What is a biomarker? Research investments and lack of clinical integration necessitate a review of biomarker terminology and validation schema

Scandinavian Journal of Clinical and Laboratory Investigation

Volumn 70, Issue SUPPL. 242, 2010, Pages 6-14

  Ptolemy, Adam S ^a   Rifai, Nader ^a  

^a BOSTON CHILDREN S HOSPITAL   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BIOLOGICAL MARKER;

CLINICAL EVALUATION; CLINICAL RESEARCH; DIAGNOSTIC ACCURACY; DIAGNOSTIC TEST; DISEASE MARKER; FINANCIAL MANAGEMENT; GENOMICS; OUTCOME ASSESSMENT; PATIENT CARE; PRIORITY JOURNAL; PROTEOMICS; PUBLICATION; QUALITATIVE ANALYSIS; REVIEW; SENSITIVITY AND SPECIFICITY; VALIDATION PROCESS; ANIMAL; DIAGNOSIS, MEASUREMENT AND ANALYSIS; HUMAN; MEDICAL RESEARCH; NATIONAL HEALTH ORGANIZATION; NOMENCLATURE; REPRODUCIBILITY; STANDARD; UNITED STATES;

ANIMALS; BIOLOGICAL MARKERS; BIOMEDICAL RESEARCH; CLINICAL LABORATORY TECHNIQUES; HUMANS; LABORATORY TECHNIQUES AND PROCEDURES; NATIONAL INSTITUTES OF HEALTH (U.S.); REPRODUCIBILITY OF RESULTS; RESEARCH SUPPORT AS TOPIC; TERMINOLOGY AS TOPIC; UNITED STATES;

EID: 77953148386     PISSN: 00365513     EISSN: 15027686     Source Type: Journal    
DOI: 10.3109/00365513.2010.493354     Document Type: Review

References (34)

