title: Study design considerations for evaluating the efficacy and safety of pancreatic enzyme replacement therapy in patients with cystic fibrosis.
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.
authors.affiliation: Case Western Reserve University School of Medicine, Cleveland, OH, USA.
authors.forename: Michael W
authors.initials: MW
authors.lastname: Konstan
authors.affiliation: State University of New York at Buffalo, Buffalo, NY, USA ; Cystic Fibrosis Foundation Data Safety Monitoring Board, Tucson, AZ, USA.
authors.forename: Drucy
authors.initials: D
authors.lastname: Borowitz
authors.affiliation: University of Washington, Seattle, WA, USA ; CF Therapeutics Development Network Coordinating Center, Seattle, WA, USA.
authors.forename: Nicole
authors.initials: N
authors.lastname: Mayer-Hamblett
authors.affiliation: Stanford University, Palo Alto, CA, USA.
authors.forename: Carlos
authors.initials: C
authors.lastname: Milla
authors.affiliation: University of Florida, Gainesville, FL, USA.
authors.forename: Leslie
authors.initials: L
authors.lastname: Hendeles
authors.affiliation: Cystic Fibrosis Foundation Data Safety Monitoring Board, Tucson, AZ, USA ; University of Michigan, Ann Arbor, MI, USA.
authors.forename: Susan
authors.initials: S
authors.lastname: Murray
authors.affiliation: University of Washington, Seattle, WA, USA ; CF Therapeutics Development Network Coordinating Center, Seattle, WA, USA.
authors.forename: Richard A
authors.initials: RA
authors.lastname: Kronmal
authors.affiliation: Seattle Children's Hospital , Seattle, WA, USA.
authors.forename: Susan
authors.initials: S
authors.lastname: Casey
authors.affiliation: University of Washington, Seattle, WA, USA ; CF Therapeutics Development Network Coordinating Center, Seattle, WA, USA.
authors.forename: Lynn M
authors.initials: LM
authors.lastname: Rose
authors.affiliation: Cystic Fibrosis Foundation Data Safety Monitoring Board, Tucson, AZ, USA ; University of Arizona, Tucson, AZ, USA.
authors.forename: Wayne J
authors.initials: WJ
authors.lastname: Morgan
authors.affiliation: University of Washington, Seattle, WA, USA ; CF Therapeutics Development Network Coordinating Center, Seattle, WA, USA.
authors.forename: Bonnie W
authors.initials: BW
authors.lastname: Ramsey
keywords: coefficient of fat absorption; cystic fibrosis; pancreatic enzyme replacement therapy; pancreatic insufficiency; study design