Surrogate Endpoints and Drug Regulation: What Is Needed to Clarify the Evidence

Abstract

The FDA's new table of surrogate endpoints used for drug approvals is an important step forward for overseeing the use of biomarkers in clinical trials. Nevertheless, we present several ways in which the table can be improved.

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References

A. S.

Kesselheim and

Karlawish , “Biomarkers Unbound — The Supreme Court's Ruling on Diagnostic-Test Patents,” New England Journal of Medicine 366, no. 25 (2012): 2338-2340.

T. R.

Fleming and

D. L.

DeMets , “Surrogate End Points in Clinical Trials: Are We Being Misled?” Annals of Internal Medicine 125, no. 7 (1996): 605-613.

US Food and Drug Administration, “Table of Surrogate Endpoints That Were the Basis of Drug Approval or Licensure,” available at <https://www.fda.gov/Drugs/DevelopmentApprovalProcess/DevelopmentResources/ucm613636.htm> (last visited July 17, 2019).

Id.

E. L.

Korn ,

P. S.

Albert , and

L. M.

McShane , “Assessing Surrogates as Trial Endpoints Using Mixed Models,” Statistics in Medicine 24, no. 2 (2005): 163-182.

K. J.

Lipska and

H. M.

Krumholz , “Is Hemoglobin A1c the Right Outcome for Studies of Diabetes?” JAMA 317, no. 10 (2017): 1017-1018.

H. K.

Reddel ,

D. R.

Taylor ,

E. D.

Bateman ,

L. P.

Boulet ,

H. A.

Boushey ,

W. W.

Busse ,

T. B.

Casale ,

Chanez ,

P. L.

Enright ,

P. G.

Gibson , and

J.C.

de Jongste , “An official American Thoracic Society/European Respiratory Society Statement: Asthma Control and Exacerbations: Standardizing Endpoints for Clinical Asthma Trials and Clinical Practice,” American Journal of Respiratory and Critical Care Medicine 180, no. 1 (2009): 59-99.

I. J.

Saldanha ,

Dickersin ,

Wang , and

Li , “Outcomes in Cochrane Systematic Reviews Addressing Four Common Eye Conditions: An Evaluation of Completeness and Comparability,” PloS One 9, no. 10 (2014): e109400.