ArticleIntractable & rare diseases research2020
Cost-effectiveness analysis of enzyme replacement therapy (ERT) for treatment of infantile-onset Pompe disease (IOPD) in the Iranian pharmaceutical market.
Article in Intractable & rare diseases research, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
2 citing papers in PubMed, 2 citations in OpenAlex.
- The cost-effectiveness of enzyme replacement therapies versus best supportive care for treating late onset Pompe disease in the UK NHS.The European journal of health economics : HEPAC : health economics in prevention and care · 2026Article
- Cost-Effectiveness of Newborn Screening for Infantile-Onset Pompe Disease in Japan.International journal of neonatal screening · 2026Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors at 3 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
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.
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Registered trials
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