Voluntary exploratory data submissions to the US FDA and the EMA: experience and impact

被引：62

作者：

Goodsaid, Federico M. ^{[1
]}

Amur, Shashi ^{[1
]}

Aubrecht, Jiri

Burczynski, Michael E. ^{[3
]}

Carl, Kevin ^{[5
]}

Catalano, Jennifer ^{[4
]}

Charlab, Rosane ^{[1
]}

Close, Sandra ^{[6
]}

Cornu-Artis, Catherine ^{[7
]}

Essioux, Laurent ^{[8
]}

Fornace, Albert J., Jr. ^{[9
,10
]}

Hinman, Lois ^{[5
]}

Hong, Huixiao ^{[11
]}

Hunt, Ian

Jacobson-Kram, David

Jawaid, Ansar

Laurie, David ^{[7
]}

Lesko, Lawrence

Li, Heng-Hong ^{[9
,10
]}

Lindpaintner, Klaus ^{[13
]}

Mayne, James ^{[2
]}

Morrow, Peter ^{[6
]}

Papaluca-Amati, Marisa ^{[14
]}

Robison, Timothy W. ^{[12
]}

Roth, John ^{[3
]}

Schuppe-Koistinen, Ina

Shi, Leming ^{[11
]}

Spleiss, Olivia ^{[8
]}

Tong, Weida ^{[11
]}

Truter, Sharada L.

Vonderscher, Jacky ^{[3
]}

Westelinck, Agnes ^{[15
]}

Zhang, Li ^{[1
]}

Zineh, Issam ^{[1
]}

机构：

[1] US FDA, Genom Grp, Off Clin Pharmacol, Off Translat Sci,Ctr Drug Evaluat & Res, Silver Spring, MD 20903 USA

[2] Pfizer Global R&D, Worldwide Regulatory Strategy & Policy, Groton, CT 06340 USA

[3] F Hoffmann La Roche, Nutley, NJ 07110 USA

[4] US FDA, Div Cell & Gene Therapies, Off Cellular Tissue & Gene Therapies, Ctr Biol Evaluat & Res, Rockville, MD 20852 USA

[5] Novartis Pharmaceut, E Hanover, NJ 07936 USA

[6] Eli Lilly & Co, Lilly Corp Ctr, Indianapolis, IN 46285 USA

[7] Novartis Pharmaceut, CH-4133 Basel, Switzerland

[8] F Hoffmann La Roche, CH-4070 Basel, Switzerland

[9] Georgetown Univ, Dept Biochem & Mol & Cellular Biol, Washington, DC 20007 USA

[10] Georgetown Univ, Lombardi Comprehens Canc Ctr, Washington, DC 20007 USA

[11] US FDA, Natl Ctr Toxicol Res, Jefferson, AR 72079 USA

[12] US FDA, Div Pulm & Allergy Prod, Off New Drugs, Ctr Drug Evaluat & Res, Silver Spring, MD 20903 USA

[13] Strateg Diagnost Inc, St Petersburg 197022, Russia

[14] European Med Agcy, Preauthorisat Unit, Sector Safety & Efficacy Med, London E14 4HB, England

[15] GlaxoSmithKline, Collegeville, PA 19426 USA

来源：

NATURE REVIEWS DRUG DISCOVERY | 2010年 / 9卷 / 06期

关键词：

RENAL-CELL CARCINOMA; BLOOD MONONUCLEAR-CELLS; DIHYDROPYRIMIDINE DEHYDROGENASE; CLINICAL-OUTCOMES; PROTOCOL BIOPSIES; DRUG DEVELOPMENT; XIMELAGATRAN; GENOTOXICITY; TRANSPLANTATION; CLOPIDOGREL;

D O I：

10.1038/nrd3116

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

Heterogeneity in the underlying mechanisms of disease processes and inter-patient variability in drug responses are major challenges in drug development. To address these challenges, biomarker strategies based on a range of platforms, such as microarray gene-expression technologies, are increasingly being applied to elucidate these sources of variability and thereby potentially increase drug development success rates. With the aim of enhancing understanding of the regulatory significance of such biomarker data by regulators and sponsors, the US Food and Drug Administration initiated a programme in 2004 to allow sponsors to submit exploratory genomic data voluntarily, without immediate regulatory impact. In this article, a selection of case studies from the first 5 years of this programme - which is now known as the voluntary exploratory data submission programme, and also involves collaboration with the European Medicines Agency - are discussed, and general lessons are highlighted.

引用

页码：435 / U44

页数：16

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