Evidence map›Paper›PMID 30414722›Full record

ReviewMolecular therapy : the journal of the American Society of Gene Therapy2018

Addressing the Value of Gene Therapy and Enhancing Patient Access to Transformative Treatments.

Rachel Salzman, Francesca Cook, Timothy Hunt, Harry L Malech, Philip Reilly, Betsy Foss-Campbell, David Barrett

Abstract readReview
PubMed Full text at PMC Publisher
In one paragraph

Review in Molecular therapy : the journal of the American Society of Gene Therapy, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 60 papers, 3 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
60citing papers in PubMed, 3 pooled it
–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

60 citing papers in PubMed, 3 syntheses or guidelines pooled it.

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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

7 authors.

Rachel SalzmanALD Connect, Middleton, MA, USA; American Society of Gene and Cell Therapy, Value Initiative Workgroup, Milwaukee, WI, USA.
Francesca CookREGENXBIO Inc., Rockville, MD, USA; American Society of Gene and Cell Therapy, Value Initiative Workgroup, Milwaukee, WI, USA.
Timothy HuntEditas Medicine, Inc., Cambridge, MA, USA; American Society of Gene and Cell Therapy, Value Initiative Workgroup, Milwaukee, WI, USA.
Harry L MalechLaboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, NIH, Bethesda, MD, USA; American Society of Gene and Cell Therapy, Value Initiative Workgroup, Milwaukee, WI, USA.
Philip ReillyThird Rock Ventures, Boston, MA, USA; American Society of Gene and Cell Therapy, Value Initiative Workgroup, Milwaukee, WI, USA.
Betsy Foss-CampbellAmerican Society of Gene and Cell Therapy, Milwaukee, WI, USA.
David BarrettAmerican Society of Gene and Cell Therapy, Milwaukee, WI, USA. Electronic address: dbarrett@asgct.org.

Funding

Gene Therapy Basic Research to Treat Inherited Primary Immune DeficienciesZIAAI000644 · NIAID · NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES · PI MALECH, HARRY L · 2009 to 2025
$12.1M
Basic Research on Hematopoietic Human Stem CellsZIAAI000988 · NIAID · NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES · PI MALECH, HARRY L · 2009 to 2019
$6.5M
6 · The paper itself

Abstract

Although high upfront costs for the high value of gene therapy have resulted in concerns about sufficient reimbursement to allow patient access to these therapies, the significant benefits of gene therapies will not be realized unless patients have access to them. Stakeholders are discussing these issues, and the payment models being developed for the newly approved gene therapies provide an early indication of the flexibility that will be needed from treatment manufacturers, payers, and policy makers to optimize patient access. Maximizing patient access to effective gene therapies is one integral part of the overall mission of the American Society of Gene and Cell Therapy, along with maximizing the quality of therapies and minimizing their costs.

Indexed as

Genetic TherapyHealth Services AccessibilityQuality ImprovementAnimalsCost-Benefit AnalysisCost of IllnessHealth Care CostsHumansOutcome Assessment, Health CareReimbursement Mechanismsbenefitscostcoveragepatient accesspayerpaymentpayorpositionreimbursementvalue

Identifiers

PMID30414722
PMCPMC6277509

What OpenQuestion holds

Textmetadata
Read underepoch 390

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.