Integration of Pharmacogenetics and Pharmacogenomics in Drug Development: Implications for Regulatory and Medical Decision Making in Pediatric Diseases

被引:13
作者
Piana, Chiara [1 ]
Surh, Linda
Furst-Recktenwald, Sabine [2 ]
Iolascon, Achille [3 ,4 ]
Jacqz-Aigrain, Evelyne M. [5 ]
Jonker, In Eke [6 ]
Russo, Roberta [7 ]
van Schalk, Ron H. N. [8 ]
Wessels, Judith [9 ]
Della Pasqua, Oscar E. [1 ]
机构
[1] Leiden Univ, Leiden Amsterdom Ctr Drug Res, Div Pharmacol, NL-2300 RA Leiden, Netherlands
[2] F Hoffmann La Roche Ltd, Translat Med, Basel, Switzerland
[3] Univ Naples Federico II, Dept Biochem & Med Biotechnol, Naples, Italy
[4] CEINGE Adv Biotechnol, Dept Biochem & Med Biotechnol, Naples, Italy
[5] Hop Robert Debre, Dept Paediat Pharmacol & Pharmacogenet, INSERM, CIC 9202, Paris, France
[6] Xendo Pharma Serv B V Div Regulatory Affairs, Leiden, Netherlands
[7] CEINGE Adv Biotechnol, Naples, Italy
[8] Erasmus MC, Dept Clin Chem, Rotterdam, Netherlands
[9] LUMC, Dept Clin Pharm & Toxicol, Leiden, Netherlands
关键词
Pharmacogenetics; regulatory/scientific affairs; clinical pharmacology; pediatric drug development; medical practice; STEVENS-JOHNSON SYNDROME; HLA-B-ASTERISK-1502; ALLELE; POLYMORPHISMS; ASSOCIATION; CHILDREN; RISK; PHARMACOLOGY; BIOMARKERS; THERAPY; PHARMACOKINETICS;
D O I
10.1177/0091270011401619
中图分类号
R9 [药学];
学科分类号
1007 ;
摘要
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.
引用
收藏
页码:704 / 716
页数:13
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