The following is a summary of “Community Perspective on Pancreatic Enzyme Replacement Therapy in Cystic Fibrosis” published in the November 2022 issue of Pediatric Gastroenterology and Nutrition by Borowitz et al.
Individuals diagnosed with cystic fibrosis (CF) and exocrine pancreatic insufficiency are required to undergo pancreatic enzyme replacement therapy (PERT) to forestall malnutrition and gastrointestinal (GI) symptoms. Therefore, the CF community places a high premium on the research and development of new approaches that are more effective in managing GI problems.
Researchers conducted a survey to determine the perspectives of people who are affected by CF regarding the symptoms that are attributed to the current PERT and the challenges that are associated with it, to determine the 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. PERT is a prevalent cause of persistent gastrointestinal (GI) problems.
The amount of time that needed to be dedicated to the research was kept to a minimum, and the priority that was placed above all others was the safety of the patients. They illustrate 4 generalizable techniques for incorporating patient experience early on in the research process to help develop novel pharmaceuticals and improve trial enrollment. This is done to improve trial enrollment and aid in the creation of novel pharmaceuticals. These are some of the strategies that should help boost enrollment in the study.