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Children with multiple sclerosis should not become therapeutic hostages
1Klausrose Consulting, Pediatric Drug Development and More, Aeussere Baselstrasse 308, 4125 Riehen, Switzerland.
Therapeutic Advances in Neurological Disorders
|September 2, 2016
Summary
Regulatory agencies like the FDA and EMA require pediatric clinical trials, but for rare conditions like pediatric multiple sclerosis (pMS), recruitment challenges may render studies unethical and clinically irrelevant.
Area of Science:
- Pediatric Neurology
- Regulatory Science
- Clinical Trial Design
Background:
- Regulatory agencies in the US (FDA) and EU (EMA) mandate pediatric clinical trials for new drug approvals.
- Pediatric multiple sclerosis (pMS) serves as a model to evaluate the feasibility and clinical value of such trials.
Purpose of the Study:
- To assess the clinical utility and recruitment potential of pediatric multiple sclerosis (pMS) studies.
- To discuss US and EU pediatric legislation using pMS as a case study.
Main Methods:
- Analysis of pMS clinical trials mandated by the FDA and EMA.
- Review of industry-sponsored pMS studies registered on clinicaltrials.gov and clinicaltrialsregister.eu.
Main Results:
- The FDA requires four pMS trials, while the EMA mandates fifteen.
- pMS is a rare disease, and neither agency prioritizes drugs for its specific treatment.
Conclusions:
- Multiple EMA-requested pMS studies face significant recruitment challenges, potentially yielding irrelevant outcomes for evidence-based medicine.
- Such trials may be considered unethical due to low probability of meaningful results, leading to off-label drug use in children.
- International collaboration is suggested for prioritizing and testing new multiple sclerosis compounds in pediatric populations.
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