Seeking a better landscape for therapy development in neuromuscular disorders

ABSTRACT Although the neuromuscular field has seen accelerated approval of a drug for Duchenne muscular dystrophy (DMD) and full approval of one for spinal muscular atrophy, these experiences have shown that objective data and an adequate level of effect are essential for drug approval and reimburse...

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Bibliographic Details
Published in:Muscle & nerve 2018-01, Vol.57 (1), p.16-19
Main Authors: Larkindale, Jane, Porter, John D.
Format: Article
Language:English
Subjects:
Atrophy
Biomarkers
clinical research
data sharing
Diseases
Drugs
Duchenne muscular dystrophy
Duchenne's muscular dystrophy
Dystrophy
Genetic Therapy
Humans
Information Dissemination
Muscles
Muscular dystrophy
Muscular Dystrophy, Duchenne - therapy
natural history
Neuromuscular diseases
Neuromuscular Diseases - therapy
neuromuscular disorders
Physical Therapy Modalities
Spinal muscular atrophy
therapy development
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Summary:ABSTRACT Although the neuromuscular field has seen accelerated approval of a drug for Duchenne muscular dystrophy (DMD) and full approval of one for spinal muscular atrophy, these experiences have shown that objective data and an adequate level of effect are essential for drug approval and reimbursement. The appropriateness and validity of biomarkers and clinically meaningful endpoints and an understanding of disease progression rates all played essential roles in the levels of evidence for these drugs. Such tools are best developed through integration of clinical data. The siloing of clinical data for rare neuromuscular diseases represents a considerable barrier to achieving better care and novel therapies for patients living with neuromuscular diseases. We discuss a data‐sharing model implemented for DMD and urge cultural changes in the ways natural history and clinical trial data are collected and shared across all neuromuscular diseases in order to benefit the primary stakeholder, the patient. Muscle Nerve 57: 16–19, 2018
ISSN:0148-639X
1097-4598
DOI:10.1002/mus.25961