Economic Evaluation of Selumetinib for Treating Pediatric Patients With Neurofibromatosis Type 1 Inoperable Plexiform Neurofibromas in Thailand
Author(s)
Parnnaphat Luksameesate, PhD, Jariya Anansansophon, B.Sc., Chanthawat Patikorn, PharmD, PhD, Suthira Taychakhoonavudh, BSc, MS, PhD.
Department of Social and Administrative Pharmacy, Faculty of Pharmaceutical Sciences, Chulalongkorn University, Bangkok, Thailand.
Department of Social and Administrative Pharmacy, Faculty of Pharmaceutical Sciences, Chulalongkorn University, Bangkok, Thailand.
Presentation Documents
OBJECTIVES: Neurofibromatosis type 1 (NF1) is a genetic disorder affecting 1 in 3,000 people, leading to tumors such as plexiform neurofibromas (PN) that cause pain, disfigurement, and reduced quality of life in children. Selumetinib, a MEK inhibitor, has shown effectiveness in shrinking PN tumors but comes with high costs. This study aims to estimate the cost-utility analysis of selumetinib compared to best supportive care (BSC) for the treatment of pediatric patients with NF1 and symptomatic inoperable PNs.
METHODS: The Markov model was utilized to evaluate costs and health outcomes, including Quality Adjusted Life Years (QALY) and Life Years (LY), from a societal perspective. Unit costs and resource utilization were gathered from standard cost lists of the Thai Health Technology Assessment, published literature, the Drug and Medical Supply Information Center, and expert opinions. Health outcomes were derived from an electronic database of NF1 and PN patients in the United Kingdom. Transitional probabilities were obtained from published literature. The incremental cost-effectiveness ratio (ICER) will be calculated to present the cost-utility analysis. One-way and probabilistic sensitivity analyses were performed to evaluate the model's uncertainty.
RESULTS: The study results indicated that Selumetinib costs 27,681,992 Thai Baht (THB) for 20.52 QALYs, whereas BSC costs 2,905,535 THB for 17.71 QALYs. The incremental cost of 24,776,457 THB for a 2.81 QALY gain resulted in an ICER of 8,806,762 THB/QALY, which exceeds Thailand’s cost-effectiveness threshold of 160,000 THB/QALY. The subgroup analysis revealed variations in ICER across tumor locations, ranging from 8,799,177 THB/QALY for the trunk to 8,880,903 THB/QALY for the head and neck.
CONCLUSIONS: Selumetinib has demonstrated clinical benefits; however, its high cost poses challenges for reimbursement within Thailand’s healthcare system. To address this issue, evidence from this study should be used to explore and support alternative funding solutions.
METHODS: The Markov model was utilized to evaluate costs and health outcomes, including Quality Adjusted Life Years (QALY) and Life Years (LY), from a societal perspective. Unit costs and resource utilization were gathered from standard cost lists of the Thai Health Technology Assessment, published literature, the Drug and Medical Supply Information Center, and expert opinions. Health outcomes were derived from an electronic database of NF1 and PN patients in the United Kingdom. Transitional probabilities were obtained from published literature. The incremental cost-effectiveness ratio (ICER) will be calculated to present the cost-utility analysis. One-way and probabilistic sensitivity analyses were performed to evaluate the model's uncertainty.
RESULTS: The study results indicated that Selumetinib costs 27,681,992 Thai Baht (THB) for 20.52 QALYs, whereas BSC costs 2,905,535 THB for 17.71 QALYs. The incremental cost of 24,776,457 THB for a 2.81 QALY gain resulted in an ICER of 8,806,762 THB/QALY, which exceeds Thailand’s cost-effectiveness threshold of 160,000 THB/QALY. The subgroup analysis revealed variations in ICER across tumor locations, ranging from 8,799,177 THB/QALY for the trunk to 8,880,903 THB/QALY for the head and neck.
CONCLUSIONS: Selumetinib has demonstrated clinical benefits; however, its high cost poses challenges for reimbursement within Thailand’s healthcare system. To address this issue, evidence from this study should be used to explore and support alternative funding solutions.
Conference/Value in Health Info
2025-09, ISPOR Real-World Evidence Summit 2025, Tokyo, Japan
Value in Health Regional, Volume 49S (September 2025)
Code
RWD112
Topic Subcategory
Health & Insurance Records Systems
Disease
SDC: Rare & Orphan Diseases