The U.S. Food and Drug Administration (FDA) approved Ocaliva (obeticholic acid) on May 27. Manufactured by Intercept Pharmaceuticals, Inc. (Intercept), Ocaliva is approved to treat adults with primary biliary cholangitis (PBC) (previously known as primary biliary cirrhosis) in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA, or as a single therapy in adults unable to tolerate UDCA.1
What does Ocaliva treat?
PBC is a chronic disease that causes the small bile ducts in the liver to become inflamed, damaged and ultimately disappear. The liver slowly deteriorates and is unable to function normally. To date, the primary treatment for PBC has been Ursodiol (ursodeoxycholic acid), which can delay disease progression and improve survival rates without a liver transplant. Approximately 25 percent of people with PBC do not respond to Ursodiol.2
How much will Ocaliva cost?
The exact price for Ocaliva is not yet known. For forecasting purposes, Prime estimated the cost of treatment to be $60,000 per year or $5,000 per month. Prime estimates that 2 per 100,000 commercial members will use Ocaliva at one year post launch for the treatment of PBC.3
Using Prime’s forecast for potential use, the estimated cost to health plans would be $0.10 per member per month (PMPM) for commercially insured members at one year post launch.3
Intercept is in the early stages of investigating Ocaliva for the treatment of nonalcoholic steatohepatitis (NASH). Review of the use of Ocaliva in NASH may occur as early as 2018, but is currently anticipated in 2021.4
NASH is a common, often “silent” liver disease that resembles alcoholic liver disease, but can occur in people who drink little or no alcohol. 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. It affects 2 to 5 percent of the population.5
There are currently no specific drug therapies to treat NASH. The approval of Ocaliva could lead to off-label use. Potential for off-label use could bring significant costs
Prime estimates that 52 per 100,000 commercial members could use Ocaliva at one year post launch for the treatment of PBC and off-label treatment of NASH for a cost of $60,000/year.3
(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. OCA is included in FastPAth, which essentially would only allow for use for the FDA-approved indication.)
Using Prime’s forecast for potential use, the maximally estimated cost to health plans could be $2.60 per member per month (PMPM) for commercially insured members.3
Strategies to manage Ocaliva
Prime and Blue Cross are prepared to manage Ocaliva and help control costs. Utilization management (UM) criteria will be in place to ensure clinically appropriate, safe use of this new medicine and to help control costs for employers.
UM criteria like FastPAth can prevent off-label use to treat NASH, for example. Off-label use could cause significant increases in spend before the FDA has ruled on the drug’s effectiveness to treat NASH.
For questions, please contact your health plan representative.
1. U.S. Food and Drug Administration. “FDA approves Ocaliva for rare, chronic liver disease.” May 31, 2016. http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm503964.htm.
2. 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
3. Prime internal analysis, 2015.
4. Garde, Damian. “Intercept's much-hyped NASH drug misses the mark in Phase II.” FierceBiotech. October 28, 2015. http://www.fiercebiotech.com/r-d/intercept-s-much-hyped-nash-drug-misses-mark-phase-ii.
5. National Institute of Diabetes and Digestive and Kidney Diseases. “Nonalcoholic Steatohepatitis: Facts.” Accessed on April 12, 2016.
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