensions of marketing rights known as "pediatric exclusivity" if FDA-requested pediatric trials were conducted. As a result, there have been over 100 product labeling changes. The publication status of studies completed for pediatric exclusivity has not been evaluated. OBJECTIVE: To quantify the dissemination of results of studies conducted for pediatric exclusivity into the peer-review literature. DESIGN: Cohort study of all trials conducted for pediatric exclusivity between 1998 and 2004 as determined by MEDLINE and EMBASE searches through 2005, the subsequent labeling changes, and the publication of those studies in peer-reviewed journals. We categorized any labeling changes resulting from the studies as positive or negative for the drug under study. We then evaluated aspects of the studies and product label changes that were associated with subsequent publication in peer-reviewed medical journals. MAIN OUTCOME MEASURES: Publication of the trial data in peer-reviewed journals. RESULTS: Between 1998 and 2004, 253 studies were submitted to the FDA for pediatric exclusivity: 125 (50%) evaluated efficacy, 51 (20%) were multi-dose pharmacokinetic, 34 (13%) were single-dose pharmacokinetic, and 43 (17%) were safety studies. Labeling changes were positive for 127/253 (50%) of studies; only 113/253 (45%) were published. Efficacy studies and those with a positive labeling change were more likely to be published. CONCLUSIONS: The pediatric exclusivity program has been successful in encouraging drug studies in children. However, the dissemination of these results in the peer-reviewed literature is limited. Mechanisms to more widely disperse this information through publication warrant further evaluation.

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Published In

JAMA

DOI

EISSN

1538-3598

Publication Date

September 13, 2006

Volume

296

Issue

10

Start / End Page

1266 / 1273

Location

United States

Related Subject Headings

  • United States Food and Drug Administration
  • United States
  • Pharmaceutical Preparations
  • Peer Review, Research
  • Pediatrics
  • General & Internal Medicine
  • Drug Labeling
  • Clinical Trials as Topic
  • 42 Health sciences
  • 32 Biomedical and clinical sciences
 

Citation

APA
Chicago
ICMJE
MLA
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Benjamin, D. K., Smith, P. B., Murphy, M. D., Roberts, R., Mathis, L., Avant, D., … Li, J. S. (2006). Peer-reviewed publication of clinical trials completed for pediatric exclusivity. JAMA, 296(10), 1266–1273. https://doi.org/10.1001/jama.296.10.1266
Benjamin, Daniel K., Philip Brian Smith, M Dianne Murphy, Rosemary Roberts, Lisa Mathis, Debbie Avant, Robert M. Califf, and Jennifer S. Li. “Peer-reviewed publication of clinical trials completed for pediatric exclusivity.JAMA 296, no. 10 (September 13, 2006): 1266–73. https://doi.org/10.1001/jama.296.10.1266.
Benjamin DK, Smith PB, Murphy MD, Roberts R, Mathis L, Avant D, et al. Peer-reviewed publication of clinical trials completed for pediatric exclusivity. JAMA. 2006 Sep 13;296(10):1266–73.
Benjamin, Daniel K., et al. “Peer-reviewed publication of clinical trials completed for pediatric exclusivity.JAMA, vol. 296, no. 10, Sept. 2006, pp. 1266–73. Pubmed, doi:10.1001/jama.296.10.1266.
Benjamin DK, Smith PB, Murphy MD, Roberts R, Mathis L, Avant D, Califf RM, Li JS. Peer-reviewed publication of clinical trials completed for pediatric exclusivity. JAMA. 2006 Sep 13;296(10):1266–1273.
Journal cover image

Published In

JAMA

DOI

EISSN

1538-3598

Publication Date

September 13, 2006

Volume

296

Issue

10

Start / End Page

1266 / 1273

Location

United States

Related Subject Headings

  • United States Food and Drug Administration
  • United States
  • Pharmaceutical Preparations
  • Peer Review, Research
  • Pediatrics
  • General & Internal Medicine
  • Drug Labeling
  • Clinical Trials as Topic
  • 42 Health sciences
  • 32 Biomedical and clinical sciences