The introduction of new medications in pediatric multiple sclerosis: Open issues and challenges
Research output: Contribution to journal › Journal article › Research › peer-review
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.
Original language | English |
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Journal | Multiple Sclerosis Journal |
Volume | 27 |
Issue number | 3 |
Pages (from-to) | 479-482 |
Number of pages | 4 |
ISSN | 1352-4585 |
DOIs | |
Publication status | Published - 2021 |
Bibliographical note
Publisher Copyright:
© The Author(s), 2020.
- clinical trials, disease-modifying drugs, observational studies, Pediatric multiple sclerosis, randomized controlled trials
Research areas
ID: 303772425