Challenges of biomarkers in drug discovery and development

Expert Opinion on Drug Discovery

Volumn 7, Issue 6, 2012, Pages 457-461

  Goodsaid, Federico ^a  

^a VERTEX PHARMACEUTICALS   (United States)

Author keywords

Biomarkers; Clinical studies; Qualification

Indexed keywords

B RAF KINASE; BRENTUXIMAB VEDOTIN; BUSULFAN; CAPECITABINE; CISPLATIN; CRIZOTINIB; CYTOCHROME P450 2D6; DYSTROPHIN; EPIDERMAL GROWTH FACTOR RECEPTOR; EPIDERMAL GROWTH FACTOR RECEPTOR 2; ERLOTINIB; FULVESTRANT; GEFITINIB; GLUCOSYLCERAMIDASE; GLUCURONOSYLTRANSFERASE 1A1; IMATINIB; K RAS PROTEIN; LAPATINIB; LETROZOLE; MERCAPTOPURINE; NILOTINIB; PANITUMUMAB; PHARMACOLOGICAL BIOMARKER; RASBURICASE; TAMOXIFEN; TIOGUANINE; TOSITUMOMAB; TRASTUZUMAB; UNINDEXED DRUG; VEMURAFENIB;

ALZHEIMER DISEASE; CLINICAL TRIAL (TOPIC); COGNITIVE DEFECT; DRUG LABELING; DRUG RESEARCH; DRUG SYNTHESIS; DUCHENNE MUSCULAR DYSTROPHY; ENZYME REPLACEMENT; GAUCHER DISEASE; GENE MUTATION; HUMAN; NEPHROTOXICITY; PATIENT SELECTION; PHENOTYPE; PRIORITY JOURNAL; PRODUCT DEVELOPMENT; PROTEIN EXPRESSION; PROTEIN TARGETING; REVIEW;

ANIMALS; BIOLOGICAL MARKERS; DRUG APPROVAL; DRUG DISCOVERY; DRUG INDUSTRY; HUMANS;

EID: 84861500754     PISSN: 17460441     EISSN: 1746045X     Source Type: Journal    
DOI: 10.1517/17460441.2012.679615     Document Type: Review

References (18)

1. Hodgson DR, Whittaker RD, Herath A, et al. Biomarkers in oncology drug development. Mol Oncol 2009;3:24-32
2. FDA Table of Pharmacogenomic Biomarkers in Drug Labels. Available from: http://www.fda.gov/Drugs/ ScienceResearch/ResearchAreas/ Pharmacogenetics/ ucm083378.htm [Last accessed 17 March 2012]
3. Hanby AM. Aspects of molecular phenotype and its correlations with breast cancer behaviour and taxonomy. Br J Cancer 2005;92:613-17
4. Guptaa N, Oppenheima IM, Kauvara EF, et al. Type 2 Gaucher disease: phenotypic variation and genotypic heterogeneity. Blood Cells Mol Dis 2011;46:75-84
5. Beutler E, Nguyen NJ, Henneberger MW, et al. Gaucher disease: gene frequencies in the Ashkenazi Jewish population. Am J Hum Genet 1993;52:85-8
6. Beutler E. Enzyme replacement in Gaucher Disease. PLoS Med 2004;1:118-21
7. Gaucher's Disease enzyme replacement therapy FDA sample label. Available from: http://www.accessdata.fda.gov/ drugsatfda-docs/label/2010/022575lbl. pdf [Last accessed 17 March 2012]
8. Foster K, Foster H, Dickson JG. Gene therapy progress and prospects: duchenne muscular dystrophy. Gene Ther 2006;13:1677-85
9. Aartsma-Rus A, den Dunnen JT, van Ommen GJ. New insights in gene-derived therapy: the example of Duchenne muscular dystrophy. Ann NY Acad Sci 2010;1214:199-212 . Background information for exon-skipping therapies.
10. Aartsma-Rus A, van Deutekom JC, Fokkema IF, et al. Entries in the Leiden Duchenne muscular dystrophy mutation database: an overview of mutation types and paradoxical cases that confirm the reading-frame rule. Muscle Nerve 2006;34:135-44
11. Goodsaid F, Papaluca M. Evolution of biomarker qualification at the health authorities. Nat Biotechnol 2010;28:441-3
12. Consensus Report. Evaluation of Biomarkers and Surrogate Endpoints in Chronic Disease. Institute of Medicine of the National Academy of Sciences (US); Washington, DC: 2010
13. FDA Biomarker Qualification Process. Available from: http://www.fda.gov/ Drugs/DevelopmentApprovalProcess/ DrugDevelopmentToolsQualification Program/ucm284621.htm [Last accessed 17 March 2012]
14. EMA Biomarker Qualification Process. Available from: http://www.ema. europa. eu/docs/en-GB/document-library/ Regulatory-and-procedural-guideline/ 2009/10/WC500004201.pdf [Last accessed 17 March 2012]
15. Dieterle F, Sistare F, Goodsaid F, et al. Renal biomarker qualification submission: a dialog between the FDA-EMEA and predictive safety testing consortium. Nat Biotechnol 2010;28:455-62
16. EMA Qualification for Alzheimer Biomarkers. Available from: http://www. ema.europa.eu/docs/en-GB/ document-library/Regulatory-and- procedural-guideline/ 2011/05/ WC500106357.pdf [Last accessed 17 March 2012]
17. Review Submission of the Qualification of Seven Biomarkers of Drug-Induced Nephrotoxicity in rats. Available from: http://www.fda.gov/ downloads/Drugs/ DevelopmentApprovalProcess/ DrugDevelopmentToolsQualification Program/UCM285031.pdf [Last accessed 17 March 2012]
18. Biomarkers Related to Drug or Biotechnology Product Development: Context, Structure and Format of Qualification Submissions. Available from: http://www.ich.org/fileadmin/ Public-Web-Site/ICH-Products/ Guidelines/Efficacy/ E16/Step4/ E16-Step-4.pdf [Last accessed 17 March 2012]