Hostname: page-component-745bb68f8f-5r2nc Total loading time: 0 Render date: 2025-01-11T22:46:44.361Z Has data issue: false hasContentIssue false
  • English
  • Français

Biomarkers as Surrogate Endpoints: Ongoing Opportunities for Validation

Published online by Cambridge University Press:  01 January 2021

Audrey D. Zhang  and
Joseph S. Ross
Get access Rights & Permissions [Opens in a new window]

Abstract

Surrogate endpoints are a common application of biomarkers to estimate clinical benefit in clinical trials, despite questions about reliability. This article discusses ongoing opportunities for their validation, in the context of a regulatory environment in which they are increasingly championed.

Type
Symposium Articles
Information
Journal of Law, Medicine & Ethics , Volume 47 , Issue 3: Biomarker Research and Validation: Current Challenges, Future Opportunities , Fall 2019 , pp. 393 - 395
Copyright
Copyright © American Society of Law, Medicine and Ethics 2019

Access options

Get access to the full version of this content by using one of the access options below. (Log in options will check for institutional or personal access. Content may require purchase if you do not have access.)

References

Prasad, V., Kim, C., Burotto, M., and Vandross, A., “The Strength of Association Between Surrogate End Points and Survival in Oncology: A Systematic Review of Trial-Level Meta-Analyses,” JAMA Internal Medicine 175, no. 8 (2015): 1389-1398, doi: http://dx.doi.org/10.1001/jamainternmed.2015.2829.CrossRefGoogle Scholar
Yudkin, J. S., Lipska, K. J., and Montori, V. M., “The Idolatry of the Surrogate,” BMJ 343 (2011): D7995, doi: http://dx.doi.org/10.1136/bmj.d7995; Rodriguez-Gutierrez, R. and McCoy, R. G., “Measuring What Matters in Diabetes,” JAMA 321, no. 19 (2019): 1865-1866, doi: http://dx.doi.org/10.1001/jama.2019.4310.CrossRefGoogle Scholar
Downing, N. S., Aminawung, J. A., Shah, N. D., Krumholz, H. M., and Ross, J. S., “Clinical Trial Evidence Supporting FDA Approval of Novel Therapeutic Agents, 2005-2012,” JAMA 311, no. 4 (2014): 368–377, doi: http://dx.doi.org/10.1001/jama.2013.282034.CrossRefGoogle Scholar
Subpart H—Accelerated Approval of New Drugs for Serious or Life-Threatening Illnesses; 1992, available at <https://www.ecfr.gov/cgi-bin/text-idx?SID=9dac4b84312496344a53b899f75218e8&mc=true&node=sp21.5.314.h&rgn=div6> (last visited May 24, 2019).+(last+visited+May+24,+2019).>Google Scholar
Beaver, J. A., Howie, L. J., Pelosof, L., et al., “A 25-Year Experience of US Food and Drug Administration Accelerated Approval of Malignant Hematology and Oncology Drugs and Biologics: A Review,” JAMA Oncology 4, no. 6 (2018): 849-856, doi: http://dx.doi.org/10.1001/jamaoncol.2017.5618.CrossRefGoogle Scholar
Pease, A. M., Krumholz, H.M. Downing, N. S., Aminawung, J. A., Shah, N. D., and Ross, J. S., “Postapproval Studies of Drugs Initially Approved by the FDA on the Basis of Limited Evidence: Systematic Review,” BMJ 357 (2017): j1680, doi: http://dx.doi.org/10.1136/bmj.j1680.Google Scholar
Wallach, J. D., Ross, J. S., and Naci, H., “The US Food and Drug Administration's Expedited Approval Programs: Evidentiary Standards, Regulatory Trade-Offs, and Potential Improvements,” Clinical Trials 15, no. 3 (2018): 219-229, doi: http://dx.doi.org/10.1177/1740774518770648; Wallach, J. D., Egilman, A. C., Dhruva, S. S., et al., “Postmarket Studies Required by the US Food and Drug Administration for New Drugs and Biologics Approved Between 2009 and 2012: Cross Sectional Analysis,” BMJ 361 (2018): k2031, doi: http://dx.doi.org/10.1136/bmj.k2031.CrossRefGoogle Scholar
Strom, B. L., Buyse, M., Hughes, J., and Knoppers, B. M., “Data Sharing, Year 1 — Access to Data from Industry-Sponsored Clinical Trials,” New England Journal of Medicine 371, no. 22 (2014): 2052-2054, doi: http://dx.doi.org/10.1056/NEJMp1411794.CrossRefGoogle Scholar
Ross, J. S., Waldstreicher, J., Bamford, S., et al., “Overview and Experience of the YODA Project with Clinical Trial Data Sharing After 5 Years,” Scientific Data 5 (2018): 180268, doi: http://dx.doi.org/10.1038/sdata.2018.268.Google Scholar