Development, Implementation, and Use of a Neurology Therapeutics Committee

Child Neurology Open
Edward B ClarkJoshua L Bonkowsky

Abstract

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.

References

Apr 30, 2013·Handbook of Clinical Neurology·Patrick Aubourg
Nov 18, 2015·Annals of Neurology·Jerry R MendellUNKNOWN Eteplirsen Study Group and Telethon Foundation DMD Italian Network
Apr 28, 2017·Orphanet Journal of Rare Diseases·S RyderJ Kleijnen
Aug 3, 2017·Pediatrics·Eyal CohenJay G Berry
Oct 5, 2017·The New England Journal of Medicine·Florian EichlerDavid A Williams
Nov 2, 2017·The New England Journal of Medicine·Jerry R MendellBrian K Kaspar
Nov 2, 2017·The New England Journal of Medicine·Richard S FinkelUNKNOWN ENDEAR Study Group
Dec 22, 2017·Expert Opinion on Emerging Drugs·Kristin M Hudock, John Paul Clancy
Apr 25, 2018·The New England Journal of Medicine·Angela SchulzUNKNOWN CLN2 Study Group

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Methods Mentioned

BETA
antisense oligonucleotide

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