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Study design considerations for evaluating the efficacy and safety of pancreatic enzyme replacement therapy in patients with cystic fibrosis

Year: 2013
Type: article
Abstract: In 2006, the US FDA issued a 'Guidance for Industry' regarding submission of New Drug Applications for pancreatic enzyme replacement therapy (PERT) products. Five oral delayed-release PERT products have been approved by the FDA, and several others are under development and/ or evaluation for New Drug Application submission. We present in this paper recommendations of the Cystic Fibrosis Foundation's Cystic Fibrosis (CF) Therapeutics Development Network and Data Safety Monitoring Board regarding study design considerations for evaluating PERT products in patients with CF. Careful attention to study design and accuracy of the outcome measures has confirmed our understanding of the efficacy and safety of PERT for the treatment of exocrine pancreatic insufficiency of CF.
DOI: 10.4155/cli.13.63
Authors: Michael W Konstan, Drucy Borowitz, Nicole Mayer-Hamblett, Carlos Milla, Leslie Hendeles, Susan Murray, Richard A Kronmal, Susan Casey, Lynn M Rose, Wayne J Morgan, Bonnie W Ramsey
Institutions: Case Western Reserve University, Cystic Fibrosis Foundation, University at Buffalo, State University of New York, University of Washington, Stanford University, University of Florida, University of Michigan, Seattle Children's Hospital, University of Arizona
Language: English
Field: Medicine
Open Access status: green
Award: G627775621
OpenAlex ID: W2005976845
FWCI: 1.221
Cites: 47
Cited by: 12
Related to: 10