A systematic review of the economic evaluations of Enzyme Replacement Therapy in Lysosomal Storage Diseases

Cost Effectiveness and Resource Allocation

Review

Panagiotis Petrou¹  Eleni Ioanna Katsigianni¹ 

[1] Pharmacy School, Department of Life &Health Sciences, School of Sciences and Engineering, University of Nicosia, 2417, Nicosia, Cyprus;

关键词: Cost-effectiveness;    Enzyme replacement therapy (ERT);    Gaucher disease;    Fabry disease;    Pompe disease;    Lysosomal acid lipase (LAL) deficiency;    Lysosomal storage diseases (LSD);    Cost;    Quality of life;   

DOI  :  10.1186/s12962-022-00369-w

 received in 2022-04-30, accepted in 2022-07-05,  发布年份 2022

来源: Springer

【 摘 要 】

ObjectiveThe objective of this paper is to assess the economic profile of Enzyme Replacement Therapy (ERT) to symptomatic patients with Pompe, Fabry, Gaucher disease and Lysosomal acid lipase (LAL) deficiency.MethodsA systematic search was performed to retrieve and critically assess economic evaluations of enzyme replacement therapy. Publications were screened according to predefined criteria and evaluated according to the Quality of Economic Studies. Data were narratively synthesized.ResultsThe Incremental Cost-Effectiveness Ratio greatly exceeded willingness to pay thresholds. The cost of the medication dominated the sensitivity analysis. For Infantile-onset Pompe’s disease, the incremental cost-effectiveness ratio (ICER) was estimated at €1.043.868 per Quality-adjusted life year (QALY) based on the dose of alglucosidase 40 mg/kg/ week, and €286.114 per QALY for 20 mg of alglucosidase/kg/2 weeks. For adults patients presenting with Pompe disease the reported was ICER € 1.8 million/ QALY. In the case of Fabry disease, the ICER per QALY amounts to 6.1 million Euros/QALY. Respectively for Gaucher’s disease, the ICER /QALY was estimated at € 884,994 per QALY. Finally, for patients presenting LAL deficiency NCPE perpetuated an ICER of €2,701,000/QALY.DiscussionERT comprise a promising treatment modality for orphan diseases; nevertheless, it is interlaced with a substantial economic burden. Moreover, the available data on the cost-effectiveness ratio are scarce. For certain diseases, such as Fabry, a thorough selection of patients could exert a beneficial effect on the reported ICER. Steep price reductions are imperative for these products, in the conventional reimbursement pathway or a new assessment framework should be elaborated, which in principle, should target uncertainty.

【 授权许可】

CC BY   
© The Author(s) 2022. corrected publication 2022

【 图 表 】

Fig. 6