{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,2,28]],"date-time":"2026-02-28T04:32:55Z","timestamp":1772253175874,"version":"3.50.1"},"reference-count":28,"publisher":"Innovative Healthcare Institute","issue":"2","license":[{"start":{"date-parts":[[2024,5,1]],"date-time":"2024-05-01T00:00:00Z","timestamp":1714521600000},"content-version":"unspecified","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"},{"start":{"date-parts":[[2024,5,1]],"date-time":"2024-05-01T00:00:00Z","timestamp":1714521600000},"content-version":"unspecified","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2024,5]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Introduction<\/jats:title>\n                    <jats:p>Lomitapide is approved for lowering low-density lipoprotein cholesterol (LDL-C) in homozygous familial hypercholesterolemia, which is a rare genetic disorder. The evidence regarding its safety and efficacy from a small clinical trial requires further validation for effectiveness and safety in the real world. This study aimed to use institutional data on the effectiveness and safety of lomitapide to assist in formulating a perspective on adding it to the formulary.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>This was a retrospective review of patients who were actively prescribed lomitapide at King Abdulaziz Medical City, Riyadh, Saudi Arabia, from 2019 to 2022. Data collection included demographics, confirmed gene mutation results, duration of lomitapide therapy, baseline, on-treatment, last LDL-C levels, percent reduction in LDL-C after 1-3 months of therapy (whichever was first available), other LDL-C lowering therapies used, liver function tests, adverse effects, and compliance.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>Eight adult patients were included in the review, with a mean age of 25.5 years. Approximately 75% were female, and the duration of treatment with lomitapide ranged from 9 months to 3 years. None of the patients were on continuous LDL apheresis. The mean baseline LDL-C at presentation to our facility was 17.2 mmol\/L (range, 11.78\u201321.97 mmol\/L), the mean percent drop in LDL-C with lomitapide was 34.1% (range, 0%\u201387%), gastrointestinal disturbances were documented in 50% of the patients, and no cases of severe liver toxicities or increase in liver enzymes were seen.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>In our cohort of adult patients, lomitapide showed an overall modest reduction in LDL-C, with no cases of increase in liver enzymes and documented intolerance, indicating that most patients were likely noncompliant. This review revealed important considerations when reimbursing expensive medications for rare diseases. Real-world evidence in real-time can support healthcare systems in price negotiations and reaching mutual agreements that can eventually improve patient access to care.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.36401\/jqsh-23-32","type":"journal-article","created":{"date-parts":[[2024,1,20]],"date-time":"2024-01-20T07:00:23Z","timestamp":1705734023000},"page":"59-62","source":"Crossref","is-referenced-by-count":2,"title":["Lomitapide: A Medication Use Evaluation and a Formulary Perspective"],"prefix":"10.36401","volume":"7","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-2459-1485","authenticated-orcid":true,"given":"Laila Carolina Abu","family":"Esba","sequence":"first","affiliation":[{"name":"Pharmaceutical Care Department, King Abdulaziz Medical City, King Saud Bin Abdulaziz University for Health Sciences, King Abdullah International Medical Research Centre, Riyadh, Saudi Arabia"},{"name":"King Saud Bin Abdulaziz University for Health Sciences, Riyadh, Saudi Arabia"},{"name":"King Abdullah International Medical Research Centre, Riyadh, Saudi Arabia"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Hani","family":"Alharbi","sequence":"additional","affiliation":[{"name":"Pharmaceutical Care Department, King Fahd Hospital, University of Immam Abulrahman Bin Faisal, Dammam, Saudi Arabia"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"22027","reference":[{"key":"i2589-9449-7-2-59-b01","article-title":"UpToDate. Familial hypercholesterolemia in adults: overview","author":"Rosenson","year":"2020","unstructured":"Rosenson\n              R,\n            \n            \n              Durrington,\n              P.