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Drug Development for Cystic Fibrosis
  • Don Sanders,
  • James Chmiel
Don Sanders
Riley Hospital for Children at Indiana University Health

Corresponding Author:[email protected]

Author Profile
James Chmiel
Riley Hospital for Children at Indiana University Health
Author Profile


The first drug specific for cystic fibrosis (CF) was approved in 1993, and since then several other drugs have been approved. Median predicted survival in people with CF has improved from approximately 30 years to 44.4 years over that same period. In 2020, highly-effective modulators of the cystic fibrosis transmembrane conductance regulator became available to approximately 90% of people with CF ages 12 years and older. These transformative therapies will surely reduce morbidity and further extend longevity. The drug development pipeline is filled with therapies that address most aspects of CF disease. As survival and CF therapies have advanced, and the complexity of CF care increases, the process of drug development has become more sophisticated. In addition, detecting meaningful changes in clinical outcomes has become more difficult as the health status of people with CF improves. Design of clinical trials in CF has become more complex, and innovative approaches are required to continue to advance drug development. This review provides a general overview of drug development from the pre-clinical phase through Phase IV. Special considerations with respect to CF are integrated into the discussion of each phase of drug development. The dawn of a new era has arrived for people with CF. As CF care evolves, drug development must continue to evolve as well, until a one-time cure is available to all people with CF.
30 Jun 2020Submitted to Pediatric Pulmonology
01 Jul 2020Submission Checks Completed
01 Jul 2020Assigned to Editor
02 Jul 2020Reviewer(s) Assigned
28 Jul 2020Review(s) Completed, Editorial Evaluation Pending
29 Jul 2020Editorial Decision: Revise Major
27 Aug 20201st Revision Received
28 Aug 2020Assigned to Editor
28 Aug 2020Submission Checks Completed
28 Aug 2020Reviewer(s) Assigned
10 Sep 2020Review(s) Completed, Editorial Evaluation Pending
10 Sep 2020Editorial Decision: Revise Minor
11 Sep 20202nd Revision Received
12 Sep 2020Submission Checks Completed
12 Sep 2020Assigned to Editor
12 Sep 2020Reviewer(s) Assigned
13 Sep 2020Review(s) Completed, Editorial Evaluation Pending
13 Sep 2020Editorial Decision: Accept
Feb 2021Published in Pediatric Pulmonology volume 56 issue S1. 10.1002/ppul.25075