Mayer-Hamblett, N., Van Koningsbruggen-Rietschel, S., Nichols, D. P., VanDevanter, D. R., Davies, J. C., Lee, T., Durmowicz, A. G., Ratjen, F., Konstan, M. W., Pearson, K., Bell, S. C., Clancy, J. P., Taylor-Cousar, J. L., De Boeck, K., Donaldson, S. H., Downey, D. G., Flume, P. A., Drevinek, P., Goss, C. H., Fajac, I, Magaret, A. S., Quon, B. S., Singleton, S. M., VanDalfsen, J. M. and Retsch-Bogart, G. Z. (2020). Building global development strategies for cf therapeutics during a transitional cftr modulator era. J. Cyst. Fibros, 19 (5). S. 677 - 688. AMSTERDAM: ELSEVIER. ISSN 1873-5010

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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. (C) 2020 European Cystic Fibrosis Society. Published by Elsevier B.V. All rights reserved.

Item Type: Journal Article
Creators:
CreatorsEmailORCIDORCID Put Code
Mayer-Hamblett, N.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Van Koningsbruggen-Rietschel, S.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Nichols, D. P.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
VanDevanter, D. R.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Davies, J. C.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Lee, T.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Durmowicz, A. G.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Ratjen, F.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Konstan, M. W.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Pearson, K.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Bell, S. C.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Clancy, J. P.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Taylor-Cousar, J. L.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
De Boeck, K.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Donaldson, S. H.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Downey, D. G.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Flume, P. A.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Drevinek, P.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Goss, C. H.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Fajac, IUNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Magaret, A. S.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Quon, B. S.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Singleton, S. M.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
VanDalfsen, J. M.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
Retsch-Bogart, G. Z.UNSPECIFIEDUNSPECIFIEDUNSPECIFIED
URN: urn:nbn:de:hbz:38-320461
DOI: 10.1016/j.jcf.2020.05.011
Journal or Publication Title: J. Cyst. Fibros
Volume: 19
Number: 5
Page Range: S. 677 - 688
Date: 2020
Publisher: ELSEVIER
Place of Publication: AMSTERDAM
ISSN: 1873-5010
Language: English
Faculty: Unspecified
Divisions: Unspecified
Subjects: no entry
Uncontrolled Keywords:
KeywordsLanguage
LUNG CLEARANCE INDEX; HIGH-DOSE IBUPROFEN; CYSTIC-FIBROSIS; PSEUDOMONAS-AERUGINOSA; SPUTUM BIOMARKERS; DRUG-DEVELOPMENT; CLINICAL-TRIALS; IVACAFTOR; INFLAMMATION; CHILDRENMultiple languages
Respiratory SystemMultiple languages
URI: http://kups.ub.uni-koeln.de/id/eprint/32046

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