Cystic fibrosis drug trial design in the era of CFTR modulators associated with substantial clinical benefit: stakeholders' consensus view
Language English Country Netherlands Media print-electronic
Document type Journal Article, Research Support, Non-U.S. Gov't
Grant support
Department of Health - United Kingdom
PubMed
32527602
DOI
10.1016/j.jcf.2020.05.012
PII: S1569-1993(20)30162-4
Knihovny.cz E-resources
- Keywords
- CFTR modulators, Clinical trials, Cystic fibrosis,
- MeSH
- Cystic Fibrosis drug therapy genetics MeSH
- Clinical Trials as Topic organization & administration MeSH
- Consensus MeSH
- Humans MeSH
- Cystic Fibrosis Transmembrane Conductance Regulator drug effects MeSH
- Drug Development organization & administration MeSH
- Research Design MeSH
- Check Tag
- Humans MeSH
- Publication type
- Journal Article MeSH
- Research Support, Non-U.S. Gov't MeSH
- Names of Substances
- Cystic Fibrosis Transmembrane Conductance Regulator MeSH
CFTR modulators associated with substantial clinical benefit are expected to rapidly improve the baseline condition of people with cystic fibrosis (PWCF) as well as decrease the rate of lung function decline, the occurrence of pulmonary exacerbations and likely even other disease complications. These changes in clinical status of PWCF introduced by clinically effective modulator therapy will have major repercussions on modalities of future CF drug development. As part of its 'Strategic Plan to speed up Access to new Drugs', the European Cystic Fibrosis Society (ECFS) convened a meeting in Brussels on November 27th 2019 with relevant stakeholders (CF researchers and clinicians, patient organization and pharmaceutical company representatives, regulators, health technology assessors; see Acknowledgments for list of attendees) to discuss the future of clinical trials in cystic fibrosis (CF) in the context of HEMT entering the clinical arena. The following is the conclusion of the presentations and discussions. It is hoped that these concepts will be considered in future regulatory guidelines and may provide rationale and support for alternative trial designs.
Centre for Experimental Medicine Queen's University Belfast Northern Ireland United Kingdom
Department of Paediatrics and CF Center Hadassah Hebrew University Hospital Jerusalem Israel
Department of Paediatrics University Hospital of Leuven University of Leuven Leuven Belgium
Imperial College London London UK Royal Brompton and Harefield NHS Foundation Trust London UK
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