A risk-management approach for effective integration of biomarkers in clinical trials: Perspectives of an NCI, NCRI, and EORTC working group

The Lancet Oncology

Volumn 15, Issue 4, 2014, Pages

  Hall, Jacqueline Anne ^a   Salgado, Roberto ^b,c   Lively, Tracy ^d   Sweep, Fred ^e   Schuh, Anna ^f  

^a EORTC DATA CENTER   (Belgium)

^b JULES BORDET INSTITUTE   (Belgium)

^c UNIVERSITY OF ANTWERP   (Belgium)

^d NATIONAL CANCER INSTITUTE   (United States)

^e RADBOUD UNIVERSITY MEDICAL CENTER   (Netherlands)

^f CHURCHILL HOSPITAL   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

BIOLOGICAL MARKER;

CLINICAL EFFECTIVENESS; EUROPEAN ORGANIZATION FOR RESEARCH AND TREATMENT OF CANCER; FOOD AND DRUG ADMINISTRATION; HEALTH CARE ORGANIZATION; HUMAN; MEDICAL SERVICE; NATIONAL CANCER RESEARCH INSTITUTE; NATIONAL HEALTH ORGANIZATION; NOTE; ORGANIZATIONAL DOWNSIZING; ORGANIZATIONAL EFFICIENCY; OUTCOME ASSESSMENT; PATIENT SAFETY; PRIORITY JOURNAL; PROCESS DEVELOPMENT; PROGRAM DEVELOPMENT; QUALITY CONTROL; RISK ASSESSMENT; RISK FACTOR; RISK MANAGEMENT; UNITED KINGDOM; UNITED STATES;

CHECKLIST; CLINICAL TRIALS AS TOPIC; CONSENSUS; EUROPE; HUMANS; NATIONAL CANCER INSTITUTE (U.S.); NEOPLASMS; PATIENT SELECTION; RESEARCH DESIGN; RESEARCH SUPPORT AS TOPIC; RISK ASSESSMENT; RISK FACTORS; RISK MANAGEMENT; TREATMENT OUTCOME; TUMOR MARKERS, BIOLOGICAL; UNITED STATES;

EID: 84897053056     PISSN: 14702045     EISSN: 14745488     Source Type: Journal    
DOI: 10.1016/S1470-2045(13)70607-7     Document Type: Note

References (76)

