Assessing the Value for Money of Enzyme Replacement Therapy in Gaucher Disease Types 1 and 3b: Can Expanded Coverage Be Justified?

Waranya Rattanavipapong (), Thunyarat Anothaisintawee, Wanrudee Isaranuwatchai, Duangrurdee Wattanasirichaigoon, Thipwimol Tim-Aroon, Khunton Wichajarn, Achara Sathienkijkanchai, Pimlak Charoenkwan, Kanya Suphapeetiporn, Chanchai Traivaree, Chulaluck Kuptanon and Yot Teerawattananon
Additional contact information
Waranya Rattanavipapong: Ministry of Public Health
Thunyarat Anothaisintawee: Mahidol University
Wanrudee Isaranuwatchai: Ministry of Public Health
Duangrurdee Wattanasirichaigoon: Mahidol University
Thipwimol Tim-Aroon: Mahidol University
Khunton Wichajarn: Khon Kaen University
Achara Sathienkijkanchai: Mahidol University
Pimlak Charoenkwan: Chiang Mai University
Kanya Suphapeetiporn: Chulalongkorn University
Chanchai Traivaree: Phramongkutklao Hospital and Phramongkutklao College of Medicine
Chulaluck Kuptanon: Ministry of Public Health
Yot Teerawattananon: Ministry of Public Health

PharmacoEconomics - Open, 2025, vol. 9, issue 4, No 7, 626 pages

Abstract: Abstract Background and Objectives The Health Intervention and Technology Assessment Program was commissioned to conduct a cost–utility and budget impact analysis of enzyme replacement therapy (ERT) for Gaucher disease types 1 and 3b. The findings from this assessment are to support the decision-making process regarding the potential expansion of ERT coverage within Thailand’s public health system. Methods The analysis compared the current policy, which provides treatment with imiglucerase only for patients with Gaucher disease type 1, as listed in the National List of Essential Medicine, with a proposed policy that extends coverage to include Gaucher disease types 1 and 3b with either imiglucerase or velaglucerase. Cost–utility analysis of these policy options was performed using decision tree and Markov models over a lifetime horizon from a societal perspective. The financial implications for the relevant budgetary authority over 5 years were estimated. The research methodology adheres rigorously to Thailand’s health technology assessment guidelines. Results The study found that the incremental cost-effectiveness ratios for treating both Gaucher disease types 1 and 3b are 6,769,000 and 9,359,000 baht per quality-adjusted life year (QALY) for imiglucerase and velaglucerase, respectively, which is well beyond Thailand’s cost-effectiveness threshold of 160,000 baht per QALY. Such an expansion would incur an additional budgetary burden of approximately 81 million baht for imiglucerase and 138 million baht for velaglucerase. Increasing the rate of hematopoietic stem cell transplantation (HSCT) can improve the cost-effectiveness of the expansion. Conclusions The study concludes that expanding ERT with either imiglucerase or velaglucerase to treat both Gaucher disease types 1 and 3b is not cost-effective at current prices in Thailand; however, it could become cost-effective with a reduction of approximately 60% in drug prices or if all eligible patients undergo HSCT.

Date: 2025
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DOI: 10.1007/s41669-025-00579-x

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