BUILDING GLOBAL DEVELOPMENT STRATEGIES FOR CF THERAPEUTICS DURING A TRANSITIONAL CFTR MODULATOR ERA
| Autor: | John P. Clancy, K. De Boeck, Michael W. Konstan, Pavel Drevinek, George Z. Retsch-Bogart, Amalia Magaret, Isabelle Fajac, Bradley S. Quon, Anthony G. Durmowicz, S.M. Singleton, Donald R. VanDevanter, Jane C. Davies, Scott C. Bell, Scott H. Donaldson, Patrick A. Flume, K. Pearson, Jennifer L. Taylor-Cousar, Christopher H. Goss, Damian G Downey, S. van Koningsbruggen-Rietschel, Nicole Mayer-Hamblett, Felix Ratjen, Tim W.R. Lee, Jill M. VanDalfsen, Dave P. Nichols |
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| Jazyk: | angličtina |
| Rok vydání: | 2020 |
| Předmět: |
0301 basic medicine
Pulmonary and Respiratory Medicine Standard of care International Cooperation Harmonization Article 03 medical and health sciences 0302 clinical medicine Pairwise sequence alignment Medicine Humans Pediatrics Perinatology and Child Health business.industry Drug Development/organization & administration Clinical study design Cystic Fibrosis Transmembrane Conductance Regulator/drug effects 030104 developmental biology 030228 respiratory system Drug development Risk analysis (engineering) Pediatrics Perinatology and Child Health Cystic Fibrosis/drug therapy business International development Cftr modulator |
| Zdroj: | J Cyst Fibros 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 & Retsch-Bogart, G Z 2020, ' Building global development strategies for cf therapeutics during a transitional cftr modulator era ', Journal of Cystic Fibrosis, vol. 19, no. 5, pp. 677-687 . https://doi.org/10.1016/j.jcf.2020.05.011 |
| DOI: | 10.1016/j.jcf.2020.05.011 |
| Popis: | 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. |
| Databáze: | OpenAIRE |
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