Cost-effectiveness of evolocumab in treatment of heterozygous familial hypercholesterolaemia in Bulgaria: measuring health benefit by effectively treated patient-years*

Abstract

Background: An elevated level of low-density lipoprotein cholesterol (LDL-C) constitutes one of the most important modifiable risk factors for cardiovascular disease (CVD). Individuals with heterozygous familial hypercholesterolaemia (HeFH) are particularly vulnerable to CVD events. The addition of evolocumab to statins has shown marked reductions in LDL-C levels. The objective of this analysis is to demonstrate the clinical and economic value of LDL-C lowering with evolocumab from the Bulgarian public health care perspective. Methods: A disease-specific measure of health benefit was devised: Effectively treated patient-years (ETPYs) combine length of life with the likelihood of attaining best-practice recommendations on LDL-C lowering. “Effective treatment” was defined as a reduction in LDL-C levels of ≥50%. A Markov cohort state-transition model was adapted, considering a life-long treatment duration. Demographics, baseline characteristics and efficacy data were taken from the RUTHERFORD-2 trial. The model uses the relationship between LDL-C lowering and reduced CVD event rates observed in the meta-analyses conducted by the Cholesterol Treatment Trialists’ Collaboration. Outcomes and costs (from year 2015) were discounted at an annual rate of 5%. Sensitivity analyses were conducted to assess uncertainty surrounding the results. Results: The total incremental costs of evolocumab added to statins versus statins alone are BGN 120,329 while adding 9.30 ETPYs over lifetime. These results imply an incremental cost per ETPY of BGN 12,937 (US$ 7,215; € 6,604). The use of evolocumab is associated with a relative reduction in the CVD event rate by 38% (18% per 1 mmol/L). Conclusions: Adding evolocumab to statins may be considered cost-effective in light of an additional expense per patient-year gained in which individuals with HeFH receive effective treatment under the terms of international prevention guidelines. ETPYs are an intuitive and clinically meaningful measure of patient benefit that, in relation to costs, can support health care decision-making that considers quality of care.

Cost-utility analysis of evolocumab in patients with ASCVD in Italy

“…Gli inibitori del PCSK9 rappresentano una nuova opportunità per i pazienti per i quali il livello di LDL-C non risulta adeguatamente controllato mediante la terapia con statine alla dose massimamente tollerata. In questo studio, si è tentato di adattare al contesto nazionale un modello economico precedentemente pubblicato (13,(25)(26)(27)(28)(29)(30)(31), basato sulla riduzione del rischio di eventi cardiovascolari associata a evolocumab, emersa dallo studio clinico FOURIER, al fine di indagare la costo-efficacia di evolocumab in pazienti con ASCVD clinicamente evidente nel contesto italiano. L'ICER stimato per evolocumab in aggiunta alla terapia standard per le popolazioni Recent MI, Multiple events e Multiple events MI < 1 anno è risultato inferiore rispetto a una soglia di accettabilità di costo-efficacia pari a € 40.000 (rispettivamente pari a € 39.547, a € 35.744 e a € 29.949, quando la terapia standard risulta caratterizzata da statine con o senza ezetimibe, e pari a € 39.781, a € 35.986 e a € 30.190, quando la terapia standard risulta caratterizzata da statine con ezetimibe).…”

“…Il modello è stato sviluppato adattando al contesto nazionale un modello di costo-utilità precedentemente pubblicato in letteratura (13,(25)(26)(27)(28)(29)(30)(31).…”

The Cost-Effectiveness of Hyperlipidemia Medication in Low- and Middle-Income Countries: A Review

“…Over half of the included studies (n = 13) come from upper middle-income countries [24][25][26][27][28][29][30][31][32][33][34][35][36], four are from lower middle-income countries [37][38][39][40], two are from low-income countries [41,42], and three contain countries of multiple income levels or conduct analysis at a regional level [43][44][45]. Studies come from every region except North America, with seven from Latin America and Caribbean [24,25,28,29,31,35,44], seven from East Asia and the Pacific [26,27,32,33,34,37], two from South Asia [38,40], two from Sub-Saharan Africa [41,42], one from Europe [30], one from Middle East [36], and two with countries from multiple regions [43,45]. These studies produce results for 14 individual countries: Argentina [25,31], Brazil [28,…”

“…Studies come from every region except North America, with seven from Latin America and Caribbean [24,25,28,29,31,35,44], seven from East Asia and the Pacific [26,27,32,33,34,37], two from South Asia [38,40], two from Sub-Saharan Africa [41,42], one from Europe [30], one from Middle East [36], and two with countries from multiple regions [43,45]. These studies produce results for 14 individual countries: Argentina [25,31], Brazil [28,29], Bulgaria [30], Colombia [28], China [32,34,45], Ethiopia [42], India [38,40,45], Iran [36], Mexico [24,35,45], Ghana [45], Philippines [39], South Africa [45], Tanzania [41], Thailand [26,27,33], and Vietnam [37]. Supplementary Table 3 summarizes the study characteristics with details about intervention type, treatment setting, outcomes reported,...…”

Cost-Effectiveness Analysis of Evolocumab for the Treatment of Dyslipidemia in the Kingdom of Saudi Arabia

“…A previously published Markov state transition model was adapted for the Saudi public healthcare setting to estimate the cost effectiveness of evolocumab in lowering LDL-C for patients with ASCVD or HeFH [18,[24][25][26][27][28]. The model was populated with local inputs for baseline patient characteristics, background therapy, event rates, background mortality, and costs.…”