Regulatory perspective on remaining challenges for utilization of pharmacogenomics-guided drug developments

Pharmacogenomics

Volumn 14, Issue 2, 2013, Pages 195-203

  Otsubo, Yasuto ^a   Ishiguro, Akihiro ^a   Uyama, Yoshiaki ^a,b  

^a PHARMACEUTICALS AND MEDICAL DEVICES AGENCY   (Japan)

^b CHIBA UNIVERSITY   (Japan)

Author keywords

biomarker; drug development; international harmonization; personalized medicine; pharmacogenomics; regulatory science

Indexed keywords

BIOLOGICAL MARKER; CARBAMAZEPINE; CETUXIMAB; CRIZOTINIB; DRUG; FIRTECAN; GEFITINIB; HLA A ANTIGEN; HLA B ANTIGEN; LAPATINIB; MOGAMULIZUMAB; PANITUMUMAB;

ADVANCED CANCER; ALLELE; AREA UNDER THE CURVE; BREAST CANCER; CANCER RECURRENCE; CLINICAL TRIAL (TOPIC); COLORECTAL CANCER; COLORECTAL CARCINOMA; COMPANION DIAGNOSTIC; DIAGNOSTIC KIT; DIAGNOSTIC PROCEDURE; DRUG BLOOD LEVEL; DRUG CONTROL; DRUG EFFICACY; DRUG INDUSTRY; DRUG LABELING; DRUG RESEARCH; DRUG UTILIZATION; EDUCATION; ETHNICITY; FLUORESCENCE IN SITU HYBRIDIZATION; GENE; GENE MUTATION; GENETIC ANALYSIS; GENETIC ASSOCIATION; GOVERNMENT REGULATION; HEALTH CARE DELIVERY; HLAA 3101 GENE; HLAB 1502 GENE; HUMAN; IMMUNOHISTOCHEMISTRY; INTERNATIONAL COOPERATION; LUNG NON SMALL CELL CANCER; MASS COMMUNICATION; NEUTROPENIA; PHARMACOGENOMICS; POLYMERASE CHAIN REACTION; POPULATION RESEARCH; PRACTICE GUIDELINE; PUBLIC-PRIVATE PARTNERSHIP; RACE DIFFERENCE; RECURRENT CANCER; REVIEW; SAMPLING; STEVENS JOHNSON SYNDROME; T CELL LEUKEMIA;

BIOLOGICAL MARKERS; DRUG DISCOVERY; DRUG INDUSTRY; DRUG LABELING; GOVERNMENT REGULATION; GUIDELINES AS TOPIC; INTERNATIONAL COOPERATION; JAPAN; PHARMACOGENETICS;

EID: 84872516779     PISSN: 14622416     EISSN: 17448042     Source Type: Journal    
DOI: 10.2217/pgs.12.194     Document Type: Review

References (37)

