The introduction of new medications in pediatric multiple sclerosis: Open issues and challenges

Publikation: Bidrag til tidsskriftTidsskriftartikelForskningfagfællebedømt

  • Angelo Ghezzi
  • Maria Pia Amato
  • Gilles Edan
  • Hans Peter Hartung
  • Eva Kubala Havrdová
  • Ludwig Kappos
  • Xavier Montalban
  • Carlo Pozzilli
  • Sørensen, Per Soelberg
  • Maria Trojano
  • Patrich Vermersch
  • Giancarlo Comi

Disease-modifying drugs (DMDs) for multiple sclerosis (MS) have been evaluated in pediatric patients in observational studies demonstrating a similar, even better clinical effect compared to adults, with a similar safety. Only fingolimod has been tested in a randomized controlled trial (RCT) and is approved for pediatric multiple sclerosis (ped-MS). Numerous methodological, practical, and ethical issues underline that RCTs are difficult to conduct in ped-MS. This also creates a lack of safety information. To facilitate the availability of new agents in ped-MS, we encourage to develop a different approach based on pharmacokinetic/pharmacodynamic studies to yield information on optimal doses and implementation of obligatory registries to obtain information on safety as primary endpoint.

OriginalsprogEngelsk
TidsskriftMultiple Sclerosis Journal
Vol/bind27
Udgave nummer3
Sider (fra-til)479-482
Antal sider4
ISSN1352-4585
DOI
StatusUdgivet - 2021

Bibliografisk note

Funding Information:
The author(s) declared the following potential conflicts of interest with respect to the research, authorship, and/or publication of this article: A.G. received honoraria for speaking and consultancy from Merck-Serono, Novartis, Biogen Idec, and Genzyme in the last 3 years. M.P.A. received personal compensation for serving on scientific advisory boards, steering committees, speaker honoraria, and research grants from Merck, Novartis, TEVA, Roche, and Sanofi Genzyme in the last 3 years. G.E. received honoraria for serving on steering and data monitoring committees, speaker’s fees, and research grants from Bayer Healthcare, Celgene Receptos, Merck, Novartis, Roche, Sanofi Genzyme, and Teva with approval by the director of the University Hospital. H.-P.H. received honoraria for serving on steering and data monitoring committees and speaker’s fees from Bayer Healthcare, Biogen, Celgene Receptos, GeNeuro, MedImmune, Merck, Novartis, Roche, Sanofi Genzyme, and TG Therapeutics with approval by the Rector of Heinrich-Heine-University. E.K.H. received honoraria/research support from Biogen, Merck-Serono, Novartis, Roche, and Teva; advisory boards for Actelion, Biogen, Celgene, Merck-Serono, Novartis, and Sanofi Genzyme; and supported by the Czech Ministry of Education research project PROGRES Q27/LF1. L.K.’s institution (University Hospital Basel) received research support in the last 3 years at the Department: steering committee, advisory board, and consultancy fees from Actelion, Alkermes, Almirall, Bayer, Biogen, Celgene/Receptos, df-mp, Excemed, GeNeuro SA, Genzyme, Japan Tobacco, Merck, Minoryx, Mitsubishi Pharma, Novartis, F. Hoffmann-La Roche Ltd, Sanofi-Aventis, Santhera, Teva, and Vianex and license fees for Neurostatus-UHB products; the Research of the MS Center in Basel has been supported by grants from Bayer, Biogen, Novartis, the Swiss MS Society, the Swiss National Research Foundation, Innoswiss, the European Union, and Roche Research Foundations. In the last year, the Research of the MS Center in Basel has been supported by grants and support (Actelion, Bayer, Baxalta, Biogen, Celgene/Receptos, CSL Behring, Desitin, Genzyme, Japan Tobacco, Merck, Novartis, Pfizer, Roche, Sanofi, Santhera, and Teva). X.M. received speaking honoraria and travel expenses for participation in scientific meetings, has been a steering committee member of clinical trials, or participated in advisory boards of clinical trials in the past years with Actelion, Alexion, Bayer, Biogen, Celgene, EMD Serono, Genzyme, Immunic, Medday, Merck, Nervgen, Novartis, Roche, Sanofi Genzyme, Teva Pharmaceutical, TG Therapeutics, Excemed, MSIF, and NMSS. X.M. received research support through the institution St. Michael’s Hospital from Abbvie, Biogen, Medday, Roche, and Sanofi and through the institution Multiple Sclerosis Centre of Catalonia (Cemcat) from Biogen, Merck, Novartis, Roche, Sanofi Genzyme, and Teva Pharmaceutical. C.P. has served on scientific advisory boards for Actelion, Biogen, Genzyme, Hoffmann-La Roche, Merck, Novartis, Sanofi, Teva, and Almirall and has received consulting and/or speaking fees, research support and travel grants from Almirall, Biogen, Genzyme, Hoffmann-La Roche Merck, Novartis, and Teva. P.S.S. received personal compensation for serving on scientific advisory boards, steering committees, independent data monitoring committees, or have received speaker honoraria for Merck, Novartis, TEVA, GlaxoSmithKline, and Celgene in the last 3 years. M.T. received personal compensation for serving on scientific advisory boards for Biogen, Novartis, Roche, and Genzyme; has received speaker honoraria from Biogen, Sanofi-Aventis, Merck, Teva, Genzyme, and Novartis; and has received research grants for her Institution from Biogen Idec, Merck-Serono, and Novartis. P.V. received honoraria and consulting fees from Biogen, Sanofi Genzyme, Novartis, Teva, Merck, Roche, and Celgene. P.V. received research supports from Novartis, Sanofi Genzyme, and Roche. G.C. received compensation for consulting and/or speaking activities from Novartis, Teva, Sanofi Genzyme, Genzyme, Merck, Merck-Serono, Celgene Group, Biogen Idec, Roche, Almirall SpA, Forward Pharma, Medday, and Excemed in the past 2 years.

Publisher Copyright:
© The Author(s), 2020.

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