Considering Functional Outcomes as Efficacy Endpoints in Pediatric Low-Grade Glioma Clinical Trials: An FDA Educational Symposium

Jason Fangusaro, Children's Hospital of Atlanta, Emory University and the Aflac Cancer Center, Atlanta, Georgia.
Robert A. Avery, The Children's Hospital of Philadelphia and the University of Pennsylvania Perelman School of Medicine, Philadelphia, Pennsylvania.
Michael J. Fisher, The Children's Hospital of Philadelphia and the University of Pennsylvania Perelman School of Medicine, Philadelphia, Pennsylvania.
Roger J. Packer, Center for Neuroscience and Behavioral Medicine and Brain Tumor Institute, Children's National Hospital, and The George Washington University School of Medicine, Washington, DC.
Karin S. Walsh, Center for Neuroscience and Behavioral Medicine and Brain Tumor Institute, Children's National Hospital, and The George Washington University School of Medicine, Washington, DC.
Antoinette Schouten-van Meeteren, Princess Máxima Center for Pediatric Oncology, Utrecht, the Netherlands.
Dominik Karres, Pediatric Medicines Office, Scientific Evidence Generation Department, Human Medicines Division, European Medicines Agency (EMA), Amsterdam, the Netherlands.
Diana Bradford, Center for Drug Evaluation and Research, Office of New Drugs, U.S. Food and Drug Administration, Silver Spring, Maryland.
Vishal Bhatnagar, Center for Drug Evaluation and Research, Office of New Drugs, U.S. Food and Drug Administration, Silver Spring, Maryland.
Harpreet Singh, Center for Drug Evaluation and Research, Office of New Drugs, U.S. Food and Drug Administration, Silver Spring, Maryland.
Paul G. Kluetz, Center for Drug Evaluation and Research, Office of New Drugs, U.S. Food and Drug Administration, Silver Spring, Maryland.
Martha Donoghue, Center for Drug Evaluation and Research, Office of New Drugs, U.S. Food and Drug Administration, Silver Spring, Maryland.
Elizabeth S. Duke, Center for Drug Evaluation and Research, Office of New Drugs, U.S. Food and Drug Administration, Silver Spring, Maryland.

Document Type

Journal Article

Publication Date

6-3-2024

Journal

Clinical cancer research : an official journal of the American Association for Cancer Research

Volume

30

Issue

11

DOI

10.1158/1078-0432.CCR-23-3386

Abstract

In October 2022, the FDA Oncology Center of Excellence hosted an educational symposium entitled, "Considering Functional Outcomes as Efficacy Endpoints in Pediatric Low-Grade Glioma (pLGG) Clinical Trials." The symposium brought together patient advocates, regulators from the FDA and the European Medicines Agency (EMA), and an international group of academic thought leaders in the field of pediatric neuro-oncology to discuss the potential role of functional outcomes, including visual acuity, motor function, and neurocognitive performance, as endpoints in clinical trials enrolling patients with pLGG. The panel discussed challenges and opportunities regarding the selection, implementation, and evaluation of clinical outcome assessments in these functional domains and outlined key considerations for their inclusion in future clinical trial design and role in new drug development.

Department

Neurology

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