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Paediatric cardiovascular clinical trials: an analysis of and the Food and Drug Administration Pediatric Drug Labeling Database*

  • Kevin D. Hill (a1) (a2), Heather T. Henderson (a1), Christoph P. Hornik (a1) (a2) and Jennifer S. Li (a1) (a2)


Recent regulatory initiatives in the United States of America and Europe have transformed the paediatric clinical trials landscape by significantly increasing capital investment and paediatric trial volume. The purpose of this manuscript was to review the impact of these initiatives on the paediatric cardiovascular trials landscape when compared with other paediatric sub-specialties. We also evaluate factors that may have contributed to the success or failure of recent major paediatric cardiovascular trials so as to inform the optimal design and conduct of future trials in the field.


Corresponding author

Correspondence to: K. D. Hill, MD MSCI, DUMC, Department of Pediatrics, Division of Pediatric Cardiology, Duke University Medical Center and the Duke Clinical Research Institute, Durham, NC 27105, United States of America. Tel: +919 668 8305; Fax: +919 681 8927; E-mail:


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Presented at Johns Hopkins All Children’s Heart Institute, International Pediatric Heart Failure Summit, Saint Petersburg, Florida, United States of America, 4–5 February, 2015.



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