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

Spencer Phillips Hey; William B Feldman; Emily H Jung; Elvira D'Andrea; Aaron S Kesselheim

doi:10.1177/1073110519876167

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

J Law Med Ethics. 2019 Sep;47(3):381-387. doi: 10.1177/1073110519876167.

Authors

Spencer Phillips Hey¹, William B Feldman¹, Emily H Jung¹, Elvira D'Andrea¹, Aaron S Kesselheim¹

Affiliation

¹ Spencer Phillips Hey, Ph.D., is with the Program On Regulation, Therapeutics, And Law (PORTAL), Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women's Hospital, Harvard Medical School, Boston, Massachusetts and Center for Bioethics, Harvard Medical School. William B. Feldman, M.D., D.Phil., is with the Program On Regulation, Therapeutics, And Law (PORTAL), Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women's Hospital, Harvard Medical School, Boston, Massachusetts and the Division of Pulmonary and Critical Care Medicine, Department of Medicine, Brigham and Women's Hospital. Emily H. Jung, A.B., is with the Program On Regulation, Therapeutics, And Law (PORTAL), Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women's Hospital, Harvard Medical School, Boston, Massachusetts. Elvira D'Andrea, M.D., M.P.H., is with the Program On Regulation, Therapeutics, And Law (PORTAL), Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women's Hospital, Harvard Medical School, Boston, Massachusetts. Aaron S. Kesselheim, M.D., J.D., M.P.H., Program On Regulation, Therapeutics, And Law (PORTAL), Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women's Hospital, Harvard Medical School, Boston, Massachusetts and the Center for Bioethics, Harvard Medical School.

PMID: 31560631
DOI: 10.1177/1073110519876167

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.

Publication types

Research Support, Non-U.S. Gov't

MeSH terms

Biomarkers*
Drug Approval / legislation & jurisprudence
Drug Approval / organization & administration
Guidelines as Topic / standards*
Humans
United States
United States Food and Drug Administration*

Substances

Biomarkers