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The best and worst of times in therapy development for myasthenia gravis
Michael Benatar1, Gary Cutter2, Henry J Kaminski3
1Department of Neurology, University of Miami, Miami, Florida.
Abstract:
Within the last 5 years, the US Food and Drug Administration (FDA) has approved complement and neonatal Fc receptor (FcRN) inhibitors for treatment of generalized myasthenia gravis, and several other therapies are in late-stage clinical trials or under regulatory review. However, questions about which patients are most likely to benefit from which therapies, and the relative effectiveness of these very expensive drugs, has resulted in uncertainty around the place that they should occupy in the existing therapeutic armamentarium. MGNet (a Rare Diseases Clinical Research Consortium funded by the National Institute of Neurological Diseases and Stroke) held two meetings during the 14th International Conference of the Myasthenia Gravis Foundation of America to discuss the most critical needs for clinical trial readiness and biomarker development in the context of therapy development for myasthenia gravis. Herein we provide a summary of these discussions, but not a consensus opinion, and offer a series of recommendations to guide focused research in the most critical areas. We welcome ongoing discussion through comments on this work.
Insights
New FDA-approved therapies for generalized myasthenia gravis (gMG) offer hope, but optimal patient selection and cost-effectiveness remain unclear. Discussions focused on critical needs for clinical trial readiness and biomarker development to guide future gMG treatment strategies.
Area of Science:
- Neurology
- Immunology
- Pharmacology
Background:
- Recent US Food and Drug Administration (FDA) approvals of complement and neonatal Fc receptor (FcRN) inhibitors for generalized myasthenia gravis (gMG).
- Several other novel therapies for gMG are in late-stage clinical trials or under regulatory review.
- Uncertainty exists regarding patient selection, relative effectiveness, and optimal positioning of these expensive therapies within the current treatment landscape.
Purpose of the Study:
- To summarize discussions from MGNet-convened meetings on critical needs for clinical trial readiness and biomarker development in gMG therapy advancement.
- To provide recommendations for focused research to address current uncertainties in gMG treatment.
Main Methods:
- Convened two meetings during the 14th International Conference of the Myasthenia Gravis Foundation of America.
- Facilitated discussions among experts on clinical trial readiness and biomarker development for gMG therapies.
- Synthesized discussions to identify critical research needs and formulate recommendations.
Main Results:
- Identified key challenges in optimizing the use of newly approved and emerging gMG therapies.
- Highlighted the need for robust clinical trial designs and validated biomarkers for patient stratification.
- Emphasized the importance of comparative effectiveness research for expensive gMG treatments.
Conclusions:
- Focused research on clinical trial readiness and biomarker development is crucial for advancing gMG therapy.
- Recommendations are provided to guide future research efforts in this area.
- Ongoing discussion and collaboration are encouraged to refine gMG treatment strategies.
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