COST-EFFECTIVENESS ANALYSIS OF PROPHYLAXIS VS. ON-DEMAND TREATMENT FOR HAEMOPHILIA B CHILDREN WITHOUT INHIBITOR IN CHINA
Author(s)
Liu Q1, Xin Q2, Chen Z2, Li L3, Runhui W2
1Hematology Oncology Center, Beijing Children’s Hospital, Capital Medical University, Beijing, 11, China, 2Hematology Oncology Center, Beijing Children’s Hospital, Capital Medical University, Beijing, China, 3Shanghai Palan DataRx Co. Ltd., Shanghai, 31, China
Presentation Documents
OBJECTIVES Haemophilia B(HB) is a hereditary bleeding disorder caused by a deficiency of coagulation factor IX (FIX), which represents 15~20% of the hemophilia patients. Currently, replacement therapy is the dominant treatment, including on-demand (OD) and prophylaxis treatment. Clinical studies have demonstrated significant benefits of prophylaxis treatment with FIX versus OD treatment. However, these benefits are associated with an increase in FIX consumption and considerable costs, and most of Chinese HB pediatric patients are on OD treatment. The objective of this study was to assess the cost effectiveness of prophylaxis treatment compared with OD treatment in HB children without inhibitors from the payer perspectives. METHODS We developed a Markov model to perform a cost-utility analysis that comparing prophylaxis versus OD treatment for HB. The model ran over a 16-year time horizon using 1 yearly cycle. Transition probabilities and utility weights were estimated using published studies, and cost were gathered from HB patients with medical insurance in Beijing Children hospital, including drug cost, utilization of both in- and outpatient . Both one-way and probabilistic sensitivity analysis were performed to assess the robustness of results. RESULTS The model projected prophylaxis had an incremental gain of 1.62 quality-adjusted life years of with an incremental cost of $13,943.The incremental cost per quality-adjusted life years gained for individuals with haemophilia B receiving prophylaxis were $8611.26 compared with OD arm, which is under the willingness-to-pay threshold(3 times of GDP per capital) in Beijing of $ 29,306.33. Moreover, one-way sensitivity analyses showed that the results were sensitive to the transition probability of normal to joint bleeding and PSA results showed that it had 97.8% probability to prove the conclusion robust. CONCLUSIONS Although prophylaxis is a costly treatment, the results show that it’s cost -effective compared with on demand treatment in HB children in China.
Conference/Value in Health Info
2019-11, ISPOR Europe 2019, Copenhagen, Denmark
Code
PRO24
Disease
Rare and Orphan Diseases