TY - JOUR
T1 - Innovative clinical trial design for pediatric therapeutics
AU - Laughon, Matthew M.
AU - Benjamin, Daniel K.
AU - Capparelli, Edmund V.
AU - Kearns, Gregory L.
AU - Berezny, Katherine
AU - Paul, Ian
AU - Wade, Kelly
AU - Barrett, Jeff
AU - Smith, Phillip Brian
AU - Cohen-Wolkowiez, Michael
N1 - Funding Information:
Matthew M Laughon receives support from the US Government for his work in pediatric and neonatal clinical pharmacology (Government Contract HHSN267200700051C, principal investigator: Benjamin) and from the NICHD (1K23HL092225-01). Daniel K Benjamin Jr receives support from the US Government for his work in pediatric and neonatal clinical pharmacology (1R01HD057956-02, 1R01FD003519-01, 1U10-HD45962-06, 1K24HD058735-01, and Government Contract HHSN267200700051C), the nonprofit Thrasher Research Foundation for his work in neonatal candidiasis (www.thrasherresearch.org) and from industry for neonatal and pediatric drug development. Phillip Brian Smith receives support from the US Government for his work in pediatric and neonatal clinical pharmacology (Government Contract HHSN267200700051C, principal investigator: Benjamin), from the NICHD (1K23HD060040-01) and from AHRQ (1R18AE000028-01). Michael Cohen-Wolkowiez receives support from the NICHD (1K23HD064814-01) and the nonprofit Thrasher Research Foundation for his work in neonatal pharmacology. This study was supported by contract #HHSN275000002I-00001 under the Best Pharmaceuticals for Children Act. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.
PY - 2011/9
Y1 - 2011/9
N2 - Until approximately 15 years ago, sponsors rarely included children in the development of therapeutics. US and European legislation has resulted in an increase in the number of pediatric trials and specific label changes and dosing recommendations, although infants remain an understudied group. The lack of clinical trials in children is partly due to specific challenges in conducting trials in this patient population. Therapeutics in special populations, including premature infants, obese children and children receiving extracorporeal life support, are even less studied. National research networks in Europe and the USA are beginning to address some of the gaps in pediatric therapeutics using novel clinical trial designs. Recent innovations in pediatric clinical trial design, including sparse and scavenged sampling, population pharmacokinetic analyses and 'opportunistic' studies, have addressed some of the historical challenges associated with clinical trials in children.
AB - Until approximately 15 years ago, sponsors rarely included children in the development of therapeutics. US and European legislation has resulted in an increase in the number of pediatric trials and specific label changes and dosing recommendations, although infants remain an understudied group. The lack of clinical trials in children is partly due to specific challenges in conducting trials in this patient population. Therapeutics in special populations, including premature infants, obese children and children receiving extracorporeal life support, are even less studied. National research networks in Europe and the USA are beginning to address some of the gaps in pediatric therapeutics using novel clinical trial designs. Recent innovations in pediatric clinical trial design, including sparse and scavenged sampling, population pharmacokinetic analyses and 'opportunistic' studies, have addressed some of the historical challenges associated with clinical trials in children.
UR - http://www.scopus.com/inward/record.url?scp=80052765511&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=80052765511&partnerID=8YFLogxK
U2 - 10.1586/ecp.11.43
DO - 10.1586/ecp.11.43
M3 - Review article
C2 - 21980319
AN - SCOPUS:80052765511
SN - 1751-2433
VL - 4
SP - 643
EP - 652
JO - Expert Review of Clinical Pharmacology
JF - Expert Review of Clinical Pharmacology
IS - 5
ER -