Real-world evidence in cystic fibrosis modulator development: Establishing a path forward.

Competing Interests: Declaration of Competing Interest Dr. Magaret reports grants from NIH and Cystic Fibrosis Foundation, during the conduct of the study; Mr. Warden reports grants from National Heart, Lung, and Blood Institute, and from Cystic Fibrosis Foundation, during the conduct of the study; Dr. Mayer-Hamblett reports grants from Cystic Fibrosis Foundation and from NIH, during the conduct of the study; personal fees from Calithera, personal fees from Kala, outside the submitted work; Drs. Simon and Heltshe have nothing to disclose.