1. Schilsky RL, Doroshow JH, Leblanc M, Conley BA Development and use of integral assays in clinical trials. Clin Cancer Res 2012, 18:1540-1546.
2. Murugaesu N, Chew SK, Swanton C Adapting clinical paradigms to the challenges of cancer clonal evolution. Am J Pathol 2013, 182:1962-1971.
3. Comparison chart of guidelines P3G consotrium, (accessed Dec 13, 2013). http://www.p3gobservatory.org/repository/guidelines.htm.
4. Sweep FC, Fritsche HA, Gion M, Klee GG, Schmitt M Considerations on development, validation, application, and quality control of immuno(metric) biomarker assays in clinical cancer research: an EORTC-NCI working group report. Int J Oncol 2003, 23:1715-1726. for the EORTC-NCI Working Group.
5. 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 2012, 18:1515-1523.
6. McShane LM, Cavenagh MM, Lively TG, et al. Criteria for the use of omics-based predictors in clinical trials. Nature 2013, 502:317-320.
7. McShane LM, Cavenagh MM, Lively TG, et al. Criteria for the use of omics-based predictors in clinical trials: explanation and elaboration. BMC Med 2013, 11:220.
8. Freidlin B, Korn EL Biomarker enrichment strategies: matching trial design to biomarker credentials. Nat Rev Clin Oncol 2013, published online Nov 26. 10.1038/nrclinonc.2013.218.
9. Hayes DF, Allen J, Compton C, et al. Breaking a vicious cycle. Sci Transl Med 2013, 5:196.
10. Oversight of clinical investigations: a risk-based approach to monitoring. Guidance for industry US Department of Health and Human Services, Food and Drug AdministrationFood and Drug Administration, Center for Drug Evaluation and Research (CDER)Center for Drug Evaluation and Research (CDER), Center for Biologics Evaluation and Research (CBER)Center for Biologics Evaluation and Research (CBER), Center for Devices and Radiological Health (CDRH)Center for Devices and Radiological Health (CDRH), Office of Good Clinical Practice (OGCP)Office of Good Clinical Practice (OGCP), Office of Regulatory Affairs (ORA)Office of Regulatory Affairs (ORA), (accessed Jan 14, 2014). http://www.fda.gov/downloads/Drugs/.../Guidances/UCM269919.pdf.
11. Reflection paper on risk based quality management in clinical trials European Medicines Agency, (accessed Jan 14, 2014). http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2013/11/WC500155491.pdf.
12. Prognostic/predictive biomarker (BM) roadmap Cancer Research UK (CRUK), (accessed Jan 14, 2014). http://www.cancerresearchuk.org/prod_consump/groups/cr_common/@fre/@fun/documents/generalcontent/cr_027486.pdf.
13. Simon RM, Paik S, Hayes DF Use of archived specimens in evaluation of prognostic and predictive biomarkers. J Natl Cancer Inst 2009, 101:1446-1452.
14. Lewandowski J, Coxon K, Chambers L, Brown B Workshop report: biomarkers, biostatistics and novel clinical trial design (accessed Feb 20, 2014). http://csg.ncri.org.uk/wp-content/uploads/2013/10/NCRI-CSG_Workshop_Biomarkers-Biostatistics-and-Novel-Clinical-Trial-Design-Workshop_Meeting-Report.pdf.
15. Guidance on the maintenance of regulatory compliance in laboratories that perform the analysis or evaluation of clinical trial samples Medicine and Healthcare products Regulatory Agency (MHRA), (accessed Jan 14, 2014). http://www.mhra.gov.uk/home/groups/is-insp/documents/websiteresources/con051910.pdf.
16. ISO 15189 medical laboratories: requirements for quality and competence International Organization for Standardization (ISO), (accessed Jan 14, 2014). http://www.iso.org/iso/catalogue_detail?csnumber=56115.
17. Barker RW, Brindley DA, Schuh A Establish good genomic practice to guide medicine forward. Nat Med 2013, 19:530.
18. Study checklist for large randomized phase 2 and any phase 3 trials with biomarker assays/imaging tests Biomarker, ImagingImaging, QOL Studies Funding Program (BIQSFP)QOL Studies Funding Program (BIQSFP), (accessed Jan 14, 2014). http://www.cancer.gov/aboutnci/organization/ccct/funding/BIQSFP/BIQSFP-12-Biomarker-Imaging-Study-Checklist.
