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. 2019 Feb 14;6:2329048X19830473.
doi: 10.1177/2329048X19830473. eCollection 2019.

Development, Implementation, and Use of a Neurology Therapeutics Committee

Free PMC article

Development, Implementation, and Use of a Neurology Therapeutics Committee

Edward B Clark et al. Child Neurol Open. .
Free PMC article

Erratum in


Innovative therapeutics are transforming care of children with previously untreatable neurological disorders. However, there are challenges in the use of new therapies: the medicine may not be effective in all patients, administration may not be tolerated, and matching therapy choice to patient is complex. Finally, costs are high, which imposes financial burdens on insurance companies, families, and the health-care system. Our objective was to address challenges for clinical implementation of the new therapeutics. We sought to develop a process that would be personalized for patient and disease, encourage appropriate use of a therapeutic agent while mitigating pressure on a clinician to prescribe the therapy in all instances, and assist third-party payers in approving therapeutic use based on safety and efficacy. We report our creation of a Neurology Therapeutics Committee for pediatric patients. We review the committee's mechanisms, describe its use and report outcomes, and suggest the Neurology Therapeutics Committee's broader applicability.

Keywords: gene therapy; nusinersen; pediatric neurology; spinal muscular atrophy; therapeutics.

Conflict of interest statement

Declaration of Conflicting Interests: The authors declared no potential conflicts of interest with respect to the research, authorship, and/or publication of this article.


Figure 1.
Figure 1.
Letter of findings from the Utah Neurology Therapeutics Committee.

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