Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2011 Sep;4(5):643-52.
doi: 10.1586/ecp.11.43.

Innovative clinical trial design for pediatric therapeutics

Matthew M Laughon  1 , Daniel K Benjamin Jr, Edmund V Capparelli, Gregory L Kearns, Katherine Berezny, Ian M Paul, Kelly Wade, Jeff Barrett, Phillip Brian Smith, Michael Cohen-Wolkowiez
Affiliations
Review

Innovative clinical trial design for pediatric therapeutics

Matthew M Laughon et al. Expert Rev Clin Pharmacol. 2011 Sep.

Abstract

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.

Keywords: clinical trial simulation; opportunistic study; pediatrics; pharmacodynamics; pharmacokinetics; pharmacometrics; therapeutics.

PubMed Disclaimer

Similar articles

See all similar articles

Cited by

See all "Cited by" articles

References

    1. Shirkey HC. Therapeutic orphans – everybody’s business. Ann. Pharmacother. 2006;40(6):1174. - PubMed
    1. Choonara I. Unlicensed and off-label drug use in children: implications for safety. Expert Opin. Drug Saf. 2004;3(2):81–83. - PubMed
    1. Roberts R, Rodriguez W, Murphy D, et al. Pediatric drug labeling: improving the safety and efficacy of pediatric therapies. JAMA. 2003;290(7):905–911. - PubMed
    1. Turner S, Nunn AJ, Fielding K, et al. Adverse drug reactions to unlicensed and off-label drugs on paediatric wards: a prospective study. Acta Paediatr. 1999;88(9):965–968. - PubMed
    1. Avenel S, Bomkratz A, Dassieu G, et al. The incidence of prescriptions without marketing product license in a neonatal intensive care unit. Arch. Pediatr. 2000;7(2):143–147. - PubMed

Websites

    1. FDA: Guidance for industry – general considerations for pediatric pharmacokinetic studies for drugs and biological products. www.fda.gov/downloads/Drugs/uidanceComplianceRegulatoryInformation/Guida....
    1. ClinicalTrials.gov: Fluconazole Prophylaxis for the Prevention of Candidiasis in Infants Less Than 750 Grams Birthweight. http://clinicaltrials.gov/ct2/show/NCT00734539.
    1. Medicines for Children Research Network. www.mcrn.org.uk.
    1. EGAN: Global Research in Paediatrics. www.egan.eu/en/our-activities/projects/grip.
    1. NIH: Best pharmaceuticals for children act. http://bpca.nichd.nih.gov/clinical/network/index.cfm.

Publication types

MeSH terms

Substances