Peer-Reviewed Publication of Clinical Trials Completed for Pediatric Exclusivity

Benjamin, Daniel K.; Smith, P. Brian; Murphy, M. Dianne; Roberts, Rosemary; Mathis, Lisa L.; Avant, Debbie; Califf, Robert M.; Li, Jennifer S.

doi:10.1001/jama.296.10.1266

Cited by 140 publications

(125 citation statements)

References 30 publications

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“…[33] Asthma and vaccines contributed 29% of the trials in our study. Studies of asthma were done chiefly in children and studies of vaccines in infants.…”

Section: /25mentioning

confidence: 78%

“…Nevertheless, despite a few initiatives to promote research in neonates, [32] most paediatric studies are done in children and adolescents, as shown by a study of 253 trials conducted to obtain paediatric exclusivity extensions. [33] Drugs studied in these trials are mainly used in older children and adolescents for conditions such as hypertension, systemic autoimmune disorders, and gastrointestinal disorders. [33] We identified important deficiencies in the reporting of age characteristics of populations included in PRCTs.…”

Section: /25mentioning

confidence: 99%

“…[33] Drugs studied in these trials are mainly used in older children and adolescents for conditions such as hypertension, systemic autoimmune disorders, and gastrointestinal disorders. [33] We identified important deficiencies in the reporting of age characteristics of populations included in PRCTs. In 17% of the PRCTs published in 2006, at least one major age characteristic was missing.…”

Section: /25mentioning

confidence: 99%

“…Low publication rates of PRCTs done to obtain exclusivity extensions have been found. [33] PRCTs of interventions whose use is well established in adults may be unlikely to be accepted for publication by peer-reviewed journals. Publication bias may occur when the studies are conducted in narrow fields or with small effect sizes or when they fail to show efficacy of the study intervention.…”

Section: /25mentioning

confidence: 99%

“…However, only data published in peer-reviewed journals are likely to have a major impact on clinical practice. [11,33] Labelling issues constitute only the most visible part of the far-reaching deficiencies that characterize paediatric clinical research. Because the rules applying to drug use are more restrictive than in other fields (e.g., there is no equivalent of labelling in surgery), the imbalance in labelling between adults and children and across paediatric age groups is more easily recognizable, compared to other problems affecting paediatric research.…”

Section: /25mentioning

confidence: 99%

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Fewer infants than older patients in paediatric randomised controlled trials

et al. 2010

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Financial disclosure or conflict of interest for all authors: NoneWord count: 3428 2/25 ABSTRACT To determine whether the youngest age groups are less likely to be included in paediatric randomised controlled trials (PRCTs) than older children, we conducted a PubMed search using the keyword "randomised controlled trial" and the limit "all child: 0 -18 years". We retrieved 417 articles published in 2006 in 34 leading journals classified as general medical journals, paediatric medical journals, or specialist medical journals. We arbitrarily selected 144 articles, at random. For each study, we evaluated population age characteristics (central tendency, range, and dispersion), study design, sample size and topic. Of the 144 studies, only 82 were first reports of paediatric randomised controlled trials (PRCTs). Among the other studies, many were done in adults. Of the 82 PRCTs, only 11% included newborns and 26% infants; 59% included children and 39% adolescents. Using the same search strategy to retrieve PRCTs in the same journals in the last four months of 2009 retrieved 66 PRCTs, of which 17% included newborns, 24% infants, 61% children and 55% adolescents. The three health conditions most often reported were respiratory diseases, infectious diseases, and mental and behavioural disorders. In 34 leading journals, PRCTs were significantly less likely to include newborns and infants than older paediatric patients. Given the huge impact of PRCTs on paediatric health, additional efforts are needed to promote studies in newborns and infants, as well as studies of the impact of recent European and American regulations designed to encourage paediatric drug trials.

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“…[33] Asthma and vaccines contributed 29% of the trials in our study. Studies of asthma were done chiefly in children and studies of vaccines in infants.…”

Section: /25mentioning

confidence: 78%