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

Author:

Fangusaro Jason1ORCID,Avery Robert A.2ORCID,Fisher Michael J.2ORCID,Packer Roger J.3ORCID,Walsh Karin S.3ORCID,Schouten-van Meeteren Antoinette4ORCID,Karres Dominik5ORCID,Bradford Diana6ORCID,Bhatnagar Vishal6ORCID,Singh Harpreet6ORCID,Kluetz Paul G.6ORCID,Donoghue Martha6ORCID,Duke Elizabeth S.6ORCID

Affiliation:

1. 1Children's Hospital of Atlanta, Emory University and the Aflac Cancer Center, Atlanta, Georgia.

2. 2The Children's Hospital of Philadelphia and the University of Pennsylvania Perelman School of Medicine, Philadelphia, Pennsylvania.

3. 3Center for Neuroscience and Behavioral Medicine and Brain Tumor Institute, Children's National Hospital, and The George Washington University School of Medicine, Washington, DC.

4. 4Princess Máxima Center for Pediatric Oncology, Utrecht, the Netherlands.

5. 5Pediatric Medicines Office, Scientific Evidence Generation Department, Human Medicines Division, European Medicines Agency (EMA), Amsterdam, the Netherlands.

6. 6Center for Drug Evaluation and Research, Office of New Drugs, U.S. Food and Drug Administration, Silver Spring, Maryland.

Abstract

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.

Funder

U.S. Food and Drug Administration

Publisher

American Association for Cancer Research (AACR)

Cited by 1 articles. 订阅此论文施引文献 订阅此论文施引文献,注册后可以免费订阅5篇论文的施引文献,订阅后可以查看论文全部施引文献

同舟云学术

1.学者识别学者识别

2.学术分析学术分析

3.人才评估人才评估

"同舟云学术"是以全球学者为主线,采集、加工和组织学术论文而形成的新型学术文献查询和分析系统,可以对全球学者进行文献检索和人才价值评估。用户可以通过关注某些学科领域的顶尖人物而持续追踪该领域的学科进展和研究前沿。经过近期的数据扩容,当前同舟云学术共收录了国内外主流学术期刊6万余种,收集的期刊论文及会议论文总量共计约1.5亿篇,并以每天添加12000余篇中外论文的速度递增。我们也可以为用户提供个性化、定制化的学者数据。欢迎来电咨询!咨询电话:010-8811{复制后删除}0370

www.globalauthorid.com

TOP

Copyright © 2019-2024 北京同舟云网络信息技术有限公司
京公网安备11010802033243号  京ICP备18003416号-3