医学
临床试验
胶质瘤
肿瘤科
重症监护医学
内科学
癌症研究
作者
Jason Fangusaro,Robert A. Avery,Michael J. Fisher,Roger J. Packer,Karin S. Walsh,Antoinette Y. N. Schouten–van Meeteren,Dominik Karres,Diana Bradford,V. Bhatnagar,Harpreet Singh,Paul G. Kluetz,Martha Donoghue,Elizabeth S. Duke
标识
DOI:10.1158/1078-0432.ccr-23-3386
摘要
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.
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