January 31, 2017
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Ocaliva needs ‘substantial price reduction’ to be cost-effective for PBC patients

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Ocaliva appears promising for improving long-term outcomes in patients with primary biliary cholangitis, but it is not cost-effective at its current annual price, according to estimates from a simulation study.

Last year the FDA approved Ocaliva (obeticholic acid, Intercept Pharmaceuticals) — a novel bile acid analogue — as a second-line treatment for primary biliary cholangitis alone or in combination with ursodeoxycholic acid. The approval followed results from the POISE trial, which showed obeticholic acid improved biochemical markers of liver function in patients with PBC.

Jagpreet Chhatwal
Jagpreet Chhatwal

Obeticholic acid “represents a significant advance in treatment for PBC patients who had an inadequate response to [ursodeoxycholic acid],” Jagpreet Chhatwal, PhD, of the Institute for Technology Assessment at Massachusetts General Hospital, and Harvard Medical School, and colleagues wrote. “However, the POISE trial provides only 12 months of study, and no long-term clinical effectiveness data are yet available. In addition, the list price of [obeticholic acid], ie, nearly $70,000/year, is substantially higher than that of the previous standard of care, [ursodeoxycholic acid], which costs around $3,000/year; and its cost-effectiveness for PBC treatment is unknown.”

Chhatwal and colleagues therefore sought to estimate the long-term clinical outcomes and cost-effectiveness of obeticholic acid using a microsimulation model, which simulated long-term outcomes of PBC patients treated with ursodeoxycholic acid alone vs. obeticholic acid plus ursodeoxycholic acid. They incorporated efficacy data from the POISE trial and published natural history data into the model, and validated model outcomes with PBC Global Study data.

Compared with ursodeoxycholic alone, the model showed that over 15 years, obeticholic acid plus ursodeoxycholic acid could reduce the cumulative incidences of decompensated cirrhosis from 12.2% to 4.5%, hepatocellular carcinoma from 9.1% to 4%, liver transplants from 4.5% to 1.2% and liver-related deaths from 16.2% to 5.7%. In addition, it could increase 15-year transplant-free survival from 61.1% to 72.9%.

“Treating 10,000 patients using [obeticholic acid plus ursodeoxycholic acid] could prevent 770 cases of decompensated cirrhosis, 510 cases of HCC, 330 liver transplants, and 1,050 liver-related deaths,” Chhatwal and colleagues wrote.

On the other hand, the model showed that the addition of obeticholic acid would increase the lifetime cost of PBC treatment from $63,100 to $902,000, a 1,330% increment. The discounted quality-adjusted life years were 10.74 for ursodeoxycholic acid and 11.78 for obeticholic acid plus ursodeoxycholic acid, with corresponding costs-per-patient of $142,3000 and $633,900, respectively. This would result in an incremental cost-effectiveness ratio of $473,400 per quality-adjusted life year gained for obeticholic acid vs. ursodeoxycholic acid.

“Using a willingness-to-pay threshold of $100,000/QALY, [obeticholic acid] was not a cost-effective option in PBC patients who have an inadequate response to [ursodeoxycholic acid] monotherapy,” Chhatwal and colleagues concluded. “Our analysis projects that the use of [obeticholic acid] will substantially improve long-term clinical outcomes of PBC patients. However, substantial price reduction (below $18,450/year, a 73% reduction from the list price) is needed to make treatment with [obeticholic acid] cost-effective in PBC patients.” – by Adam Leitenberger

Disclosures: Chhatwal reports he consults, advises and has received grants from Gilead, and advises for Merck.