Presentation (CTI)

OBJECTIVES: A novel adeno-associated virus (AAV) gene therapy for treating hemophilia B, BBM-H901, recently published the results of its key clinical trial that 26 patients achieved the factor IX level of 55 IU/dL at 52 weeks post-infusion. This study aimed to assess the potential cost-effectiveness of BBM-H901 compared to routine prophylaxis infusions of standard half-life recombinant Factor IX (SHL rFIX) from the perspective of the healthcare system in China.
METHODS: A lifetime, five-state Markov model, including "No Bleeding", "Non-Joint Bleeding", "Joint Bleeding", "Arthroplasty", and "Death", was developed. The cycle length was one week, and all the patients started from the “No Bleeding” state at 18 years old. The proportion of patients experiencing arthroplasty was modeled using a micro-simulation experiment, predicting how the history of joint bleedings was associated with the occurrence of arthroplasty. Given the absence of lifetime follow-up data, a conservative extrapolation method was explored to assess the durability of gene therapy. According to a previous cost-effectiveness analysis study, we assumed that the Factor IX level would decrease by 1 IU/dL annually from the clinical trial endpoint of 55 IU/dL until it reached 3 IU/dL, at which point the therapy would be deemed a failure, and patients would transition to prophylaxis treatment. Parameters of clinical indicators, utilities, and costs were derived from published literature and real-world research.
RESULTS: Compared to prophylaxis infusion of SHL rFIX, BBM-H901 demonstrated higher quality-adjusted life years (QALYs) gains due to a lower frequency of injection and bleeding, along with cost savings of avoiding routine prophylaxis injection twice a week, resulting in a dominant outcome.
CONCLUSIONS: BBM-H901 was shown to be an economically dominant alternative, offering lower costs and higher QALYs, compared to SHL rFIX for hemophilia B patients in China.