Economic Burden of Gaucher Disease: A Systematic Literature Review
Author(s)
Aikyadeep Mandal, M.Pharm, Taruni Priya Donkana, Pharm D, Jyoti Garg, M.Pharm, Amit Ahuja, M.Pharm.
Lumanity, Gurugram, India.
Lumanity, Gurugram, India.
OBJECTIVES: Gaucher disease (GD) is a rare inherited lysosomal storage disorder causing fatty substance accumulation in organs, leading to enlarged spleen and liver, bone pain, fractures, fatigue, bruising and sometimes nervous system involvement. This review summarizes the available evidence on its economic burden.
METHODS: Embase® and Medline® were searched to identify relevant English-language articles published after 2015 reporting data for the economic burden in paediatric and adult patients with GD.
RESULTS: Of the 578 records reviewed, 12 unique studies were included. Three studies reported US data, two for China and one each for Germany, Brazil, Colombia, India, Iran, Greece and Russia. In the US, the total annual direct costs per patient in 2021 ranged from $243,281 to $746,747, with treatment using taliglucerase alfa in adults having the highest financial impact. Germany’s annual treatment cost was €471,449, with 99% attributed to infusion-related expenses. Annual direct cost of treatment in China was $46,790.60 in 2018, with pharmaceutical treatment costing $29,907.60. In Greece, the average annual per patient costs were €137,074 in 2023, with drug costs accounting for 88% of the total. The average annual cost of treatment per patient was $125,649 in Colombia in 2023 and $19,763 in Iran in 2017. In India, the total cost for 30 clinic patients was ₹254,574,743 ($3,440,199.20). Among these, 14 patients treated with enzyme replacement therapy or substrate reduction therapy incurred ₹252,967,279, while 16 treatment-naïve patients incurred ₹1,615,064. Resource utilization data in China showed an average of 8.8 outpatient and 4.0 inpatient visits per year, while a US study found that only 0.3% of patients required hospitalization.
CONCLUSIONS: GD imposes a substantial economic burden worldwide, with significant variation in treatment costs across countries. Our findings emphasize the need for cost-effective management strategies and further research to reduce the financial impact on healthcare systems and patients globally.
METHODS: Embase® and Medline® were searched to identify relevant English-language articles published after 2015 reporting data for the economic burden in paediatric and adult patients with GD.
RESULTS: Of the 578 records reviewed, 12 unique studies were included. Three studies reported US data, two for China and one each for Germany, Brazil, Colombia, India, Iran, Greece and Russia. In the US, the total annual direct costs per patient in 2021 ranged from $243,281 to $746,747, with treatment using taliglucerase alfa in adults having the highest financial impact. Germany’s annual treatment cost was €471,449, with 99% attributed to infusion-related expenses. Annual direct cost of treatment in China was $46,790.60 in 2018, with pharmaceutical treatment costing $29,907.60. In Greece, the average annual per patient costs were €137,074 in 2023, with drug costs accounting for 88% of the total. The average annual cost of treatment per patient was $125,649 in Colombia in 2023 and $19,763 in Iran in 2017. In India, the total cost for 30 clinic patients was ₹254,574,743 ($3,440,199.20). Among these, 14 patients treated with enzyme replacement therapy or substrate reduction therapy incurred ₹252,967,279, while 16 treatment-naïve patients incurred ₹1,615,064. Resource utilization data in China showed an average of 8.8 outpatient and 4.0 inpatient visits per year, while a US study found that only 0.3% of patients required hospitalization.
CONCLUSIONS: GD imposes a substantial economic burden worldwide, with significant variation in treatment costs across countries. Our findings emphasize the need for cost-effective management strategies and further research to reduce the financial impact on healthcare systems and patients globally.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
EE360
Topic
Economic Evaluation, Study Approaches
Topic Subcategory
Cost/Cost of Illness/Resource Use Studies, Work & Home Productivity - Indirect Costs
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, Rare & Orphan Diseases