Surrogate Endpoints in Pediatric Studies Submitted to the US FDA

Dionna J Green; Haihao Sun; Janelle Burnham; Xiaomei I Liu; John van den Anker; Jean Temeck; Lynne Yao; Susan K McCune; Gilbert J Burckart

doi:10.1002/cpt.1117

Surrogate Endpoints in Pediatric Studies Submitted to the US FDA

Clin Pharmacol Ther. 2019 Mar;105(3):555-557. doi: 10.1002/cpt.1117. Epub 2018 Aug 9.

Authors

Dionna J Green¹, Haihao Sun², Janelle Burnham¹, Xiaomei I Liu³, John van den Anker³, Jean Temeck², Lynne Yao^{3

4}, Susan K McCune², Gilbert J Burckart¹

Affiliations

¹ Office of Clinical Pharmacology, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, Maryland, USA.
² Office of Pediatric Therapeutics, Commissioner's Office, US Food and Drug Administration, Silver Spring, Maryland, USA.
³ Children's National Medical Center, Washington, DC, USA.
⁴ Office of New Drugs, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver, Spring, Maryland, USA.

Abstract

The 21^st Century Cures Act was passed in December, 2016, and included a number of provisions to facilitate drug approval. Considerable discussion was generated related to some aspects of the Act, especially to the use of surrogate endpoints (SEs) as a means to shorten the time required prior to receiving US Food and Drug Administration (FDA) approval.¹ The objective of this analysis was to identify the use and outcomes of SEs and clinical endpoints in pediatric drug development trials.

Publication types

Review

MeSH terms

Biomarkers
Child
Clinical Trials as Topic / methods*
Clinical Trials as Topic / standards*
Drug Approval / methods*
Humans
United States
United States Food and Drug Administration / standards*

Substances

Biomarkers

Grants and funding

FD999999/ImFDA/Intramural FDA HHS/United States