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

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.

METHODS

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.

RESULTS

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.

CONCLUSIONS

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.

Informations de copyright

Copyright © 2022. Published by Elsevier B.V.

Déclaration de conflit d'intérêts

Declaration of competing interest The authors declare the following financial interests/personal relationships which may be considered as potential competing interests: Rita Faria declares that, since the research was completed, she has become an employee of Astellas Pharma Europe Ltd. Stephen Weng was part of an institution that received grants from the NIHR SPCR for research related to Familial Hypercholesterolaemia, consulting fees from his Academic Advisory Committee for Road to Health Ltd, Honoraria and travel fees from Amgen for lectures on familial hypercholesterolaemia, was previously a committee member for the MHRA CPRD Independent Scientific Advisory Committee and is currently employed by Janssen R&D. Steve Humphries has received Support from the British Heart Foundation (PG 008/08) and is the director of the UK Paediatric FH Register which has received support from a grant from the International Atherosclerosis Society (Pfizer number 24052829) and a medical director and minor share holder of a UCL Spin-out company StoreGene which offers DNA testing for individuals with FH. Nadeem Qureshi has received grants from NIHR SPCR and MRC (NUOF), Honoraria from Amgen for lectures on familial hypercholesterolaemia, is a Member of the Board for the NIHR School for Primary Care Research (2021-) and a Member of Medical, Scientific & Research Committee of HeartUK. Edward Cox, Pedro Saramago Goncalves, Ralph Akyea, Barbara Iyen and Beth Woods have no conflicts of interest to declare.