Abstract
In patients with cystic fibrosis (CF), clinical trials are of paramount importance. Here, the current status of drug development in CF is discussed and future directions highlighted. Methods for pre-clinical testing of drugs with potential activity in CF patients including relevant animal models are described. Study design options for phase II and phase III studies involving CF patients are provided, including required patient numbers, safety issues and surrogate end point parameters for drugs, tested for different disease manifestations. Finally, regulatory issues for licensing new therapies for CF patients are discussed, including new directives of the European Union and the structure of a European clinical trial network for clinical studies involving CF patients is proposed.
Original language | English |
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Pages (from-to) | 85-99 |
Number of pages | 15 |
Journal | Journal of Cystic Fibrosis |
Volume | 6 |
Issue number | 2 |
Early online date | 12 Mar 2007 |
DOIs | |
Publication status | Published - Apr 2007 |
Keywords
- drug development
- pre-clinical drug testing
- animal models
- surrogate end points
- safety issues
- drug licensing
- european clinical trial network for CF
- Consesnsus study Group
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Dive into the research topics of 'Clinical trials in cystic fibrosis'. Together they form a unique fingerprint.Impacts
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Improving outcomes for people with cystic fibrosis through evidence based clinical trials
(Participant), Martin, L. (Participant), Downey, D. (Participant), (Participant) & (Participant)
Impact: Health Impact, Quality of Life Impact