Ettienne EB, Russo E, Striano P, Grant-Kels JM, Rose K. Did pediatric drug development advance epilepsy treatment in young patients? It is time for new research goals. World J Methodol 2024; 14(2): 92371 [PMID: 38983658 DOI: 10.5662/wjm.v14.i2.92371]
Corresponding Author of This Article
Klaus Rose, MD, MS, CEO, klausrose Consulting, Pediatric Drug Development and more, Medical Science, Äussere Baselstrasse 308, CH-4125 Riehen, Switzerland. klaus.rose@klausrose.net
Research Domain of This Article
Medicine, Research & Experimental
Article-Type of This Article
Minireviews
Open-Access Policy of This Article
This article is an open-access article which was selected by an in-house editor and fully peer-reviewed by external reviewers. It is distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/
Jane M Grant-Kels, Department of Dermatology, University of Connecticut Health Center, Farmington, CT 06032, United States
Klaus Rose, klausrose Consulting, Pediatric Drug Development and more, Medical Science, CH-4125 Riehen, Switzerland
Author contributions: The idea for this manuscript emerged in correspondence and discussion of all involved authors. Rose K circulated a first draft which was critically reviewed and amended by all other authors; Grant-Kels JM, Ettienne EB, Russo E, and Striano P edited the English language; Rose K wrote the final manuscript based on feedback from all other authors; Grant-Kels JM did a final English language polishing. The final manuscript was circulated and approved by all co-authors.
Conflict-of-interest statement: The authors declare no conflict of interest.
Open-Access: This article is an open-access article that was selected by an in-house editor and fully peer-reviewed by external reviewers. It is distributed in accordance with the Creative Commons Attribution NonCommercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: https://creativecommons.org/Licenses/by-nc/4.0/
Corresponding author: Klaus Rose, MD, MS, CEO, klausrose Consulting, Pediatric Drug Development and more, Medical Science, Äussere Baselstrasse 308, CH-4125 Riehen, Switzerland. klaus.rose@klausrose.net
Received: January 23, 2024 Revised: February 13, 2024 Accepted: April 16, 2024 Published online: June 20, 2024 Processing time: 142 Days and 18.2 Hours
Abstract
Modern drugs have changed epilepsy, which affects people of all ages. However, for young people with epilepsy, the framework of drug development has stalled. In the wake of the thalidomide catastrophe, the misconception emerged that for people < 18 years of age drugs, including antiseizure medications (ASMs), need separate proof of efficacy and safety, overall called "pediatric drug development". For ASMs, this has changed to some degree. Authorities now accept that ASMs are effective in < 18 years as well, but they still require "extrapolation of efficacy," as if minors were another species. As a result, some of the pediatric clinical epilepsy research over the past decades was unnecessary. Even more importantly, this has hampered research on meaningful research goals. We do not need to confirm that ASMs work before as they do after the 18th birthday. Instead, we need to learn how to prevent brain damage in young patients by preventing seizures and optimize ASMs’ uses. Herein we discuss how to proceed in this endeavor.
Core Tip: For young people with epilepsy, the framework of drug development has stalled. The misconception emerged that for people < 18 years drugs, including antiseizure medications (ASMs), need separate proof of efficacy and safety, overall called "pediatric drug development". For ASMs, the authorities require "extrapolation of efficacy", as if minors were another species. Relevant parts of pediatric epilepsy research were pointless, and research on meaningful goals was hampered. ASMs work also before the 18th birthday. We should learn to prevent brain damage in young patients by preventing seizures and by optimize ASMs’ use.
