Cost-Effectiveness Analysis of Enzyme Replacement Therapy (Ert) for Treatment of Infantile-Onset Pompe Disease (Iopd) in the Iranian Pharmaceutical Market

Cost-Effectiveness Analysis of Enzyme Replacement Therapy (Ert) for Treatment of Infantile-Onset Pompe Disease (Iopd) in the Iranian Pharmaceutical Market Publisher

Hashempour R¹ ; Davari M^{2, 3} ; Pourreza A¹ ; Alaei M⁴ ; Ahmadi B¹

Show Affiliations

1. Department of Health Management and Economics, School of Public Health, Tehran University of Medical Science, Tehran, Iran
2. Department of Pharmacoeconomics and Pharmaceutical Administration, Faculty of Pharmacy, Tehran University of Medical Science, Tehran, Iran
3. Pharmaceutical Management and Economics Research Center, Tehran University of Medical Science, Tehran, Iran
4. Department of Pediatric Endocrinology, Faculty of Medicine, Shahid Beheshti University of Medical Sciences, Tehran, Iran

Source: Intractable and Rare Diseases Research Published:2020

Abstract

Infantile-onset Pompe disease (IOPD) or acid maltase deficiency is a rare metabolic disorder. It is caused by a deficiency in functioning of the enzyme acid alpha-glucosidase and leads to the accumulation of glycogen in the liver, heart, muscle, and other tissues. Myozyme is an effective drug, but it imposes a heavy financial burden on societies and healthcare systems. Therefore, this study was conducted to analyze the cost-effectiveness of Myozyme compared to conventional therapy for the treatment of IOPD. PubMed, Scopus, Web of Science, and Cochrane library databases were searched on December 2018 to identify the effectiveness of Myozyme versus conventional therapy. Then, a cost-effectiveness and a cost utility study were conducted in patients suffering from IOPD. In this cost effectiveness and cost utility analysis, Markov and decision tree models were used for modeling. Model parameters were obtained from international data, and the perspective of the payer was considered. Every cycle was one year; the model was run for 22 cycles. TreeAge pro 2011 was used for analysis. Finally, one-way and probabilistic sensitivity analyses were performed. Two papers were included and 39 patients were evaluated as the treatment group in both studies. Results revealed the effectiveness of Myozyme. Results also revealed a wide range of adverse reactions. Enzyme replacement therapy (ERT) resulted in 4.21038 quality-adjusted life years (QALY) per $381,852. The incremental cost per QALY was $96,809 and the incremental cost per life years gained (LYG) was 74,429 over a 22-year time horizon. Sensitivity analysis indicated the robustness of the results. Myozyme is effective for IOPD and could increase the life expectancy of patients significantly. However, since the calculated incremental cost per QALY was 17 times higher than the GDP per capita of Iran, Myozyme is not cost effective in Iran. © 2020 International Advancement Center for Medicine and Health Research. All rights reserved.

2. Diagnosis and Treatment of Late-Onset Pompe Disease in the Middle East and North Africa Region: Consensus Recommendations From an Expert Group, BMC Neurology (2015)

3. Qaly League Table of Iran: A Practical Method for Better Resource Allocation, Cost Effectiveness and Resource Allocation (2021)

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Style	Citing Format
MLA	Hashempour R, et al.. "Cost-Effectiveness Analysis of Enzyme Replacement Therapy (Ert) for Treatment of Infantile-Onset Pompe Disease (Iopd) in the Iranian Pharmaceutical Market." Intractable and Rare Diseases Research, vol. 9, no. 3, 2020, pp. 130-136.
APA	Hashempour R, Davari M, Pourreza A, Alaei M, Ahmadi B (2020). Cost-Effectiveness Analysis of Enzyme Replacement Therapy (Ert) for Treatment of Infantile-Onset Pompe Disease (Iopd) in the Iranian Pharmaceutical Market. Intractable and Rare Diseases Research, 9(3), 130-136.
Chicago	Hashempour R, Davari M, Pourreza A, Alaei M, Ahmadi B. "Cost-Effectiveness Analysis of Enzyme Replacement Therapy (Ert) for Treatment of Infantile-Onset Pompe Disease (Iopd) in the Iranian Pharmaceutical Market." Intractable and Rare Diseases Research 9, no. 3 (2020): 130-136.
Harvard	Hashempour R et al. (2020) 'Cost-Effectiveness Analysis of Enzyme Replacement Therapy (Ert) for Treatment of Infantile-Onset Pompe Disease (Iopd) in the Iranian Pharmaceutical Market', Intractable and Rare Diseases Research, 9(3), pp. 130-136.
Vancouver	Hashempour R, Davari M, Pourreza A, Alaei M, Ahmadi B. Cost-Effectiveness Analysis of Enzyme Replacement Therapy (Ert) for Treatment of Infantile-Onset Pompe Disease (Iopd) in the Iranian Pharmaceutical Market. Intractable and Rare Diseases Research. 2020;9(3):130-136.
BibTex	@article{ author = {Hashempour R and Davari M and Pourreza A and Alaei M and Ahmadi B}, title = {Cost-Effectiveness Analysis of Enzyme Replacement Therapy (Ert) for Treatment of Infantile-Onset Pompe Disease (Iopd) in the Iranian Pharmaceutical Market}, journal = {Intractable and Rare Diseases Research}, volume = {9}, number = {3}, pages = {130-136}, year = {2020} }
RIS	TY - JOUR AU - Hashempour R AU - Davari M AU - Pourreza A AU - Alaei M AU - Ahmadi B TI - Cost-Effectiveness Analysis of Enzyme Replacement Therapy (Ert) for Treatment of Infantile-Onset Pompe Disease (Iopd) in the Iranian Pharmaceutical Market JO - Intractable and Rare Diseases Research VL - 9 IS - 3 SP - 130 EP - 136 PY - 2020 ER -

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