19. Clinical Laboratory Improvement Amendments (CLIA) Centers for Medicare and Medicaid Services (CMS), (accessed Jan 14, 2014). http://www.cms.gov/Regulations-and-Guidance/Legislation/CLIA/index.html.
20. Teutsch SM, Bradley LA, Palomaki GE, et al. The Evaluation of Genomic Applications in Practice and Prevention (EGAPP) Initiative: methods of the EGAPP Working Group. Genet Med 2009, 11:3-14.
21. Device advice: investigational device exemption (IDE) US Food and Drug Administration, (accessed Jan 14, 2014). http://www.fda.gov/MedicalDevices/DeviceRegulationandGuidance/HowtoMarketYourDevice/InvestigationalDeviceExemptionIDE/default.htm.
22. Guidance on IDE policies and procedures US Food and Drug Administration, (accessed Jan 14, 2014). http://www.fda.gov/MedicalDevices/DEviceRegulationandGuidance/GuidanceDocuments/ucm080202.htm.
23. Use of International Standard ISO-10993. Biological Evaluation of Medical Devices Part 1: Evaluation and Testing US Department of Health and Human Services, Food and Drug AdministrationFood and Drug Administration, Center for Devices and Radiological HealthCenter for Devices and Radiological Health, Office of Device Evaluation Use of International StandardOffice of Device Evaluation Use of International Standard, (accessed Jan 14, 2014). http://www.fda.gov/downloads/MedicalDevices/DeviceRegulationandGuidance/GuidanceDocuments/UCM348890.pdf.
24. The European Parliament and the Council of the European Union. Directive 98/79/EC of the European Parliament and of the Council of 27 October 1998 on in vitro diagnostic medical devices. Official Journal of the European Communities 1998, Publications Office of the European Union (OP), p. L 331/1-/37.
25. The European Parliament and the Council of the European Union. Directive 2001/20/EC of the European Parliament and of the Council of 4 April 2001 on the approximation of the laws, regulations and administrative provisions of the Member States relating to the implementation of good clinical practice in the conduct of clinical trials on medicinal products for human use. Official Journal of the European Communities 2001, Publications Office of the European Union (OP), p. L 121/34 - /44.
26. Proposal for a regulation of the European parliament and of the council on clinical trials on medicinal products for human use, and repealing Directive 2001/20/EC European Commission, (accessed Jan 14, 2014). http://eur-lex.europa.eu/LexUriServ/LexUriServ.do?uri=COM:2012:0369:FIN:EN:PDF.
27. OECD recommendation on the governance of clinical trials OECD, (accessed Jan 14, 2014). http://www.oecd.org/sti/sci-tech/oecd-recommendation-governance-of-clinical-trials.pdf.
28. Principles of IVD medical devices classification Global Harmonization Task Force, (accessed Jan 14, 2014). http://www.imdrf.org/docs/ghtf/final/sg1/procedural-docs/ghtf-sg1-n045-2008-principles-ivd-medical-devices-classification-080219.pdf.
29. Reflection paper for laboratories that perform the analysis or evaluation of clinical trial samples European Medicine Agency, (accessed Jan 14, 2014). http://www.ema.europa.eu/docs/en_GB/document_library/Regulatory_and_procedural_guideline/2012/05/WC500127124.pdf.
30. Hall JA, Salgado R, Peters GJ, et al. Quality assurance of biomarker assays in clinical trials. Eur J Cancer 2012, 48:137.
31. Warth A, Penzel R, Brandt R, et al. Optimized algorithm for Sanger sequencing-based EGFR mutation analyses in NSCLC biopsies. Virchows Arch 2012, 460:407-414.
32. Thunnissen E, Bubendorf L, Dietel M, et al. EML4-ALK testing in non-small cell carcinomas of the lung: a review with recommendations. Virchows Arch 2012, 461:245-257.
33. Reflection paper on methodological issues associated with pharmacogenomic biomarkers in relation to clinical development and patient selection Draft European Medicines Agency, (accessed Jan 14, 2014). http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2011/07/WC500108672.pdf.
34. Lee JM, Hays JL, Noonan AM, et al. Feasibility and safety of sequential research-related tumor core biopsies in clinical trials. Cancer 2013, 119:1357-1364.