\n            \n          \n          UpToDate. Familial hypercholesterolemia in adults: overview. 2020. \nAccessed Oct 3, 2022. www.uptodate.com\/contents\/familial-hypercholesterolemia-in-adults-overview"},{"key":"i2589-9449-7-2-59-b02","series-title":"Eur Heart J","first-page":"560","article-title":"Homozygous autosomal dominant hypercholesterolaemia in the Netherlands: prevalence, genotype-phenotype relationship, and clinical outcome","volume":"36","author":"Sjouke","year":"2015","unstructured":"Sjouke\n              B,\n            \n            \n              Kusters\n              DM,\n            \n            \n              Kindt\n              I,\n            \n            et al.\n\n          Homozygous autosomal dominant hypercholesterolaemia in the Netherlands: prevalence, genotype-phenotype relationship, and clinical outcome. \nEur Heart J. \n2015;36:\n560\u2013565."},{"key":"i2589-9449-7-2-59-b03","series-title":"Adv Ther","first-page":"2159","article-title":"Homozygous familial hypercholesterolemia (HoFH) in Saudi Arabia and two cases of lomitapide use in a real-world setting","volume":"38","author":"Mahzari","year":"2021","unstructured":"Mahzari\n              M,\n            \n            \n              Zarif\n              H.\n            \n          \n          Homozygous familial hypercholesterolemia (HoFH) in Saudi Arabia and two cases of lomitapide use in a real-world setting. \nAdv Ther. \n2021;38:\n2159\u20132169."},{"key":"i2589-9449-7-2-59-b04","series-title":"Lancet","first-page":"719","article-title":"Worldwide experience of homozygous familial hypercholesterolaemia: retrospective cohort study","volume":"399","author":"Tromp","year":"2022","unstructured":"Tromp\n              TR,\n            \n            \n              Hartgers\n              ML,\n            \n            \n              Hovingh\n              GK,\n            \n            et al.\n\n          Worldwide experience of homozygous familial hypercholesterolaemia: retrospective cohort study. \nLancet. \n2022;399:\n719\u2013728."},{"key":"i2589-9449-7-2-59-b05","article-title":"Highlights of prescribing information, Juxtapid","author":"U.S. Food & Drug Adminstration.","year":"2022","unstructured":"U.S. Food & Drug Adminstration.\n          \n          Highlights of prescribing information, Juxtapid. 2022. \nAccessed Oct 3, 2022. www.accessdata.fda.gov\/drugsatfda_docs\/label\/2012\/203858s000lbl.pdf"},{"key":"i2589-9449-7-2-59-b06","series-title":"Lancet","first-page":"40","article-title":"Efficacy and safety of a microsomal triglyceride transfer protein inhibitor in patients with homozygous familial hypercholesterolaemia: a single-arm, open-label, phase 3 study","volume":"381","author":"Cuchel","year":"2013","unstructured":"Cuchel\n              M,\n            \n            \n              Meagher\n              EA,\n            \n            \n              du Toit Theron\n              H,\n            \n            et al.\n\n          Efficacy and safety of a microsomal triglyceride transfer protein inhibitor in patients with homozygous familial hypercholesterolaemia: a single-arm, open-label, phase 3 study. \nLancet. \n2013;381:\n40\u201346."},{"key":"i2589-9449-7-2-59-b07","article-title":"Drug list","author":"Saudi Food and Drug Authority","year":"2022","unstructured":"Saudi Food and Drug Authority\n          . \nDrug list. 2022. Accessed Oct 3, 2022. www.sfda.gov.sa\/en\/drugs-list?TradeName=&ScientificName=lomitapide&Agent=&ManufacturerName=&RegNo=&pg=1"},{"key":"i2589-9449-7-2-59-b08","article-title":"Ministry of National Guard Health Affairs website","author":"King Abdulaziz Medical City in Riyadh","unstructured":"King Abdulaziz Medical City in Riyadh\n          . \nMinistry of National Guard Health Affairs website. Accessed Oct 3, 2023. ngha.med.sa\/English\/MedicalCities\/Pages\/default.aspx"},{"key":"i2589-9449-7-2-59-b09","series-title":"Glob J Qual Safe Healthc","first-page":"55","article-title":"Pharmacy and therapeutics committee preparedness plan for COVID-19","volume":"3","author":"Abu Esba","year":"2020","unstructured":"Abu Esba\n              L,\n            \n            \n              Al-Abdulkarim\n              H,\n            \n            \n              Alrushidan\n              A,\n            \n            \n              Al Harbi\n              M.