Version 1
: Received: 31 July 2023 / Approved: 31 July 2023 / Online: 1 August 2023 (02:49:45 CEST)
How to cite:
Ettienne, E.B.; Russo, E.; Striano, P.; Grant-Kels, J.; Rose, K. Pediatric Epilepsy: Time For New Research Goals. Preprints2023, 2023072136. https://doi.org/10.20944/preprints202307.2136.v1
Ettienne, E.B.; Russo, E.; Striano, P.; Grant-Kels, J.; Rose, K. Pediatric Epilepsy: Time For New Research Goals. Preprints 2023, 2023072136. https://doi.org/10.20944/preprints202307.2136.v1
Ettienne, E.B.; Russo, E.; Striano, P.; Grant-Kels, J.; Rose, K. Pediatric Epilepsy: Time For New Research Goals. Preprints2023, 2023072136. https://doi.org/10.20944/preprints202307.2136.v1
APA Style
Ettienne, E.B., Russo, E., Striano, P., Grant-Kels, J., & Rose, K. (2023). Pediatric Epilepsy: Time For New Research Goals. Preprints. https://doi.org/10.20944/preprints202307.2136.v1
Chicago/Turabian Style
Ettienne, E.B., Jane Grant-Kels and Klaus Rose. 2023 "Pediatric Epilepsy: Time For New Research Goals" Preprints. https://doi.org/10.20944/preprints202307.2136.v1
Abstract
Modern drugs have changed epilepsy, which affects people of all ages. But 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 <18y antiseizure medications (ASMs) need separate proof of efficacy and safety (E&S), overall called “pediatric drug development.” This has been corrected to some extent. Authorities accept that ASMs are effective in <18y as well, but they still require "extrapolation of efficacy," as if minors were another species. In our view, the real problem is less that relevant parts of pediatric clinical epilepsy research over the past decades were useless, but that this has hampered research on meaningful challenges. We do not need to show that ASMs work also before the 18th birthday. But we need to learn how to best use ASMs to prevent brain damage in young patients and optimize ASMs use considering a broader range of aspects
Keywords
Epilepsy; Pediatric Drug Development (PDD); Therapeutic Orphans; Extrapolation of efficacy from adults to children; Antiseizure Medications (ASMs); Pediatric Investigation Plan (PIP)
Subject
Public Health and Healthcare, Public Health and Health Services
Copyright:
This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.
