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How does cholesterol burden change the case for investing in familial hypercholesterolaemia? A cost-effectiveness analysis

Faria, Rita; Saramago, Pedro; Cox, Edward; Weng, Stephen; Iyen, Barbara; Akyea, Ralph K; Humphries, Steve E; Qureshi, Nadeem; Woods, Beth


Rita Faria

Pedro Saramago

Edward Cox

Stephen Weng

Clinical Assistant Professor in Primary Care

Steve E Humphries

Beth Woods


Background and aims
This study aimed to ascertain how the long-term benefits and costs of diagnosis and treatment of familial hypercholesterolaemia (FH) vary by prognostic factors and ‘cholesterol burden’, which is the effect of long-term exposure to low-density lipoprotein cholesterol (LDL-C) on cardiovascular disease (CVD) risk.

A new cost-effectiveness model was developed from the perspective of the UK National Health Service (NHS), informed by routine data from individuals with FH. The primary outcome was net health gain (i.e., health benefits net of the losses due to costs), expressed in quality-adjusted life years (QALYs) at the £15,000/QALY threshold. Prognostic factors included pre-treatment LDL-C, age, gender, and CVD history.

If cholesterol burden is considered, diagnosis resulted in positive net health gain (i.e., it is cost-effective) in all individuals with pre-treatment LDL-C ≥ 4 mmol/L, and in those with pre-treatment LDL-C ≥ 2 mmol/L aged ≥50 years or who have CVD history. If cholesterol burden is not considered, diagnosis resulted in lower net health gain, but still positive in children aged 10 years with pre-treatment LDL-C ≥ 6 mmol/L and adults aged 30 years with pre-treatment LDL-C ≥ 4 mmol/L.

Diagnosis and treatment of most people with FH results in large net health gains, particularly in those with higher pre-treatment LDL-C. Economic evaluations of FH interventions should consider the sensitivity of the study conclusions to cholesterol burden, particularly where interventions target younger patients, and explicitly consider prognostic factors such as pre-treatment LDL-C, age, and CVD history.


Faria, R., Saramago, P., Cox, E., Weng, S., Iyen, B., Akyea, R. K., …Woods, B. (2023). How does cholesterol burden change the case for investing in familial hypercholesterolaemia? A cost-effectiveness analysis. Atherosclerosis, 367, 40-47.

Journal Article Type Article
Acceptance Date Dec 6, 2022
Online Publication Date Dec 29, 2022
Publication Date 2023-02
Deposit Date Dec 15, 2022
Publicly Available Date Dec 29, 2022
Journal Atherosclerosis
Print ISSN 0021-9150
Electronic ISSN 1879-1484
Publisher Elsevier
Peer Reviewed Peer Reviewed
Volume 367
Pages 40-47
Public URL
Publisher URL


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