35. Structured approach to benefit-risk assessment in drug regulatory decision-making. Draft PDUFA V implementation plan: February 2013. Fiscal years 2013-2017 US Food and Drug Administration, (accessed Jan 14, 2014). http://www.fda.gov/downloads/forindustry/userfees/prescriptiondruguserfee/ucm329758.pdf.
36. Benefit-risk methodology project European Medicines Agency, (accessed Jan 14, 2014). http://www.ema.europa.eu/ema/index.jsp?curl=pages/special_topics/document_listing/document_listing_000314.jsp#.
37. Gymrek M, McGuire AL, Golan D, Halperin E, Erlich Y Identifying personal genomes by surname inference. Science 2013, 339:321-324.
38. Kim KK, Browe DK, Logan HC, Holm R, Hack L, Ohno-Machado L Data governance requirements for distributed clinical research networks: triangulating perspectives of diverse stakeholders. J Am Med Inform Assoc 2013, published online Dec 3. 10.1136/amiajnl-2013-002308.
39. OECD principles and guidelines for access to research data from public funding OECD, (accessed Jan 14, 2014). http://www.oecd.org/sti/sci-tech/38500813.pdf.
40. Think tank on identifiability of biospecimens and -omic data National Cancer Institute, (accessed Jan 14, 2014). http://epi.grants.cancer.gov/workshops/identifiability/think-tank-summary.pdf.
41. Bookman EB, Din-Lovinescu C, Worrall BB, et al. Incidental genetic findings in randomized clinical trials: recommendations from the Genomics and Randomized Trials Network (GARNET). Genome Med 2013, 5:7.
42. Green RC, Berg JS, Grody WW, et al. ACMG recommendations for reporting of incidental findings in clinical exome and genome sequencing. Genet Med 2013, 15:565-574.
43. Stilgenbauer S, Bullinger L, Lichter P, Döhner H Genetics of chronic lymphocytic leukemia: genomic aberrations and VH gene mutation status in pathogenesis and clinical course. Leukemia 2002, 16:993-1007. for the German CLL Study Group (GCLLSG).
44. Perner S, Wagner PL, Demichelis F, et al. EML4-ALK fusion lung cancer: a rare acquired event. Neoplasia 2008, 10:298-302.
45. Cardoso F, Piccart-Gebhart M, Van't Veer L, Rutgers E The MINDACT trial: the first prospective clinical validation of a genomic tool. Mol Oncol 2007, 1:246-251. the TRANSBIG Consortium.
46. Evans PA, Short MA, Owen RG, et al. Residual disease detection using fluorescent polymerase chain reaction at 20 weeks of therapy predicts clinical outcome in childhood acute lymphoblastic leukemia. J Clin Oncol 1998, 16:3616-3627.
47. Werutsky G, van 't Veer LJ, Cardoso F, et al. The EORTC 10041/BIG 03-04 MINDACT Trial Quality Assurance Program: results of the questionnaire for pathologists. Eur J Cancer 2012, 48:166.
48. Hall JA, Daidone MG, Peters GJ, Harbeck N, Lacombe D, Sleijfer S Integrating collection of biospecimens in clinical trials: the approach of the European Organization for Research and Treatment of Cancer. Biopreserv Biobank 2011, 9:181-186.
49. Bogaerts J, Cardoso F, Buyse M, et al. Gene signature evaluation as a prognostic tool: challenges in the design of the MINDACT trial. Nat Clin Pract Oncol 2006, 3:540-551. for the TRANSBIG consortium.
50. Perez EA, Suman VJ, Davidson NE, et al. HER2 testing by local, central, and reference laboratories in specimens from the North Central Cancer Treatment Group N9831 intergroup adjuvant trial. J Clin Oncol 2006, 24:3032-3038.
51. Wolff AC, Hammond ME, Schwartz JN, et al. American Society of Clinical Oncology/College of American Pathologists guideline recommendations for human epidermal growth factor receptor 2 testing in breast cancer. J Clin Oncol 2007, 25:118-145.
52. Geurts-Moespot J, Leake R, Benraad TJ, Sweep CG Twenty years of experience with the steroid receptor external quality assessment program: the paradigm for tumour biomarker EQA studies. On behalf of the EROTC Receptor and Biomarker Study Group. Int J Oncol 2000, 17:13-22.
53. van Krieken JH, Siebers AG, Normanno N European consensus conference for external quality assessment in molecular pathology. Ann Oncol 2013, 24:1958-1963.
