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, S. C. Bell, J. P. Clancy, J. L. Taylor-Cousar, K. De Boeck, S. H. Donaldson, D. G. Downey, P. A. Flume, P. Drevinek, C. H. Goss, I. FajacA. S. Magaret, B. S. Quon, S. M. Singleton, J. M. VanDalfsen, G. Z. Retsch-Bogart

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review

7 Citations (Scopus)
132 Downloads (Pure)


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.

Original languageEnglish
Pages (from-to)677-687
Number of pages11
JournalJournal of Cystic Fibrosis
Issue number5
Early online date07 Jun 2020
Publication statusPublished - Sep 2020

Bibliographical note

Copyright © 2020. Published by Elsevier B.V.


  • Cystic Fibrosis/drug therapy
  • Cystic Fibrosis Transmembrane Conductance Regulator/drug effects
  • Drug Development/organization & administration
  • Humans
  • International Cooperation

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Pulmonary and Respiratory Medicine


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