1. Rifai N, Gillette MA, Carr SA. Protein biomarker discovery and validation: the long and uncertain path to clinical utility. Nat Biotechnol 2006;24:971-83.
2. Anderson NL. The clinical plasma proteome: a survey of clinical assays for proteins in plasma and serum. Clin Chem 2010;56:177-85.
3. Group B D W. Biomarkers and surrogate endpoints: preferred definitions and conceptual framework. Clin Pharmacol Ther 2001;69:89-95.
4. De Gruttola VG, Clax P, De Mets DL, Downing GJ, Elenberg SS, Friedmann L, et al. Considerations in the evaluation of surrogate endpoints in clinical trails: summary of a national institutes of health workshop. Control Clin Trials 2001;22:485-502.
5. Wagner JA. Overview of biomarkers and surrogate endpoints in drug development. Disease Markers 2002;18:41-6.
6. Boissel J-P, Collet J-P, Moleur P, Haugh M. Surrogate endpoints: a basis for a rational approach. Eur J Clin Pharmacol 1992;43:235-44.
7. Katz R. Biomarkers and surrogate markers: an FDA perspective. Journal of the American Society for Neuro-Therapeutics 2004;1:189-95.
8. Johnson JR, Williams G, Pazdur R. End points and united states food and drug approval on oncology drugs. J Clin Oncol 2003;21:1404-11.
9. Wied C C G D, Kritsidima M, Elferink A J A. The validity of biomarkers as surrogate endpoints in Alzheimer's disease by means of the quantitative surrogate validation level of evidence scheme (QSVLES). J Nutr Health Aging 2009;13:376-87.
10. Lassere MN. The biomarker-surrogacy evaluation schema: a review of the biomarker-surrogate literature and a proposal for criterion-based, quantitative, multidimensional hierarchial levels of evidence schema for evaluating the status of biomarkers as surrogate endpoints. Stat Methods Med Res 2008;17:303-40.
11. Pepe MS, Etzioni R, Feng Z, Potter JD, Thompson ML, Thornquist M, et al. Phases of biomarker development for early detection of cancer. J Natl Cancer Inst 2001;93:1054-61.
12. Feng Z, Prentice R, Srivastava S. Research issues and strategies for genomic and proteomic biomaker discovery and validation: a statistical perspective. Pharmacogenomics 2004;5:709-19.
13. Cook NR. Statistical evaluation of the prognostic versus diagnostic models: beyond the ROC curve. Clin Chem 2008;54:17-23.
14. Dupuy A, Simon RM. Critical review of published microarray studies for cancer outcome and guidelines on statistical analysis and reporting. J Natl Cancer Inst 2007;99:147-57.
15. Tahara H, Sato M, Thurin M, Wang E, Butterfield LH, Disis ML, et al. Emerging concepts in biomarker discovery; the US-Japan workshop on immunological molecular markers in oncology. J Transl Med 2009;7:45.
16. Mullins C, Lucia MS, Hayward SW, Lee JY, Levitt JM, Lin VK, et al. A comprehensive approach toward novel serum biomarkers for benign prostatic hyperplasia: the MPSA consortium. J Urol 2008;179:1243-56.
17. Hlatky MA, Greenland P, Arnett DK, Ballantyne CM, Cirqui MH, Elkind M S V, et al. Criteria for evaluation of novel markers of cardiovascular risk: a scientific statement from the American Heart Association. Circulation 2009;119:2408-16.
18. Ransohoff DF. How to improve reliability and efficiency of research about molecular markers: roles of phases, guidelines, and study design J Clin Epidemio l 2007;60:1205-19.
19. Butterfield LH, Disis ML, Fox BA, Lee PP, Khleif SN, Thurin M, et al. A systemic approach to biomarker discovery; preamble to the "iSBTc-FDA taskforce on immunotherapy biomarkers". J Trans Med 2008;6:81.
20. Osborne M. Breast cancer prevention - biomarkers and intervention. National Cancer Institute, 1986; Project Number: 5P01CA38401-03.
21. Bowden DM. Primate resource and biomarker development. National Institute of Aging 1986; Project Number: Contract.
22. Chatterjee B. Age and hormone dependent regulation of hepatic protein. National Institute on Aging, 1986; Project Number: 2R01AG003527-04.
23. Hede K. $ 104 million proteomics initiative gets green light. J Natl Cancer Inst 2005;97:1324-5.
24. Goldberg KB. Advisors reject NCI's $ 89 million plan for proteomics as too much too soon. Cancer Lett 2005;31:1-10.
25. Food and Drug Administration Department of Health and Human Services, Approval based on a surrogate endpoint or on an effect on a clinical endpoint other than survival or irreversible morbidity, 2009;5:21CFR314. 510.
26. Simon R. Lost in translation: problems and pitfalls in translating laboratory observations into clinical utility. Eur J Cancer 2008;44:2707-13.
27. Mankoff SP, Brander C, Ferrone S, Marincola FM. Lost in translation: obstacles to translational medicine. J Trans Med 2004;2:14.
28. Simon R. The use of genomics in clinical trial design. Clin Cancer Res 2008;14:5984-93.
29. Pepe MS, Feng Z, Janes H, Bossuyt PM, Potter JD. Pivotal evaluation of the accuracy of a biomarker used for classification or prediction: standards for study design. J Natl Cancer Inst 2008;100:1432-8.
30. Bossuyt PM, Reitsma JB, Burns DE, Gatsonis CA, Glasziou PP, Irwig LM, et al. Towards complete and accurate reporting of studies of diagnostic accuracy: the STARD initiative. Ann Intern Med 2003;138:40-4.
31. Shane LM, Altman DG, Sauerbrei W, Taube SE, Gion M, Clark GM. Reporting recommendations for tumour marker prognostic studies (REMARK). J Natl Cancer Inst 2005;97:1180-4. (Pubitemid 41258223)
32. Brazma A, Hingamp P, Quackenbush J, Sherlock G, Spellman P, Stoeckert C, et al. Minimum information about a microarray experiment (MIAME)-towards standards for microarray data. Nat Genet 2001;29:365-71.
33. Pepe MS, Feng Z, Longton G, Koopmeiners J. Conditional estimation of sensitivity and specificity from a phase 2 biomarker study allowing early termination for futility. Stat Med 2009;28:762-79.
34. Lijmer JG, Leeflang M, Bossuyt P M M. Proposal for phased evaluation of medical tests. Med Decis Making 2009;29: E13-E21.