Dr. Johnson of the University of Utah received consulting fees from AMO Pharma, University of Utah, and AveXis and a research grant from Biogen Idec.)
Ms. Stock of the American Academy of Neurology has no relevant financial relationships to disclose.)
Ms. Kaloides of the American Academy of Neurology has no relevant financial relationships to disclose.)
Dr. Ciafaloni of the University of Rochester received personal compensation for serving on advisory boards and/or as a consultant for Avexis, Biogen, Pfizer, PTC Therapeutics, Sarepta, Ra pharma, Wave, and Strongbridge Biopharma; and for serving on a speaker’s bureau for Biogen. Dr Ciafaloni also received research and/or grant support from Orphazyme, PTC Therapeutics, Santhera, and Sarepta.)
This article includes discussion of the high cost of new treatments for neuromuscular diseases and the Orphan Drug Act.
Neuromuscular diseases are on the forefront of therapeutic development. Three new disease-modifying therapies have been approved in the past 12 months for rare neuromuscular disorders, an unprecedented pace for any area of neurology. The favorable regulatory landscape for orphan disease has provided the impetus to invest in these disorders. However, the cost and intensity of these therapies have provided new challenges to physicians treating these debilitating illnesses. In this article, the authors describe the challenges and identify some solutions moving forward.
• Incentives within the Orphan Drug Act have promoted research in rare neuromuscular diseases.
• New disease-modifying therapies in neuromuscular disease are difficult to implement given the cost and route of administration.
• Initial implementation in multidisciplinary clinics may be the most feasible approach.
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