Key result
High-income countries had significantly higher registration rates for PCSK9 monoclonal antibodies (96.4% vs 51.6%, p<0.001) and other novel lipid-lowering therapies compared to non-high-income countries.
Why the study?
Lipid levels and ASCVD outcomes differ globally in patients with HoFH, potentially due to disparities in availability and accessibility of lipid-lowering therapies.
Does residing in a high-income country improve access to and reimbursement for novel lipid-lowering therapies for patients with homozygous familial hypercholesterolemia?
Cross-Sectional (n=87)
Yes
Does residing in a high-income country improve access to and reimbursement for novel lipid-lowering therapies for patients with homozygous familial hypercholesterolemia?
Absolute Event Rate: 96.4% vs 51.6%
p-value: p=<0.001
There are profound global disparities in access to advanced lipid-lowering therapies for HoFH, with patients in non-high-income countries facing severe barriers to guideline-directed novel treatments.
Disparities limit novel therapy access for HoFH in non-high-income countries; leaves open whether registration gaps affect cardiovascular outcomes.
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 non-high-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 non-high-income countries. Improving LLT availability and accessibility, particularly in non-high-income countries, is essential to improve outcomes in patients with HoFH.
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Schonck et al. (2025) conducted a cross-sectional in Homozygous familial hypercholesterolemia (n=87). High-income country practice setting vs. Non-high-income country practice setting was evaluated on Registration rates of PCSK9 monoclonal antibodies (p=<0.001). High-income countries had significantly higher registration rates for PCSK9 monoclonal antibodies (96.4% vs 51.6%, p<0.001) and other novel lipid-lowering therapies compared to non-high-income countries.
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