1. Uyama Y, Ishiguro A, Nakamura H, Toyoshima S. Use of biomarkers in drug development: Japanese perspectives. In: Predictive Approaches in Drug Discovery and Development: Biomarkers and In Vitro/In Vivo Correlations. Williams JA, Koup J, Lalonde R, Christ DD (Eds). John Wiley & Sons Inc., NJ, USA, 269-287 (2012).
2. Frueh FW, Amur S, Mummaneni P et al. Pharmacogenomic biomarker information in drug labels approved by the United States food and drug administration: prevalence of related drug use. Pharmacotherapy 28(8), 992-998 (2008).
3. Poste G, Carbone DP, Parkinson DR, Verweij J, Hewitt SM, Jessup JM. Leveling the playing field: bringing development of biomarkers and molecular diagnostics up to the standards for drug development. Clin. Cancer Res. 18(6), 1515-1523 (2012).
4. La Thangue NB, Kerr DJ. Predictive biomarkers: a paradigm shift towards personalized cancer medicine. Nat. Rev. Clin. Oncol. 8(10), 587-596 (2011).
5. Wang S-J, O'Neill RT, Hung HJ. Statistical considerations in evaluating pharmacogenomics-based clinical effect for confirmatory trials. Clin. Trials 7(5), 525-536 (2010).
6. Warner AW, Bhathena A, Gilardi S et al. Challenges in obtaining adequate genetic sample sets in clinical trials: the perspective of the industry pharmacogenomics working group. Clin. Pharmacol. Ther. 89(4), 529-536 (2011).
7. Ricci DS, Broderick ED, Tchelet A et al. Global requirements for DNA sample collections: results of a survey of 204 ethics committees in 40 countries. Clin. Pharmacol. Ther. 89(4), 554-561 (2011).
8. Warner A, Nelsen A, Bhathena A et al. Enabling pharmacogenomic clinical trials through sampling. Pharmacogenomics 11(12), 1649-1654 (2010).
9. Goodsaid F. Challenges of biomarkers in drug discovery and development. Expert Opin. Drug Discov. 7(6), 457-461 (2012).
10. Dieterle F, Sistare F, Goodsaid F et al. Renal biomarker qualification submission: a dialog between the FDA-EMEA and predictive safety testing consortium. Nat. Biotech. 28(5), 455-462 (2010).
11. Thiers FA, Sinskey AJ, Berndt ER. Trends in the globalization of clinical trials. Nat. Rev. Drug Discov. 7(1), 13-14 (2008).
12. Isaac M, Vamvakas S, Abadie E, Jonsson B, Gispen C, Pani L. Qualification opinion of novel methodologies in the predementia stage of Alzheimer's disease: cerebro-spinal-fluid related biomarkers for drugs affecting amyloid burden: regulatory considerations by European Medicines Agency focusing in improving benefit/risk in regulatory trials. Eur. Neuropsychopharmacol. 21(11), 781-788 (2011).
13. Minami H, Sai K, Saeki M et al. Irinotecan pharmacokinetics/ pharmacodynamics and UGT1A genetic polymorphisms in Japanese: roles of UGT1A1 6 and 28. Pharmacogenet. Genomics 17, 497-504 (2007). (Pubitemid 46905216)
14. Kaniwa N, Kurose K, Jinno H et al. Racial variability in haplotype frequencies of UGT1A1 and glucuronidation activity of a novel single nucleotide polymorphism 686c> t (p229l) found in an African-American. Drug Metab. Dispos. 33(3), 458-465 (2005). (Pubitemid 40279948)
15. Paez JG, Jänne PA, Lee JC et al. EGFR mutations in lung cancer: correlation with clinical response to gefitinib therapy. Science 304(5676), 1497-1500 (2004). (Pubitemid 38720929)
16. Lynch TJ, Bell DW, Sordella R et al. Activating mutations in the epidermal growth factor receptor underlying responsiveness of non-small-cell lung cancer to gefitinib. N. Engl. J. Med. 350(21), 2129-2139 (2004). (Pubitemid 38637993)
17. Fukuoka M, Wu Y-L, Thongprasert S et al. Biomarker analyses and final overall survival results from a Phase III, randomized, open-label, first-line study of gefitinib versus carboplatin/paclitaxel in clinically selected patients with advanced non-small-cell lung cancer in Asia (IPASS). J. Clin. Oncol. 29(21), 2866-2874 (2011).
18. Maemondo M, Inoue A, Kobayashi K et al. Gefitinib or chemotherapy for non-small-cell lung cancer with mutated EGFR. N. Engl. J. Med. 362(25), 2380-2388 (2010).
19. Mitsudomi T, Morita S, Yatabe Y et al. Gefitinib versus cisplatin plus docetaxel in patients with non-small-cell lung cancer harbouring mutations of the epidermal growth factor receptor (WJTOG3405): an open label, randomised Phase III trial. Lancet Oncol. 11(2), 121-128 (2010).
20. Chung WH, Hung SI, Hong HS et al. Medical genetics: a marker for Stevens-Johnson syndrome. Nature 428(6982), 486 (2004).
21. Chen P, Lin JJ, Lu CS et al. Carbamazepine-induced toxic effects and HLA-B 1502 screening in Taiwan. N. Engl. J. Med. 364(12), 1126-1133 (2011).
22. Kaniwa N, Saito Y, Aihara M et al. HLA-B locus in Japanese patients with antiepileptics and allopurinol-related Stevens-Johnson syndrome and toxic epidermal necrolysis. Pharmacogenomics 9(11), 1617-1622 (2008).
23. Ozeki T, Mushiroda T, Yowang A et al. Genome-wide association study identifies HLA-A 3101 allele as a genetic risk factor for carbamazepine-induced cutaneous adverse drug reactions in Japanese population. Hum. Mol. Genet. 20(5), 1034-1041 (2011).
24. McCormack M, Alfirevic A, Bourgeois S et al. HLA-A 3101 and carbamazepine-induced hypersensitivity reactions in Europeans. N. Engl. J. Med. 364(12), 1134-1143 (2011).
25. Kurose K, Sugiyama E, Saito Y. Population differences in major functional polymorphisms of pharmacokinetics/pharmacodynamics-related genes in eastern asians and europeans: implications in the clinical trials for novel drug development. Drug Metab. Pharmacokinet. 27(1), 9-54 (2012).
26. Otsubo Y, Asahina Y, Noguchi A, Sato Y, Ando Y, Uyama Y. Similarities and differences between US and Japan as to pharmacogenomic biomarker information in drug labels. Drug Metab. Pharmacokinet. 27(1), 142-149 (2012).
27. Kelley RK, Atreya C, Venook AP, Febbo PG. Predictive biomarkers in advance of a companion drug: ahead of their time? J. Natl. Compr. Canc. Netw. 10(3), 303-309 (2012).
28. Cheng S, Koch WH, Wu L. Co-development of a companion diagnostic for targeted cancer therapy. N. Biotechnol. 29(6), 682-688 (2012).
29. Committee for Medicinal Products for Human Use. Reflection Paper on Co-Development of Pharmacogenomic Biomarkers and Assays in the Context of Drug Development: Draft. EMA, London, UK (2010).
30. US FDA. Draft Guidance for Industry and Food and Drug Administration Staff: In Vitro Companion Diagnostic Devices. US FDA, MD, USA (2011).
31. Japan Health Science Foundation. Report on Basic Technology: Medical Needs in 2015. Japan Health Science Foundation, Tokyo, Japan (2005).
32. Kola I, Landis J. Can the pharmaceutical industry reduce attrition rates? Nat. Rev. Drug Discov. 3(8), 711-716 (2004).
33. Narasimhan S, Lohoff FW. Pharmacogenetics of antidepressant drugs: current clinical practice and future directions. Pharmacogenomics 13(4), 441-464 (2012).
34. Pilotto A, Panza F, Seripa D. Pharmacogenetics in geriatric medicine: challenges and opportunities for clinical practice. Curr. Drug Metab. 12(7), 621-634 (2011).
35. Goldman J, Becker ML, Jones B, Clements M, Leeder JS. Development of biomarkers to optimize pediatric patient management: what makes children different? Biomarker Med. 5(6), 781-794 (2011).
36. Biomarkers related to drug or biotechnology product development: context, structure and format of qualification submissions. International Conference on Harmonisation, Geneva Switzerland (2010). www.ich.org/fileadmin/Public-Web-Site/ ICH-Products/Guidelines/Efficacy/E16/Step4/E16-Step-4.pdf
37. PMDA Companion Diagnostics Project. www.pmda.go.jp/kijunsakusei/ companion. html