Willemijn A M Schonck, Janneke W C M Mulder, Tycho R Tromp, Laurens F Reeskamp, G Kees Hovingh, Jeanine E Roeters van Lennep, Dirk J Blom
{"title":"Global disparities in access to lipid-lowering therapies for patients with homozygous familial hypercholesterolemia - A physician survey.","authors":"Willemijn A M Schonck, Janneke W C M Mulder, Tycho R Tromp, Laurens F Reeskamp, G Kees Hovingh, Jeanine E Roeters van Lennep, Dirk J Blom","doi":"10.1016/j.jacl.2025.05.003","DOIUrl":null,"url":null,"abstract":"<p><strong>Background: </strong>Lipid levels and atherosclerotic cardiovascular disease (ASCVD) outcomes have been shown to differ globally in patients with homozygous familial hypercholesterolemia (HoFH), which may be related to availability and accessibility of lipid-lowering therapy (LLT).</p><p><strong>Objective: </strong>In the current study, we investigated global disparities in availability and accessibility of LLTs for patients with HoFH.</p><p><strong>Methods: </strong>Physicians participating in the HoFH International Clinical Collaborators (HICC, NCT04815005) were invited to complete an online survey on registration status, reimbursement, and access to various LLTs. Responses were compared between high-income and nonhigh-income countries.</p><p><strong>Results: </strong>Responses were received from 87 physicians (64.4% from high-income countries). Physicians from high-income countries reported significantly higher registration rates for proprotein convertase subtilisin/kexin type 9 monoclonal antibodies (PCSK9 mAbs) (96.4% vs 51.6%), lomitapide (83.6% vs 9.7%), evinacumab (69.1% vs 0.0%), colesevelam (50.0% vs 3.2%), and lipoprotein-apheresis (96.4% vs 45.2%). Public sector reimbursement was also more common in high-income countries for PCSK9 mAbs (90.9% vs 24.1%), lomitapide (74.5% vs 3.4%), evinacumab (60.0% vs 0.0%), colesevelam (40.0% vs 3.4%), and lipoprotein-apheresis (94.5% vs 37.9%). Access to LLTs was also higher in high-income countries for statins (91.1% vs 61.3%), ezetimibe (87.5% vs 38.7%), PCSK9 mAbs (53.6% vs 6.5%), lomitapide (32.% vs 0.0%), evinacumab (32.1% vs 3.2%), colesevelam (39.3% vs 3.2%), and lipoprotein-apheresis (57.1% vs 3.2%).</p><p><strong>Conclusion: </strong>Our results confirm significant global disparities in LLT registration, reimbursement, and access between high-income and nonhigh-income countries. Improving LLT availability and accessibility, particularly in nonhigh-income countries, is essential to improve outcomes in patients with HoFH.</p>","PeriodicalId":15392,"journal":{"name":"Journal of clinical lipidology","volume":" ","pages":""},"PeriodicalIF":3.6000,"publicationDate":"2025-05-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Journal of clinical lipidology","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1016/j.jacl.2025.05.003","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"PHARMACOLOGY & PHARMACY","Score":null,"Total":0}
引用次数: 0
Abstract
Background: Lipid levels and atherosclerotic cardiovascular disease (ASCVD) outcomes have been shown to differ globally in patients with homozygous familial hypercholesterolemia (HoFH), which may be related to availability and accessibility of lipid-lowering therapy (LLT).
Objective: In the current study, we investigated global disparities in availability and accessibility of LLTs for patients with HoFH.
Methods: Physicians participating in the HoFH International Clinical Collaborators (HICC, NCT04815005) were invited to complete an online survey on registration status, reimbursement, and access to various LLTs. Responses were compared between high-income and nonhigh-income countries.
Results: Responses were received from 87 physicians (64.4% from high-income countries). Physicians from high-income countries reported significantly higher registration rates for proprotein convertase subtilisin/kexin type 9 monoclonal antibodies (PCSK9 mAbs) (96.4% vs 51.6%), lomitapide (83.6% vs 9.7%), evinacumab (69.1% vs 0.0%), colesevelam (50.0% vs 3.2%), and lipoprotein-apheresis (96.4% vs 45.2%). Public sector reimbursement was also more common in high-income countries for PCSK9 mAbs (90.9% vs 24.1%), lomitapide (74.5% vs 3.4%), evinacumab (60.0% vs 0.0%), colesevelam (40.0% vs 3.4%), and lipoprotein-apheresis (94.5% vs 37.9%). Access to LLTs was also higher in high-income countries for statins (91.1% vs 61.3%), ezetimibe (87.5% vs 38.7%), PCSK9 mAbs (53.6% vs 6.5%), lomitapide (32.% vs 0.0%), evinacumab (32.1% vs 3.2%), colesevelam (39.3% vs 3.2%), and lipoprotein-apheresis (57.1% vs 3.2%).
Conclusion: Our results confirm significant global disparities in LLT registration, reimbursement, and access between high-income and nonhigh-income countries. Improving LLT availability and accessibility, particularly in nonhigh-income countries, is essential to improve outcomes in patients with HoFH.
背景:纯合子家族性高胆固醇血症(HoFH)患者的脂质水平和动脉粥样硬化性心血管疾病(ASCVD)结局在全球范围内存在差异,这可能与降脂治疗(LLT)的可用性和可及性有关。目的:在当前的研究中,我们调查了HoFH患者可获得性和可及性llt的全球差异。方法:邀请参加HoFH国际临床合作者(HICC, NCT04815005)的医生完成一项关于注册状态、报销和各种llt访问的在线调查。对高收入国家和非高收入国家的反应进行了比较。结果:收到87名医生的回复(64.4%来自高收入国家)。来自高收入国家的医生报告了蛋白转化酶枯草杆菌素/ keexin 9型单克隆抗体(PCSK9单克隆抗体)的注册率(96.4%对51.6%),洛米他胺(83.6%对9.7%),依维纳单抗(69.1%对0.0%),colesevilam(50.0%对3.2%)和脂蛋白单采(96.4%对45.2%)。在高收入国家,公共部门对PCSK9单克隆抗体(90.9%对24.1%)、洛米他啶(74.5%对3.4%)、依维纳单抗(60.0%对0.0%)、colesevelam(40.0%对3.4%)和脂蛋白单采(94.5%对37.9%)的报销也更为普遍。在高收入国家,他汀类药物(91.1%对61.3%)、依zetimibe(87.5%对38.7%)、PCSK9单克隆抗体(53.6%对6.5%)、洛米他胺(32。% vs 0.0%), evinacumab (32.1% vs 3.2%), colesevelam (39.3% vs 3.2%)和脂蛋白单采(57.1% vs 3.2%)。结论:我们的研究结果证实了全球高收入和非高收入国家在LLT注册、报销和可及性方面存在显著差异。改善LLT的可获得性和可及性,特别是在非高收入国家,对于改善HoFH患者的预后至关重要。
期刊介绍:
Because the scope of clinical lipidology is broad, the topics addressed by the Journal are equally diverse. Typical articles explore lipidology as it is practiced in the treatment setting, recent developments in pharmacological research, reports of treatment and trials, case studies, the impact of lifestyle modification, and similar academic material of interest to the practitioner.
Sections of Journal of clinical lipidology will address pioneering studies and the clinicians who conduct them, case studies, ethical standards and conduct, professional guidance such as ATP and NCEP, editorial commentary, letters from readers, National Lipid Association (NLA) news and upcoming event information, as well as abstracts from the NLA annual scientific sessions and the scientific forums held by its chapters, when appropriate.