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Biomarker Validation: Context and Complexities

Published online by Cambridge University Press:  01 January 2021

Lisa M. McShane
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Abstract

Validation of a biomarker-based medical product development tool or clinical test is an evidentiary process that must be tailored to the proposed use. Appropriate data and analyses are needed to demonstrate that the biomarker meets analytical and clinical performance criteria consistent with favorable benefit: risk balance.

Type
Symposium Articles
Information
Journal of Law, Medicine & Ethics , Volume 47 , Issue 3: Biomarker Research and Validation: Current Challenges, Future Opportunities , Fall 2019 , pp. 388 - 392
Copyright
Copyright © American Society of Law, Medicine and Ethics 2019

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References

FDA-NIH Biomarker Working Group, BEST (Biomarkers, EndpointS, and other Tools) Resource (Silver Spring, MD: Food and Drug Administration [US]; Bethesda, MD: National Institutes of Health [US], 2016-), available at <https://www.ncbi.nlm.nih.gov/books/NBK326791/> (last visited July 22, 2019).+(last+visited+July+22,+2019).>Google Scholar
Id.Google Scholar
Id.Google Scholar
Leptak, C., Menetski, J. P., Wagner, J. A., Aubrecht, J., Brady, L., Brumfield, M., Chin, W. W., Hoffmann, S., Kelloff, G., Lavezzari, G., Ranganathan, R., Sauer, J-M., Sistare, F. D., Zabka, T., and Wholley, D., “What Evidence Do We Need for Biomarker Qualification?” Science Translational Medicine 9, no. 417 (2017): eaal4599.CrossRefGoogle Scholar
“Biomarkers Consortium — Workshop: Defining an Evidentiary Criteria Framework for Surrogate Endpoint Qualification,” Foundation for the National Institutes of Health, available at <https://fnih.org/what-we-do/biomarkers-consortium/programs/biomarkers-consortium-workshop-defining-evidentiary-criteria-framework-surrogate-endpoint> (last visited July 22, 2019).+(last+visited+July+22,+2019).>Google Scholar
“Surrogate Endpoint Resources for Drug and Biologic Development,” U.S. Food and Drug Administration, available at <https://www.fda.gov/drugs/development-resources/surrogate-endpoint-resources-drug-and-biologic-development> (last visited July 22, 2019).+(last+visited+July+22,+2019).>Google Scholar
“Table of Surrogate Endpoints that were the Basis of Drug Approval or Licensure,” U.S. Food and Drug Administration, available at <https://www.fda.gov/drugs/development-resources/table-surrogate-endpoints-were-basis-drug-approval-or-licensure> (last visited July 22, 2019).+(last+visited+July+22,+2019).>Google Scholar
Hey, S. P., Feldman, W. B., Jung, E., D'Andrea, E., and Kesselheim, A. S., “Surrogate Endpoints and Drug Regulation: What is Needed to Clarify the Evidence,” Journal of Law, Medicine & Ethics 47, no. 3 (2019): 381-387.CrossRefGoogle Scholar
Hey, S. P., D'Andrea, E., Jung, E., Tessema, F., Lou, J., Gyawali, B., and Kesselheim, A. S., “Challenges and Opportunities for Bio-marker Validation,” Journal of Law, Medicine & Ethics 47, no. 3 (2019): 357-361.CrossRefGoogle Scholar
Teutsch, S. M., Bradley, L. A., Palomaki, G. E., Haddow, J. E., Piper, M., Calonge, N., Dotson, W. D., Douglas, M. P., and Berg, A.O. on behalf of the EGAPP Working, “The Evaluation of Genomic Applications in Practice and Prevention (EGAPP) Initiative: Methods of the EGAPP Working Group,” Genetics in Medicine 11, no. 1 (2009): 3-14; L.M. McShane and D.F. Hayes, Publication of Tumor Marker Research Results: The Necessity for Complete and Transparent Reporting, Journal of Clinical Oncology 30, no. 34 (2012): 4223-4232; D.R. Parkinson, R.T. McCormack, S.M. Keating, S.I. Gutman, S.R. Hamilton, E.A. Mansfield, M.A. Piper, P.A. DeVerka, F.W. Frueh, J.M. Jessup, L.M. McShane, S.R. Tunis, C.C. Sigman, and G.J. Kelloff, Evidence of Clinical Utility: An Unmet Need in Molecular Diagnostics for Cancer Patients, Clinical Cancer Research 20, no. 6 (2014): 1428-1444.CrossRefGoogle Scholar
“Clinical Laboratory Improvement Amendments (CLIA),” Centers for Medicare & Medicaid Services (CMS), available at <http://www.cms.gov/CLIA/05_CLIA_Brochures.asp> (last visited July 22, 2019).+(last+visited+July+22,+2019).>Google Scholar
Supra note 1.Google Scholar
Supra note 1.Google Scholar
McShane, L. M., Altman, D. G., Sauerbrei, W., Taube, S. E., Gion, M., and Clark, G.M. for the Statistics Subcommittee of the NCI-EORTC Working Group on Cancer Diagnostics, “REporting recommendations for tumor MARKer prognostic studies (REMARK),” Journal of the National Cancer Institute 97, no. 16 (2005): 1180-1184; Altman, D. G., McShane, L. M., Sauerbrei, W., and Taube, S. E., “Reporting Recommendations for Tumor Marker Prognostic Studies (REMARK): Explanation and Elaboration,” PLoS Medicine 9, no. 5 (2012): e1001216.CrossRefGoogle Scholar
“EQUATOR Network: Enhancing the QUAlity and Transparency of Health Research,” Equator Network, available at <http://www.equator-network.org/> (last visited July 22, 2019).+(last+visited+July+22,+2019).>Google Scholar
Li, R. and Sim, I., “How Clinical Trial Data Sharing Platforms Can Advance the Study of Biomarkers,” Journal of Law, Medicine & Ethics 47, no. 3 (2019): 369-373.CrossRefGoogle Scholar
National Cancer Institute NCTN/NCORP Data Archive, available at <https://nctn-data-archive.nci.nih.gov/> (last visited July 22, 2019).+(last+visited+July+22,+2019).>Google Scholar
Simon, R. M., Paik, S., and Hayes, D. F., “Use of Archived Specimens in Evaluation of Prognostic and Predictive Biomarkers,” Journal of the National Cancer Institute 101, no. 21 (2009): 1446-1452.CrossRefGoogle Scholar
Mavergames, C., Beecher, D., Becker, L., Last, A., and Ali, A., “Cochrane's Linked Data Project: How it can Advance our Understanding of Surrogate Endpoints,” Journal of Law, Medicine & Ethics 47, no. 3 (2019): 374-380.CrossRefGoogle Scholar
Shrager, J., Shapiro, M., and Hoos, W., “Is Cancer Solvable? Towards Efficient and Ethical Biomedical Science,” Journal of Law, Medicine & Ethics 47, no. 3 (2019): 362-368.CrossRefGoogle Scholar