\n            \n          \n          Pharmacy and therapeutics committee preparedness plan for COVID-19. \nGlob J Qual Safe Healthc. \n2020;3:\n55\u201364."},{"key":"i2589-9449-7-2-59-b10","series-title":"P T","first-page":"293","article-title":"P&T committee drug prioritization criteria: a tool developed by a Saudi health care system","volume":"43","author":"Esba","year":"2018","unstructured":"Esba\n              LCA,\n            \n            \n              Almodaimegh\n              H,\n            \n            \n              Alhammad\n              A,\n            \n            \n              Ferwana\n              M,\n            \n            \n              Yousef\n              C,\n            \n            \n              Ismail\n              S.\n            \n          \n          P&T committee drug prioritization criteria: a tool developed by a Saudi health care system. \nP T. \n2018;43:\n293\u2013300."},{"key":"i2589-9449-7-2-59-b11","series-title":"Adv Ther","first-page":"1200","article-title":"Efficacy of lomitapide in the treatment of familial homozygous hypercholesterolemia: results of a real-world clinical experience in Italy","volume":"34","author":"D\u2019Erasmo","year":"2017","unstructured":"D\u2019Erasmo\n              L,\n            \n            \n              Cefal\u00f9\n              AB,\n            \n            \n              Noto\n              D,\n            \n            et al.\n\n          Efficacy of lomitapide in the treatment of familial homozygous hypercholesterolemia: results of a real-world clinical experience in Italy. \nAdv Ther. \n2017;34:\n1200\u20131210."},{"key":"i2589-9449-7-2-59-b12","series-title":"Eur J Prev Cardiol","first-page":"832","article-title":"Efficacy and safety of lomitapide in homozygous familial hypercholesterolaemia: the pan-European retrospective observational study","volume":"29","author":"D\u2019Erasmo","year":"2022","unstructured":"D\u2019Erasmo\n              L,\n            \n            \n              Steward\n              K,\n            \n            \n              Cefal\u00f9\n              AB,\n            \n            et al.\n\n          Efficacy and safety of lomitapide in homozygous familial hypercholesterolaemia: the pan-European retrospective observational study. \nEur J Prev Cardiol. \n2022;29:\n832\u2013841. Erratum in: Eur J Prev Cardiol. 2022;29:1812."},{"key":"i2589-9449-7-2-59-b13","article-title":"Clinical Commissioning Policy: lomitapide for treating homozygous familial hypercholesterolaemia (adults)","author":"NHS England","year":"2002","unstructured":"NHS England\n          . \nClinical Commissioning Policy: lomitapide for treating homozygous familial hypercholesterolaemia (adults). 2002. \nAccessed Oct 3, 2022. www.england.nhs.uk\/wp-content\/uploads\/2018\/07\/1679-lomitapide.pdf"},{"key":"i2589-9449-7-2-59-b14","article-title":"CDEC final recommendation - lomitapide","author":"Cadth","year":"2022","unstructured":"Cadth\n          . \nCDEC final recommendation - lomitapide. 2022. Accessed Oct 3, 2022. www.cadth.ca\/sites\/default\/files\/cdr\/complete\/cdr_complete_SR0386_Juxtapid-Apr-21_15.pdf"},{"key":"i2589-9449-7-2-59-b01","article-title":"UpToDate. Familial hypercholesterolemia in adults: overview","author":"Rosenson","year":"2020","unstructured":"Rosenson\n              R,\n            \n            \n              Durrington,\n              P.\n            \n          \n          UpToDate. Familial hypercholesterolemia in adults: overview. 2020. \nAccessed Oct 3, 2022. www.uptodate.com\/contents\/familial-hypercholesterolemia-in-adults-overview"},{"key":"i2589-9449-7-2-59-b02","series-title":"Eur Heart J","first-page":"560","article-title":"Homozygous autosomal dominant hypercholesterolaemia in the Netherlands: prevalence, genotype-phenotype relationship, and clinical outcome","volume":"36","author":"Sjouke","year":"2015","unstructured":"Sjouke\n              B,\n            \n            \n              Kusters\n              DM,\n            \n            \n              Kindt\n              I,\n            \n            et al.