Off-label use of new liver drug may drive up costs
Obeticholic acid (OCA), is scheduled for review by the U.S. Food and Drug Administration (FDA) at the end of May 2016. OCA will treat primary biliary cirrhosis (PBC).
OCA is also in Phase 2 studies for treatment of another liver disease. When OCA comes to the market, it may attract off-label use.
What is PBC?
Primary biliary cirrhosis is a chronic disease that causes the small bile ducts in the liver to become inflamed and damaged and ultimately disappear. The liver slowly deteriorates and is unable to function normally.
To date, the only treatment for PBC is ursodeoxycholic acid (Ursodiol), which can delay disease progression and improve survival rates without a liver transplant. But approximately 25 percent of people with PBC do not respond to Ursodiol.
Prime estimates that 2 in 100,000 commercial members may use OCA to treat PBC at a cost of $60,000/year.1,2
Second possible indication for OCA
Intercept, OCA's manufacturer, is also in the early stages of investigating OCA for the treatment of nonalcoholic steatohepatitis (NASH). Early review of the use of OCA in NASH may occur as early as 2018, but is currently anticipated in 2021.
What is NASH?
NASH is a common, often “silent” liver disease. It resembles alcoholic liver disease, but can occur in people who drink little or no alcohol. Most people with NASH feel well and are not aware that they have a liver problem. It affects 2 to 5 percent of the population. NASH can be severe and can lead to cirrhosis, in which the liver is permanently damaged and scarred and no longer able to work properly.
Early, off-label use of OCA to treat NASH could increase costs significantly. UM criteria is available that can help guide doctors to prescribe OCA for PBC only, its only anticipated 2016-2017 FDA-label approved use.
People with NASH often have other medical conditions, such as diabetes, high blood pressure, or elevated cholesterol. Standard recommendations to people with this disease are to:
- Reduce their weight (if obese or overweight)
- Follow a balanced and healthy diet
- Increase physical activity
- Avoid alcohol
- Avoid unnecessary medicines
These can make a difference and are also helpful for other co-existing conditions, such as heart disease, diabetes and high cholesterol. Weight loss can improve liver tests in patients with NASH and may reverse the disease to some extent.3
Because no specific therapies for NASH exist, there is the possibility for off-label use upon OCA’s entry into the market in 2016. Prime estimates 50 of 100,000 commercial members may use OCA off-label to treat NASH for a cost of $60,000/year.1 (These numbers are estimated for one year post launch, if no utilization management is put in place to help limit use to the initial FDA-approved label, which would be PBC.)
Your Blue Cross Plan will include OCA in FastPAth, which will essentially only allow use for the FDA-approved indication.
- Prime commercial BoB analysis 2014 medical and pharmacy claims
- Hirschfield GM, Mason A, Luketic V, et al. Gastroenterology 2015;148(4):751-761. Accessed Nov 9, 2015. http://www.sciencedirect.com/science/article/pii/S0016508514015303
- National Institute for diabetes and digestive kidney diseases. Accessed at: http://www.niddk.nih.gov/health-information/health-topics/liver-disease/nonalcoholic-steatohepatitis/Pages/facts.aspx#causes