Evidence map›Paper›PMID 30800699›Full record

ArticleChild neurology open2019

Development, Implementation, and Use of a Neurology Therapeutics Committee.

Edward B Clark, Russell J Butterfield, Francis M Filloux, Joshua L Bonkowsky

Erratum issuedAbstract read
In one paragraph

Article in Child neurology open, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

4 authors.

Edward B ClarkUniversity of Utah Medical Group, University of Utah School of Medicine, Salt Lake, UT, USA.
Russell J ButterfieldDivision of Pediatric Neurology, University of Utah School of Medicine, Salt Lake City, UT, USA.
Francis M FillouxDivision of Pediatric Neurology, University of Utah School of Medicine, Salt Lake City, UT, USA.
Joshua L BonkowskyDivision of Pediatric Neurology, University of Utah School of Medicine, Salt Lake City, UT, USA.

Funding

Utah Center for Clinical and Translational ScienceUL1TR002538 · NCATS · UNIVERSITY OF UTAH · PI HESS, RACHEL, MAJERSIK, JENNIFER JUHL · 2018 to 2022
$26.0M
Genetic Control of Phenotypic Variability in Collagen VI Related Muscular DystrophiesK08NS097631 · NINDS · UNIVERSITY OF UTAH · PI BUTTERFIELD, RUSSELL JAMES · 2016 to 2020
$1.0M
NCATS NIH HHS UL1 TR002538NINDS NIH HHS K08 NS097631
6 · The paper itself

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.

Indexed as

gene therapynusinersenpediatric neurologyspinal muscular atrophytherapeutics

Identifiers

PMID30800699
PMCPMC6378459

What OpenQuestion holds

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Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.