\n\n          Homozygous autosomal dominant hypercholesterolaemia in the Netherlands: prevalence, genotype-phenotype relationship, and clinical outcome. \nEur Heart J. \n2015;36:\n560\u2013565."},{"key":"i2589-9449-7-2-59-b03","series-title":"Adv Ther","first-page":"2159","article-title":"Homozygous familial hypercholesterolemia (HoFH) in Saudi Arabia and two cases of lomitapide use in a real-world setting","volume":"38","author":"Mahzari","year":"2021","unstructured":"Mahzari\n              M,\n            \n            \n              Zarif\n              H.\n            \n          \n          Homozygous familial hypercholesterolemia (HoFH) in Saudi Arabia and two cases of lomitapide use in a real-world setting. \nAdv Ther. \n2021;38:\n2159\u20132169."},{"key":"i2589-9449-7-2-59-b04","series-title":"Lancet","first-page":"719","article-title":"Worldwide experience of homozygous familial hypercholesterolaemia: retrospective cohort study","volume":"399","author":"Tromp","year":"2022","unstructured":"Tromp\n              TR,\n            \n            \n              Hartgers\n              ML,\n            \n            \n              Hovingh\n              GK,\n            \n            et al.\n\n          Worldwide experience of homozygous familial hypercholesterolaemia: retrospective cohort study. \nLancet. \n2022;399:\n719\u2013728."},{"key":"i2589-9449-7-2-59-b05","article-title":"Highlights of prescribing information, Juxtapid","author":"U.S. Food & Drug Adminstration.","year":"2022","unstructured":"U.S. Food & Drug Adminstration.\n          \n          Highlights of prescribing information, Juxtapid. 2022. \nAccessed Oct 3, 2022. www.accessdata.fda.gov\/drugsatfda_docs\/label\/2012\/203858s000lbl.pdf"},{"key":"i2589-9449-7-2-59-b06","series-title":"Lancet","first-page":"40","article-title":"Efficacy and safety of a microsomal triglyceride transfer protein inhibitor in patients with homozygous familial hypercholesterolaemia: a single-arm, open-label, phase 3 study","volume":"381","author":"Cuchel","year":"2013","unstructured":"Cuchel\n              M,\n            \n            \n              Meagher\n              EA,\n            \n            \n              du Toit Theron\n              H,\n            \n            et al.\n\n          Efficacy and safety of a microsomal triglyceride transfer protein inhibitor in patients with homozygous familial hypercholesterolaemia: a single-arm, open-label, phase 3 study. \nLancet. \n2013;381:\n40\u201346."},{"key":"i2589-9449-7-2-59-b07","article-title":"Drug list","author":"Saudi Food and Drug Authority","year":"2022","unstructured":"Saudi Food and Drug Authority\n          . \nDrug list. 2022. Accessed Oct 3, 2022. www.sfda.gov.sa\/en\/drugs-list?TradeName=&ScientificName=lomitapide&Agent=&ManufacturerName=&RegNo=&pg=1"},{"key":"i2589-9449-7-2-59-b08","article-title":"Ministry of National Guard Health Affairs website","author":"King Abdulaziz Medical City in Riyadh","unstructured":"King Abdulaziz Medical City in Riyadh\n          . \nMinistry of National Guard Health Affairs website. Accessed Oct 3, 2023. ngha.med.sa\/English\/MedicalCities\/Pages\/default.aspx"},{"key":"i2589-9449-7-2-59-b09","series-title":"Glob J Qual Safe Healthc","first-page":"55","article-title":"Pharmacy and therapeutics committee preparedness plan for COVID-19","volume":"3","author":"Abu Esba","year":"2020","unstructured":"Abu Esba\n              L,\n            \n            \n              Al-Abdulkarim\n              H,\n            \n            \n              Alrushidan\n              A,\n            \n            \n              Al Harbi\n              M.\n            \n          \n          Pharmacy and therapeutics committee preparedness plan for COVID-19. \nGlob J Qual Safe Healthc. \n2020;3:\n55\u201364."