Assessing the Value for Money of Enzyme Replacement Therapy in Gaucher Disease Types 1 and 3b: Can Expanded Coverage Be Justified?
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.
Authors
- Kanya Suphapeetiporn (ORCID: https://orcid.org/0000-0001-5679-7547)
- Waranya Rattanavipapong (ORCID: https://orcid.org/0000-0001-8628-2149)
- Thunyarat Anothaisintawee (ORCID: https://orcid.org/0000-0003-1002-8536)
- Chulaluck Kuptanon (ORCID: https://orcid.org/0000-0003-4043-3794)
- Duangrurdee Wattanasirichaigoon (ORCID: https://orcid.org/0000-0003-2329-2176)
- Chanchai Traivaree (ORCID: https://orcid.org/0000-0001-5635-1238)
- Achara Sathienkijkanchai (ORCID: https://orcid.org/0000-0001-8275-9616)
- Wanrudee Isaranuwatchai (ORCID: https://orcid.org/0000-0002-8368-6065)
- Khunton Wichajarn (ORCID: https://orcid.org/0000-0003-4324-7487)
- Thipwimol Tim‐Aroon (ORCID: https://orcid.org/0000-0001-8044-7993)
- Pimlak Charoenkwan (ORCID: https://orcid.org/0000-0002-9123-9177)
- Yot Teerawattananon (ORCID: https://orcid.org/0000-0003-2217-2930)
Institutions
- Ministry of Public Health (TH)
- Siriraj Hospital (TH)
- Chulalongkorn University (TH)
- National University of Singapore (SG)
- Khon Kaen University (TH)
- Mahidol University (TH)
- Health Intervention and Technology Assessment Program (TH)
- Queen Sirikit National Institute of Child Health (TH)
- Phramongkutklao Hospital (TH)
- Ramathibodi Hospital (TH)
- Institute for Work & Health (CA)
- National University Health System (SG)
- Phramongkutklao College of Medicine
- Chiang Mai University (TH)
Publication Details
- Journal
- PharmacoEconomics - Open
- Published
- 2025-04-29
- DOI
- https://doi.org/10.1007/s41669-025-00579-x
- Citations
- 2
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 2.33
Funders
- King Chulalongkorn Memorial Hospital
- Thailand Science Research and Innovation
- Faculty of Medicine Siriraj Hospital, Mahidol University