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

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

SUBMITTER: Mayer-Hamblett N 

PROVIDER: S-EPMC7492419 | biostudies-literature | 2020 Sep

REPOSITORIES: biostudies-literature