Cystic fibrosis drug trial design in the era of CFTR modulators associated with substantial clinical benefit: stakeholders ’ consensus view

CFTR modulators, which treat the basic CF defect improve key clinical outcomes in PWCF, including quality of life (QoL). These drugs demonstrate disease modulation by decreasing pulmonary exacerbations as well as lung function decline, a correlate of survival [1 –5]. Registry data already indicate survival benefit for the first of these [6].

Source: Journal of Cystic Fibrosis - June 7, 2020 Category: Respiratory Medicine Authors: K. De Boeck, T. Lee, M. Amaral, P. Drevinek, J.S. Elborn, I. Fajac, E. Kerem, J.C. Davies Source Type: research