54. Guide to CAP proficiency testing/external quality assurance for international participants College of American Pathologists, (accessed Jan 14, 2014). http://www.cap.org/apps/docs/proficiency_testing/cap_proficiency_testing_guide.pdf.
55. Foroni L, Wilson G, Gerrard G, et al. Guidelines for the measurement of BCR-ABL1 transcripts in chronic myeloid leukaemia. Br J Haematol 2011, 153:179-190.
56. Williams PM, Lively TG, Jessup JM, Conley BA Bridging the gap: moving predictive and prognostic assays from research to clinical use. Clin Cancer Res 2012, 18:1531-1539.
57. Sarzotti-Kelsoe M, Cox J, Cleland N, et al. Evaluation and recommendations on good clinical laboratory practice guidelines for phase I-III clinical trials. PLoS Med 2009, 6:e1000067.
58. Recommendations for in-house development and operation of molecular diagnostic tests. Am J Clin Pathol 1999, 111:449-463. Association for Molecular Pathology statement.
59. Baggerly KA, Coombes KR What information should be required to support clinical "omics" publications?. Clin Chem 2011, 57:688-690.
60. Moore HM, Kelly AB, Jewell SD, et al. Biospecimen reporting for improved study quality (BRISQ). Cancer Cytopathol 2011, 119:92-101.
61. Hans CP, Weisenburger DD, Greiner TC, et al. Confirmation of the molecular classification of diffuse large B-cell lymphoma by immunohistochemistry using a tissue microarray. Blood 2004, 103:275-282.
62. Plummer R, Jones C, Middleton M, et al. Phase I study of the poly(ADP-ribose) polymerase inhibitor, AG014699, in combination with temozolomide in patients with advanced solid tumors. Clin Cancer Res 2008, 14:7917-7923.
63. Yap TA, Sandhu SK, Workman P, de Bono JS Envisioning the future of early anticancer drug development. Nat Rev Cancer 2010, 10:514-523.
64. Goodwin R, Giaccone G, Calvert H, Lobbezoo M, Eisenhauer EA Targeted agents: how to select the winners in preclinical and early clinical studies?. Eur J Cancer 2012, 48:170-178.
65. Baccarani M, Cortes J, Pane F, et al. Chronic myeloid leukemia: an update of concepts and management recommendations of European LeukemiaNet. J Clin Oncol 2009, 27:6041-6051.
66. Leyland-Jones B, Smith BR Serum HER2 testing in patients with HER2-positive breast cancer: the death knell tolls. Lancet Oncol 2011, 12:286-295.
67. Paik S, Shak S, Tang G, et al. A multigene assay to predict recurrence of tamoxifen-treated, node-negative breast cancer. N Engl J Med 2004, 351:2817-2826.
68. McShane LM Statistical challenges in the development and evaluation of marker-based clinical tests. BMC Med 2012, 10:52.
69. Altman DG, McShane LM, Sauerbrei W, Taube SE Reporting Recommendations for Tumor Marker Prognostic Studies (REMARK): explanation and elaboration. PLoS Med 2012, 9:e1001216.
70. Paik S, Bryant J, Tan-Chiu E, et al. Real-world performance of HER2 testing: National Surgical Adjuvant Breast and Bowel Project experience. J Natl Cancer Inst 2002, 94:852-854.
71. Simonds NI, Khoury MJ, Schully SD, et al. Comparative effectiveness research in cancer genomics and precision medicine: current landscape and future prospects. J Natl Cancer Inst 2013, 105:929-936.
72. Hegi ME, Diserens AC, Gorlia T, et al. MGMT gene silencing and benefit from temozolomide in glioblastoma. N Engl J Med 2005, 352:997-1003.
73. Norlin L, Fransson MN, Eriksson M, et al. A minimum data set for sharing biobank samples, information, and data: MIABIS. Biopreserv Biobank 2012, 10:343-348.
74. Hall JA, Brown R Developing translational research infrastructure and capabilities associated with cancer clinical trials. Expert Rev Mol Med 2013, 15:11.
75. Bonnefoi H, Piccart M, Bogaerts J, et al. TP53 status for prediction of sensitivity to taxane versus non-taxane neoadjuvant chemotherapy in breast cancer (EORTC 10994/BIG 1-00): a randomised phase 3 trial. Lancet Oncol 2011, 12:527-539. for the EORTC 10994/BIG 1-00 Study Investigators.
76. Standards for the Medical Laboratory Clinical Pathology Accreditation (UK) Ltd, (accessed Jan 14, 2014). http://www.cpa-uk.co.uk/files/PD-LAB-Standards_v2.02_Nov_2010.pdf.