{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,23]],"date-time":"2026-07-23T02:23:26Z","timestamp":1784773406094,"version":"3.55.0"},"reference-count":29,"publisher":"Ovid Technologies (Wolters Kluwer Health)","issue":"3","license":[{"start":{"date-parts":[[2017,1,17]],"date-time":"2017-01-17T00:00:00Z","timestamp":1484611200000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1"},{"start":{"date-parts":[[2017,1,17]],"date-time":"2017-01-17T00:00:00Z","timestamp":1484611200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions"}],"content-domain":{"domain":["lww.com","ovid.com"],"crossmark-restriction":true},"short-container-title":["Hepatology"],"published-print":{"date-parts":[[2017,3]]},"abstract":"<jats:p>Primary biliary cholangitis (PBC) is a chronic, progressive autoimmune liver disease that mainly affects middle\u2010aged women. Obeticholic acid (OCA), which was recently approved by the Food and Drug Administration for PBC treatment, has demonstrated positive effects on biochemical markers of liver function. Our objective was to evaluate the long\u2010term clinical impact and cost\u2010effectiveness of OCA as a second\u2010line treatment for PBC in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA. We developed a mathematical model to simulate the lifetime course of PBC patients treated with OCA+UDCA versus UDCA alone. Efficacy data were derived from the phase 3 PBC OCA International Study of Efficacy trial, and the natural history of PBC was informed by published clinical studies. Model outcomes were validated using the PBC Global Study. We found that in comparison with UDCA, OCA+UDCA could decrease the 15\u2010year cumulative incidences of decompensated cirrhosis from 12.2% to 4.5%, hepatocellular carcinoma from 9.1% to 4.0%, liver transplants from 4.5% to 1.2%, and liver\u2010related deaths from 16.2% to 5.7% and increase 15\u2010year transplant\u2010free survival from 61.1% to 72.9%. The lifetime cost of PBC treatment would increase from $63,000 to $902,000 (1,330% increment). The discounted quality\u2010adjusted life years with UDCA and OCA+UDCA were 10.74 and 11.78, respectively, and the corresponding costs were $142,300 and $633,900, resulting in an incremental cost\u2010effectiveness ratio of $473,400\/quality\u2010adjusted life year gained. The results were most sensitive to the cost of OCA.<jats:italic toggle=\"yes\">Conclusion<\/jats:italic>: OCA is a promising new therapy to substantially improve the long\u2010term outcomes of PBC patients, but at its current annual price of $69,350, it is not cost\u2010effective using a willingness\u2010to\u2010pay threshold of $100,000\/quality\u2010adjusted life year; pricing below $18,450\/year is needed to make OCA cost\u2010effective. (H<jats:sc>epatology<\/jats:sc>2017;65:920\u2010928).<\/jats:p>","DOI":"10.1002\/hep.28932","type":"journal-article","created":{"date-parts":[[2016,11,7]],"date-time":"2016-11-07T23:29:01Z","timestamp":1478561341000},"page":"920-928","update-policy":"https:\/\/doi.org\/10.1097\/lww.0000000000001000","source":"Crossref","is-referenced-by-count":84,"title":["Long\u2010term clinical impact and cost\u2010effectiveness of obeticholic acid for the treatment of primary biliary cholangitis"],"prefix":"10.1097","volume":"65","author":[{"given":"Sumeyye","family":"Samur","sequence":"first","affiliation":[{"name":"Institute for Technology Assessment,Massachusetts General Hospital,Boston,MA"},{"name":"Harvard Medical School,Boston,MA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Matthew","family":"Klebanoff","sequence":"additional","affiliation":[{"name":"Institute for Technology Assessment,Massachusetts General Hospital,Boston,MA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Reiner","family":"Banken","sequence":"additional","affiliation":[{"name":"Institute for Clinical and Economic Review,Boston,MA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Daniel S.","family":"Pratt","sequence":"additional","affiliation":[{"name":"Harvard Medical School,Boston,MA"},{"name":"Gastroenterology,Massachusetts General Hospital,Boston,MA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rick","family":"Chapman","sequence":"additional","affiliation":[{"name":"Institute for Clinical and Economic Review,Boston,MA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Daniel A.","family":"Ollendorf","sequence":"additional","affiliation":[{"name":"Institute for Clinical and Economic Review,Boston,MA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anne M.","family":"Loos","sequence":"additional","affiliation":[{"name":"Institute for Clinical and Economic Review,Boston,MA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kathleen","family":"Corey","sequence":"additional","affiliation":[{"name":"Harvard Medical School,Boston,MA"},{"name":"Gastroenterology,Massachusetts General Hospital,Boston,MA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chin","family":"Hur","sequence":"additional","affiliation":[{"name":"Institute for Technology Assessment,Massachusetts General Hospital,Boston,MA"},{"name":"Harvard Medical School,Boston,MA"},{"name":"Gastroenterology,Massachusetts General Hospital,Boston,MA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jagpreet","family":"Chhatwal","sequence":"additional","affiliation":[{"name":"Institute for Technology Assessment,Massachusetts General Hospital,Boston,MA"},{"name":"Harvard Medical 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