title: Association between pediatric clinical trials and global burden of disease.
abstract: BACKGROUND
  The allocation of research resources should favor conditions responsible for the greatest disease burden. This is particularly important in pediatric populations, which have been underrepresented in clinical research. Our aim was to measure the association between the focus of pediatric clinical trials and burden of disease and to identify neglected clinical domains.
  
  
  METHODS
  We performed a cross-sectional study of clinical trials by using trial records in ClinicalTrials.gov. All trials started in 2006 or after and studying patient-level interventions in pediatric populations were included. Age-specific measures of disease burden were obtained for 21 separate conditions for high-, middle-, and low-income countries. We measured the correlation between number of pediatric clinical trials and disease burden for each condition.
  
  
  RESULTS
  Neuropsychiatric conditions and infectious diseases were the most studied conditions globally in terms of number of trials (874 and 847 trials, respectively), while intentional injuries (5 trials) and maternal conditions (4 trials) were the least studied. Clinical trials were only moderately correlated with global disease burden (r = 0.58, P = .006). Correlations were also moderate within each of the country income levels, but lowest in low-income countries (r = .47, P = .03). Globally, the conditions most understudied relative to disease burden were injuries (-260 trials for unintentional injuries and -160 trials for intentional injuries), nutritional deficiencies (-175 trials), and respiratory infections (-171 trials).
  
  
  CONCLUSIONS
  Pediatric clinical trial activity is only moderately associated with pediatric burden of disease, and least associated in low-income countries. The mismatch between clinical trials and disease burden identifies key clinical areas for focus and investment.
authors.affiliation: Division of Emergency Medicine, Boston Children's Hospital, 300 Longwood Ave, Boston, MA 02115. florence.bourgeois@childrens.harvard.edu.
authors.forename: Florence T
authors.initials: FT
authors.lastname: Bourgeois
authors.forename: Karen L
authors.initials: KL
authors.lastname: Olson
authors.forename: John P A
authors.initials: JP
authors.lastname: Ioannidis
authors.forename: Kenneth D
authors.initials: KD
authors.lastname: Mandl
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D002986:Clinical Trials as Topic / Q000706:statistics & numerical data*; D017281:Cost of Illness; D003430:Cross-Sectional Studies; D019049:Developed Countries / Q000706:statistics & numerical data*; D003906:Developing Countries / Q000706:statistics & numerical data*; D014943:Global Health; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D015993:Life Tables; D010372:Pediatrics / Q000706:statistics & numerical data*; D012042:Registries
keywords: burden of disease; clinical trials; pediatric research