title: Integration of pharmacogenetics and pharmacogenomics in drug development: implications for regulatory and medical decision making in pediatric diseases.
abstract: This article aims to provide an overview of the current situation regarding pharmacogenetic and pharmacogenomic (PG) studies in pediatrics, with a special focus on the role of PG data in the regulatory decision-making process. Despite the gap in pharmacogenetic research due to the lack of translational studies in adults and children, several technologies exist in drug development and biomarkers validation, which could supply valuable information concerning labeling and dosing recommendations. If performed under strict good clinical practice quality criteria, such findings could be included in the submission package of new chemical entities and used as additional information for prescribers, supporting further evaluation and understanding of the efficacy and safety profile of new medicines. Even though regulatory authorities may be aware of the potential role of PG in medical practice and guidances are available about the integration of PG in drug development, most data obtained from PG studies are not used by prescribers. The challenge is to better understand whether PG markers can be used to assess potential differences in drug response during the clinical program, so PG data can be integrated into the regulatory decision-making process, enabling the introduction of labeling information that promotes optimal dosing in the pediatric population.
authors.affiliation: Division of Pharmacology, Leiden/Amsterdam Center for Drug Research, Leiden, the Netherlands.
authors.forename: Chiara
authors.initials: C
authors.lastname: Piana
authors.forename: Linda
authors.initials: L
authors.lastname: Surh
authors.forename: Sabine
authors.initials: S
authors.lastname: Furst-Recktenwald
authors.forename: Achille
authors.initials: A
authors.lastname: Iolascon
authors.forename: Evelyne M
authors.initials: EM
authors.lastname: Jacqz-Aigrain
authors.forename: Ineke
authors.initials: I
authors.lastname: Jonker
authors.forename: Roberta
authors.initials: R
authors.lastname: Russo
authors.forename: Ron H N
authors.initials: RH
authors.lastname: van Schaik
authors.forename: Judith
authors.initials: J
authors.lastname: Wessels
authors.forename: Oscar E
authors.initials: OE
authors.lastname: Della Pasqua
mesh_terms: D000328:Adult; D000367:Age Factors; D002648:Child; D017277:Drug Approval / Q000331:legislation & jurisprudence; D055808:Drug Discovery / Q000331:legislation & jurisprudence / Q000379:methods*; D054796:Drug Dosage Calculations; D004348:Drug Labeling; D005819:Genetic Markers; D006801:Humans; D061214:Patient Safety; D018579:Patient Selection; D010597:Pharmacogenetics / Q000331:legislation & jurisprudence; D017410:Practice Guidelines as Topic; D057285:Precision Medicine; D018570:Risk Assessment; D012307:Risk Factors
chemical_list: D005819:Genetic Markers