The Cystic Fibrosis Foundation has announced its support of the
Food and Drug Administration's (FDA) requirement that manufacturers of pancreatic enzyme replacements obtain FDA approval for their products within the next four years. This rule allows enzyme products to remain available to patients during that time. At the CF Foundation's urging, an FDA review found that inconsistencies in the formulation, dosage and manufacturing processes of many enzymes could significantly compromise both the safety and effectiveness of the drugs in patients with pancreatic diseases, including
cystic fibrosis (CF).
The CF Foundation has received several reports of treatment failure after pharmacists supplied a substitute enzyme in place of that prescribed by the physician. Approximately 90 percent of people with CF require supplementation with enzymes and rely on the predictability of a known dose of a specific pancreatic enzyme product. Variations in the active ingredients can alter therapeutic impact and result in undesirable side effects such as malabsorption and constipation.
The enzyme products available on the market today have never received FDA approval because pancreatic enzymes predate the 1938 passage of the FDA approval act. The FDA has allowed these products to be "grandfathered" and to remain on the market because of their lifesaving benefits for patients. The lack of quality and consistency in many enzyme products, however, has brought this lack of approval into question and has stimulated this mandate. It gives manufacturers four years to obtain FDA approval, during which time their products will continue to be available to patients, unless the manufacturer voluntarily withdraws its product.
The CF Foundation strongly encouraged the FDA to initiate a review of enzyme products to aid patient safety. Before the recent FDA decision, the CF Foundation alerted CF care center staff members, as well as patients and families, to the importance of non-substitution of enzyme prescriptions. Leslie Hendeles, Pharm.D., professor of pharmacy and pediatrics at the University of Florida, also was instrumental in the success of this initiative.
"This decision is a positive one for the thousands of individuals affected by CF. Proper digestion and absorption of food and nutrients is so important in people with CF; if they are not using products with reliable performances, their digestion and their health suffer as a result," said Hendeles. "This standardization of enzymes will eliminate unreliable products and lead to accurate product knowledge for patients, families and the healthcare professionals who treat them."