Community Perspective on Pancreatic Enzyme Replacement Therapy in Cystic Fibrosis

People with cystic fibrosis (CF) and exocrine pancreatic insufficiency must take pancreatic enzyme replacement therapy (PERT) to prevent malnutrition and gastrointestinal (GI) symptoms. Finding better ways to manage GI complaints is a high priority for the CF community. We fielded a survey to assess the perspective of people affected by CF regarding symptoms attributed to and challenges associated with current PERT, to identify factors that affect participation in PERT studies, and to understand attitudes toward an outcome measure that could be an alternative to the coefficient of fat absorption test. Persistent GI symptoms are commonly ascribed to PERT. Minimizing time commitment and maximizing patient safety were factors affecting participation in research. We demonstrate 4 generalizable ways to incorporate patient experience early in the research process to aid in development of new medications and help improve study enrollment.