Evidence map›Paper›PMID 32522463›Full record

ReviewJournal of cystic fibrosis : official journal of the European Cystic Fibrosis Society2020

Building global development strategies for cf therapeutics during a transitional cftr modulator era.

N Mayer-Hamblett, S van Koningsbruggen-Rietschel, D P Nichols, D R VanDevanter, J C Davies, T Lee, A G Durmowicz, F Ratjen, M W Konstan, K Pearson and 15 more

Open access · greenAbstract readReview
In one paragraph

Review in Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers.

0numbers the graph read from it
0cells of the map it votes in
17citing papers in PubMed
3.0field-weighted citation impact, top 7% of its field
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

17 citing papers in PubMed, 31 citations in OpenAlex.

  1. Trial
  2. Trial
  3. Review
  4. Article
  5. Article
  6. Article
  7. Review
  8. Review
  9. Article
  10. Review
  11. Article
  12. A new path for CF clinical trials through the use of historical controls.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2022
    Article
  13. Cystic Fibrosis: A Disease in Transformation, yet More Work to Be Done!American journal of respiratory and critical care medicine · 2022
    Article
  14. Observational
  15. Review
  16. DNA Methylation atGenes · 2021
    Article
  17. New therapies for people with CF in the CFTR modulator world.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2020
    Article
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

25 authors at 16 institutions in 8 countries.

N Mayer-HamblettUniversity of Washington, Seattle, WA; Seattle Children's Hospital, Seattle, WA. Electronic address: nicole.hamblett@seattlechildrens.org.
S van Koningsbruggen-RietschelCystic Fibrosis Center, Children's Hospital, University of Cologne; Faculty of Medicine and University Hospital Cologne, Cologne Germany.
D P NicholsUniversity of Washington, Seattle, WA; Seattle Children's Hospital, Seattle, WA.
D R VanDevanterCase Western Reserve University School of Medicine, Cleveland, OH.
J C DaviesNational Heart & Lung Institute, Imperial College London, London, UK; Royal Brompton & Harefield NHS Foundation Trust, London, UK.
T LeeLeeds Regional Paediatric Cystic Fibrosis Centre, Leeds, UK.
A G DurmowiczCystic Fibrosis Foundation, Bethesda, MD.
F RatjenUniversity of Toronto, Toronto, Canada.
M W KonstanCase Western Reserve University School of Medicine, Cleveland, OH; Rainbow Babies and Children's Hospital, Cleveland, OH.
K PearsonSeattle Children's Hospital, Seattle, WA.
S C BellChildren's Health Research Centre, The University of Queensland, Brisbane, Australia.
J P ClancyCystic Fibrosis Foundation, Bethesda, MD.
J L Taylor-CousarNational Jewish Health, Denver, CO.
K De BoeckUniversity of Leuven, Leuven, Belgium.
S H DonaldsonUniversity of North Carolina at Chapel Hill, Chapel Hill, NC.
D G DowneyCentre for Experimental Medicine, Queen's University Belfast, Belfast, Northern Ireland.
P A FlumeMedical University of South Carolina, Charleston, SC.
P DrevinekCharles University, Prague, Czechia, Motol University Hospital, Prague, Czechia.
C H GossUniversity of Washington, Seattle, WA; Seattle Children's Hospital, Seattle, WA.
I FajacUniversité de Paris, Paris, France.
A S MagaretUniversity of Washington, Seattle, WA; Seattle Children's Hospital, Seattle, WA.
B S QuonUniversity of British Columbia, Vancouver, British Columbia.
S M SingletonCystic Fibrosis Foundation, Bethesda, MD.
J M VanDalfsenSeattle Children's Hospital, Seattle, WA.
G Z Retsch-BogartUniversity of North Carolina at Chapel Hill, Chapel Hill, NC.
Seattle Children's Hospital · USCystic Fibrosis Foundation · USUniversity of North Carolina at Chapel Hill · USCharles University · CZChildren's Medical Research Institute · AUCystic Fibrosis Trust · GBKU Leuven · BEMedical University of South Carolina · USNational Jewish Health · USRainbow Babies & Children's Hospital · USRoyal Brompton & Harefield NHS Foundation Trust · GBUniversité Paris Cité · FRUniversity of British Columbia · CAUniversity of Cologne · DEUniversity of Toronto · CAUniversity School · US

Funding

Transform Dissemination and Implementation Science in CTSA ProgramsUL1TR002319 · NCATS · UNIVERSITY OF WASHINGTON · PI John K. Amory · 2017 to 2026
$100.0M
Re-Entry Supplement: Investigation of Oral Microbial Enzymes for the Detection and Treatment of Periodontal DiseaseUL1TR002489 · NCATS · UNIV OF NORTH CAROLINA CHAPEL HILL · PI BUSE, JOHN BERNARD, SHAHEEN, NICHOLAS J · 2018 to 2022
$48.6M
Clinical and Translational Science Collaborative of ClevelandUL1TR002548 · NCATS · CASE WESTERN RESERVE UNIVERSITY · PI MCCOMSEY, GRACE A · 2018 to 2022
$35.5M
Translational Research Center to Expedite Novel Therapies in Cystic FibrosisP30DK089507 · NIDDK · SEATTLE CHILDREN'S HOSPITAL · PI Christopher Hooper Goss · 2010 to 2026
$21.4M
Project 3 - Personalized cystic fibrosis treatment strategies with synergistic therapies Identified in vitro.P01HL128192 · NHLBI · INDIANA UNIVERSITY INDIANAPOLIS · PI GASTON, BENJAMIN · 2016 to 2020
$12.2M
South Carolina Clinical & Translational Research Institute (SCTR)UL1TR000062 · NCATS · MEDICAL UNIVERSITY OF SOUTH CAROLINA · PI BRADY, KATHLEEN T. · 2012 to 2014
$9.4M
Department of HealthNCATS NIH HHS UL1 TR000062NCATS NIH HHS UL1 TR002319NCATS NIH HHS UL1 TR002489NCATS NIH HHS UL1 TR002548NHLBI NIH HHS P01 HL128192NIDDK NIH HHS P30 DK089507
6 · The paper itself

Abstract

As CFTR modulator therapy transforms the landscape of cystic fibrosis (CF) care, its lack of uniform access across the globe combined with the shift towards a new standard of care creates unique challenges for the development of future CF therapies. The advancement of a full and promising CF therapeutics pipeline remains a necessary priority to ensure maximal clinical benefits for all people with CF. It is through collaboration across the global CF community that we can optimize the evaluation and approval process of new therapies. To this end, we must identify areas for which harmonization is lacking and for which efficiencies can be gained to promote ethical, feasible, and credible study designs amidst the changing CF care landscape. This article summarizes the counsel from core advisors across multiple international regions and clinical trial networks, developed during a one-day workshop in October 2019. The goal of the workshop was to identify, in consideration of the highly transitional era of CFTR modulator availability, the drug development areas for which global alignment is currently uncertain, and paths forward that will enable advancement of CF therapeutic development.

Indexed as

International CooperationCystic FibrosisCystic Fibrosis Transmembrane Conductance RegulatorDrug DevelopmentHumansCFTR protein, humanCystic Fibrosis Transmembrane Conductance RegulatorCFTR modulatorsClinical trialsDrug developmentGlobal perspective

Identifiers

PMID32522463
PMCPMC7492419
OpenAlexW3033287571

What OpenQuestion holds

Textmetadata
LicenceTDM
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.