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

Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society
Nicole Mayer-HamblettG Z Retsch-Bogart

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.

Citations

Aug 4, 2020·Expert Opinion on Investigational Drugs·Ranjani SomayajiScott C Bell
Jan 26, 2021·European Journal of Medicinal Chemistry·Valentina SalaAlessandra Ghigo
Jan 8, 2021·Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society·R Elaine Cagnina, Gregory S Sawicki
Jun 3, 2021·Journal of Personalized Medicine·Genevieve ShemieBartha Maria Knoppers
Aug 19, 2021·Pediatric Pulmonology·Danielle M Goetz, Adrienne P Savant

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