Universität zu Köln

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:	Creators Email ORCID ORCID Put Code Mayer-Hamblett, N. UNSPECIFIED UNSPECIFIED UNSPECIFIED Van Koningsbruggen-Rietschel, S. UNSPECIFIED UNSPECIFIED UNSPECIFIED Nichols, D. P. UNSPECIFIED UNSPECIFIED UNSPECIFIED VanDevanter, D. R. UNSPECIFIED UNSPECIFIED UNSPECIFIED Davies, J. C. UNSPECIFIED UNSPECIFIED UNSPECIFIED Lee, T. UNSPECIFIED UNSPECIFIED UNSPECIFIED Durmowicz, A. G. UNSPECIFIED UNSPECIFIED UNSPECIFIED Ratjen, F. UNSPECIFIED UNSPECIFIED UNSPECIFIED Konstan, M. W. UNSPECIFIED UNSPECIFIED UNSPECIFIED Pearson, K. UNSPECIFIED UNSPECIFIED UNSPECIFIED Bell, S. C. UNSPECIFIED UNSPECIFIED UNSPECIFIED Clancy, J. P. UNSPECIFIED UNSPECIFIED UNSPECIFIED Taylor-Cousar, J. L. UNSPECIFIED UNSPECIFIED UNSPECIFIED De Boeck, K. UNSPECIFIED UNSPECIFIED UNSPECIFIED Donaldson, S. H. UNSPECIFIED UNSPECIFIED UNSPECIFIED Downey, D. G. UNSPECIFIED UNSPECIFIED UNSPECIFIED Flume, P. A. UNSPECIFIED UNSPECIFIED UNSPECIFIED Drevinek, P. UNSPECIFIED UNSPECIFIED UNSPECIFIED Goss, C. H. UNSPECIFIED UNSPECIFIED UNSPECIFIED Fajac, I UNSPECIFIED UNSPECIFIED UNSPECIFIED Magaret, A. S. UNSPECIFIED UNSPECIFIED UNSPECIFIED Quon, B. S. UNSPECIFIED UNSPECIFIED UNSPECIFIED Singleton, S. M. UNSPECIFIED UNSPECIFIED UNSPECIFIED VanDalfsen, J. M. UNSPECIFIED UNSPECIFIED UNSPECIFIED Retsch-Bogart, G. Z. UNSPECIFIED UNSPECIFIED UNSPECIFIED
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:	Keywords Language LUNG CLEARANCE INDEX; HIGH-DOSE IBUPROFEN; CYSTIC-FIBROSIS; PSEUDOMONAS-AERUGINOSA; SPUTUM BIOMARKERS; DRUG-DEVELOPMENT; CLINICAL-TRIALS; IVACAFTOR; INFLAMMATION; CHILDREN Multiple languages Respiratory System Multiple languages
URI:	http://kups.ub.uni-koeln.de/id/eprint/32046