},{"key":"i2589-9449-7-2-59-b10","series-title":"P T","first-page":"293","article-title":"P&T committee drug prioritization criteria: a tool developed by a Saudi health care system","volume":"43","author":"Esba","year":"2018","unstructured":"Esba\n              LCA,\n            \n            \n              Almodaimegh\n              H,\n            \n            \n              Alhammad\n              A,\n            \n            \n              Ferwana\n              M,\n            \n            \n              Yousef\n              C,\n            \n            \n              Ismail\n              S.\n            \n          \n          P&T committee drug prioritization criteria: a tool developed by a Saudi health care system. \nP T. \n2018;43:\n293\u2013300."},{"key":"i2589-9449-7-2-59-b11","series-title":"Adv Ther","first-page":"1200","article-title":"Efficacy of lomitapide in the treatment of familial homozygous hypercholesterolemia: results of a real-world clinical experience in Italy","volume":"34","author":"D\u2019Erasmo","year":"2017","unstructured":"D\u2019Erasmo\n              L,\n            \n            \n              Cefal\u00f9\n              AB,\n            \n            \n              Noto\n              D,\n            \n            et al.\n\n          Efficacy of lomitapide in the treatment of familial homozygous hypercholesterolemia: results of a real-world clinical experience in Italy. \nAdv Ther. \n2017;34:\n1200\u20131210."},{"key":"i2589-9449-7-2-59-b12","series-title":"Eur J Prev Cardiol","first-page":"832","article-title":"Efficacy and safety of lomitapide in homozygous familial hypercholesterolaemia: the pan-European retrospective observational study","volume":"29","author":"D\u2019Erasmo","year":"2022","unstructured":"D\u2019Erasmo\n              L,\n            \n            \n              Steward\n              K,\n            \n            \n              Cefal\u00f9\n              AB,\n            \n            et al.\n\n          Efficacy and safety of lomitapide in homozygous familial hypercholesterolaemia: the pan-European retrospective observational study. \nEur J Prev Cardiol. \n2022;29:\n832\u2013841. Erratum in: Eur J Prev Cardiol. 2022;29:1812."},{"key":"i2589-9449-7-2-59-b13","article-title":"Clinical Commissioning Policy: lomitapide for treating homozygous familial hypercholesterolaemia (adults)","author":"NHS England","year":"2002","unstructured":"NHS England\n          . \nClinical Commissioning Policy: lomitapide for treating homozygous familial hypercholesterolaemia (adults). 2002. \nAccessed Oct 3, 2022. www.england.nhs.uk\/wp-content\/uploads\/2018\/07\/1679-lomitapide.pdf"},{"key":"i2589-9449-7-2-59-b14","article-title":"CDEC final recommendation - lomitapide","author":"Cadth","year":"2022","unstructured":"Cadth\n          . \nCDEC final recommendation - lomitapide. 2022. Accessed Oct 3, 2022. www.cadth.ca\/sites\/default\/files\/cdr\/complete\/cdr_complete_SR0386_Juxtapid-Apr-21_15.pdf"}],"container-title":["Global Journal on Quality and Safety in Healthcare"],"original-title":[],"link":[{"URL":"https:\/\/innovationsjournals-jqsh.kglmeridian.com\/view\/journals\/jqsh\/7\/2\/article-p59.xml","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/meridian.allenpress.com\/innovationsjournals-JQSH\/article-pdf\/7\/2\/59\/3368532\/i2589-9449-7-2-59.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"syndication"},{"URL":"https:\/\/innovationsjournals-jqsh.kglmeridian.com\/downloadpdf\/journals\/jqsh\/7\/2\/article-p59.xml","content-type":"text\/html","content-version":"vor","intended-application":"syndication"},{"URL":"https:\/\/innovationsjournals-jqsh.kglmeridian.com\/downloadpdf\/journals\/jqsh\/7\/2\/article-p59.xml","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,2,11]],"date-time":"2026-02-11T00:55:04Z","timestamp":1770771304000},"score":1,"resource":{"primary":{"URL":"https:\/\/innovationsjournals-jqsh.kglmeridian.com\/view\/journals\/jqsh\/7\/2\/article-p59.xml"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,5]]},"references-count":28,"journal-issue":{"issue":"2"},"URL":"https:\/\/doi.org\/10.36401\/jqsh-23-32","relation":{"has-preprint":[{"id-type":"doi","id":"10.21203\/rs.3.rs-2275638\/v1","asserted-by":"object"}]},"ISSN":["2589-9449","2666-2353"],"issn-type":[{"value":"2589-9449","type":"electronic"},{"value":"2666-2353","type":"print"}],"subject":[],"published":{"date-parts":[[2024,5]]}}}