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Questions and Answers on Exocrine Pancreatic Insufficiency Drug Products 1. What is the Food and Drug Administration (FDA) announcing today? The FDA is announcing that all exocrine pancreatic insufficiency drug products are new drugs and is announcing the conditions for continued marketing of these drug products. FDA previously announced that all pancreatic insufficiency products must be available by prescription only. These conditions relate to proving that pancreatic enzyme products (PEPs) meet standards for quality, safety, and efficacy. At this time, the FDA is requiring manufacturers of pancreatic enzyme drug products to submit new drug applications (NDAs) and receive FDA approval to market their products. The Agency has also determined that PEPs are medically necessary and accordingly, is allowing manufacturers to continue marketing and obtain approval of their products within four years. 2. What are these products used for? PEPs aid in the digestion of meals in patients who have diseases affecting their pancreatic function. PEPs contain the ingredients pancreatin and pancrelipase, both of which contain the enzymes lipase, protease, and amylase. These enzymes break down fats (lipase), proteins (protease) and complex carbohydrates (amylase) to allow absorption of these nutrients into the body. 3. Who takes pancreatic enzyme products in the United States (U.S.)? Patients who take these products are those with cystic fibrosis and chronic pancreatitis conditions. 4. What is Cystic Fibrosis (CF) and Pancreatitis? Cystic fibrosis (CF) is a genetic disease affecting approximately 30,000 children and adults in the United States. CF causes the body to produce an abnormally thick, sticky mucus, due to the faulty transport of sodium and chloride (salt) within the cells that line organs such as the lungs and pancreas, to the outer surface of these cells. The thick CF mucus also obstructs the pancreas, preventing digestive enzymes from reaching the intestines to help break down and digest food. Pancreatitis is a disease of the pancreas, affecting approximately 14,000-28,000 adults. Patients with chronic pancreatitis lack sufficient pancreatic enzymes. This lack of enzymes results in the body’s inability to properly digest fat. 5. 5. Why has FDA decided now to regulate PEPs? PEPs of porcine or bovine origin have been available in the U.S. as prescription and over-the-counter (OTC) products for the treatment of exocrine pancreatic insufficiency in children and adults with cystic fibrosis and chronic pancreatitis diseases since before the enactment of the Federal Food, Drug, and Cosmetic Act of 1938. It was in 1938 that “new drugs” were required to be the subject of NDAs. FDA has always regulated PEPs to the extent that it monitored the labeling and manufacturing processes. With the exception of a PEP approved in 1996, PEPs have been marketed without NDAs. Over the years, FDA has received reports of problems associated with these drugs. Initially, the reports dealt with adverse events associated with high doses of enzymes leading to strictures of the intestines (narrowing of the digestive tract). More recently the Agency has received a number of reports claiming that these drug products do not have the expected therapeutic effect. There are many reasons why certain products may be subtherapeutic, including inactive ingredients or inadequate amounts of active drug in the capsule. FDA has determined that more controls are needed in the manufacturing of PEPs to ensure consistent potency and quality. Given that PEPs are taken as maintenance medications, avoidance of both over- and under-treatment is critical to maintaining the health and well being of patients with CF or chronic pancreatitis. This goal is best accomplished by having products with consistent quality, potency and stability. 6. Is there a public health crisis? This announcement is not the result of a public health crisis. The FDA is taking this action to ensure that these drugs are manufactured to reproducibly high standards and are demonstrated to be safe and effective. Manufacturers of PEPs are being asked to submit marketing applications for their drug products. 7. Are groups that are likely to be impacted by this proposed rule supportive of FDA's actions? In a statement issued today by the Cystic Fibrosis Foundation, the organization stated, "The Cystic Fibrosis Foundation supports the FDA's announcement to require pancreatic enzymes to obtain NDAs. The FDA's oversight will provide assurance on the safety and potency of all pancreatic enzymes. It also will improve the health of people with CF and help to address adverse events reported by patients who take certain enzyme products." Cystic Fibrosis Foundation Statement. 8. If manufacturers do not submit marketing applications, will there be a shortage of PEPs? At this time there is no shortage of PEPs, and the Agency does not anticipate any shortages as a result of this announcement. The Agency recognizes that it will take time for applicants to obtain approved applications. In an effort to avoid a shortage, the Agency is providing manufacturers adequate time to collect the data needed to submit an application. Manufacturers have 4 years to obtain approved applications. Currently there are 39 different formulations available for patients. 9. What should doctors do at this time? Doctors should be aware that manufacturers of PEPs must receive approval for their drug products by 2008. If some current manufacturers do not receive marketing approval or choose not to seek approval, patients may need to be switched to other approved brands at that time. 10. What should I do if I am currently taking one of these products? Continue taking PEPs as prescribed by your doctor. Because these products are taken as maintenance medications, the FDA is taking this action to ensure their quality. 11. Will I have problems if I stop taking pancreatic enzymes? Yes. Because your body requires PEPs for proper digestion, it is possible that if you stop taking your medication you may have diarrhea or loose stools. Please talk with your doctor before stopping or changing any medications. 12. Are there any concerns about these products? The Agency believes that the pre-clearance of each product is necessary to provide patients with the same quality, safety and efficacy that comes with all FDA-approved products. 13. Are these drug products available only by prescription? Yes. The FDA published a final rule on April 25, 1995 concerning over-the-counter PEPs. It was determined that all PEPs had to be marketed as prescription drug products and needed an approved NDA for continued marketing. 14. What about dietary supplements that contain pancreatic enzymes? Will they remain on the market? Under the law, FDA is not authorized to require premarket approval of products legally sold in the United States as dietary supplements. The Agency’s action in this matter should have no impact on legally marketed dietary supplements. 15. Are any of the PEPs FDA approved? There is one approved prescription PEP. The trade name of this immediate release product is Cotazym. However, the manufacturer, Organon, is not currently marketing the product. 16. Will these products be recalled from the pharmacy? No. FDA is not asking for a recall of these products while manufacturers are applying for marketing approval. 17. Will my pharmacist continue to fill my prescription? May I ask for a generic? Yes, your pharmacist will continue to fill your prescriptions. However, pharmacists should be aware that these products have different formulations. At this time, FDA does not have sufficient data to determine therapeutic equivalence of the drug products. Therefore, the FDA does not recommend substitution of these products.
18.
What economic impact does FDA expect that this action will
have? 19. How can I report a serious side effect to FDA? You can report an adverse event the following ways:
20. Where can I get more information relating to PEPs? Additional information can be obtained from the following organizations: National Digestive Diseases Information
Clearinghouse The Cystic Fibrosis Foundation National Library of Medicine, National
Institutes of Health FDA
Drug Information at: 888-INFO-FDA (888-463-6332)
Date created: April 27, 2004 |
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