The Food and Drug Administration (FDA) regulatory process exists to ensure safety and efficacy of products brought to market. After 2004, when patient advocacy groups pressured the FDA, pancreatic enzyme products (PEPs) were added to the list of products required to have a new drug approval submitted before they can be marketed. This has reduced the number of available PEPs and driven up the costs of non-generic PEPs, affecting many patients suffering from cystic fibrosis.
Pancreatic enzyme products contain pancrelipase, a mixture of amylase, lipase, and protease. These digestive enzymes are essential for individuals with exocrine pancreatic insufficiency, such as those who have cystic fibrosis or chronic pancreatitis. Without PEPs, patients are at risk for malnutrition and osteoporosis, and the treatment regimen is lifelong. Previously, PEPs were easily accessible: “Up until about seven years ago, there were around 30 different prescription-based formulations for pancreatic enzyme therapy,” said Timothy Gardner, MD, at the 2013 American College of Gastroenterology Annual Scientific Meeting. “We now have six products approved.”
Dr. Gardner wanted to quantify the effect the new regulations had on the cost and accessibility of PEPs. Along with Stuart L. Gordon, he examined the IMS Health Product Report Database for PEPs. “We obtained the cost per prescription by dividing the total prescriptions by the total amount of sales during the period from 2008 to 2012,” stated Dr. Gardner. The new drug application requirement was instilled in 2009-2010. “We found … the total sales for non-generic pancreatic enzyme replacement products were $281 million before this was put in place. In 2012, for which the most recent data is available, it was up to $632 million,” concluded Dr. Gardner. “If we look at the cost per prescription before and after the NDA requirement, the cost for each individual prescription in 2008 was $247. By 2012, the cost had increased to $577.”
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The cost increase is staggering, but “because children who have cystic fibrosis often have very robust insurance programs, their prescriptions are covered,” said Dr. Gardner. On the other hand, “In my experience, this is not necessarily the case in patients with chronic pancreatitis.” According to Christopher E. Forsmark, MD, from University of Florida College of Medicine, Gainesville, “The issue is that most patients with chronic pancreatitis are not treated with enzymes, and those that are often receive an inadequate dosage. The inadequate dose is partly the fault of physicians lacking knowledge about dosing, but many patients just can’t afford the products.”
The study was limited in that it was based on administrative data that does not track all available PEPs, including generics. “But we have concluded that the NDA requirement for these products resulted in universal prescription price increases and in more non-generic prescriptions for these products. Also, at this point, three years after this requirement, there has been no post-marketing safety data published, which was the original goal of this requirement,” said Dr. Gardner. However, the FDA continues to review submitted applications for PEPs and reaches out to affected patients, such as those with cystic fibrosis, through FAQs.