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Background/Objectives <p>Inhibitor development against recombinant factor VIII (rFVIII) is a significant complication in the prophylactic treatment of severe hemophilia A. The economic impact of varying inhibitor risks among rFVIII products is unclear. We compare the cost effectiveness of two rFVIII products with different incidences of inhibitor development (antihemophilic factor [recombinant], plasma/albumin-free method [rAHF-PFM] vs BAY 81-8973) for prophylaxis in Chinese previously untreated patients (PUPs) with severe hemophilia A.</p> Methods <p>From a Chinese healthcare system perspective, a Markov model simulated the lifetime outcomes (inhibitors, bleeding, complications) for PUPs with severe hemophilia A receiving lifetime prophylactic treatment with rAHF-PFM or BAY 81-8973 (incidence of inhibitor development: 30.3% vs 54.8%). Data were sourced from literature, clinical experts, and public sources. Cost effectiveness was assessed using base-case, one-way sensitivity, and probabilistic sensitivity analysis (PSA).</p> Results <p>The base-case analysis indicated that rAHF-PFM extended survival by 0.055 years (17.965 vs 17.910) and increased quality-adjusted life years (QALYs) by 0.779 QALYs (9.290 vs 8.511), while saving Chinese Yuan (CNY) 4,578,403 (CNY 12,060,222 vs CNY 16,638,625) compared with BAY 81-8973 in PUPs with severe hemophilia A. Results were most sensitive to the annualized bleeding rate and utility of patients free of bleeding episodes. PSA with 5000 Monte Carlo simulations estimated a 97.5% probability that rAHF-PFM was the cost-effective option.</p> Conclusions <p>Prophylactic treatment with rAHF-PFM is likely more cost effective than BAY 81-8973 for Chinese PUPs with severe hemophilia A. This finding is primarily driven by the lower incidence of inhibitor development and fewer bleeding episodes associated with rAHF-PFM in the model.</p>

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Impact of Inhibitor Development on the Cost Effectiveness of Prophylactic Treatment with Recombinant Factor VIII in Previously Untreated Patients with Severe Hemophilia A

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2025年12月4日

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  • Li Yang,
  • Jie Peng,
  • Congling Gu,
  • Zhenguo Wang,
  • Genyong Zuo

摘要

Background/Objectives

Inhibitor development against recombinant factor VIII (rFVIII) is a significant complication in the prophylactic treatment of severe hemophilia A. The economic impact of varying inhibitor risks among rFVIII products is unclear. We compare the cost effectiveness of two rFVIII products with different incidences of inhibitor development (antihemophilic factor [recombinant], plasma/albumin-free method [rAHF-PFM] vs BAY 81-8973) for prophylaxis in Chinese previously untreated patients (PUPs) with severe hemophilia A.

Methods

From a Chinese healthcare system perspective, a Markov model simulated the lifetime outcomes (inhibitors, bleeding, complications) for PUPs with severe hemophilia A receiving lifetime prophylactic treatment with rAHF-PFM or BAY 81-8973 (incidence of inhibitor development: 30.3% vs 54.8%). Data were sourced from literature, clinical experts, and public sources. Cost effectiveness was assessed using base-case, one-way sensitivity, and probabilistic sensitivity analysis (PSA).

Results

The base-case analysis indicated that rAHF-PFM extended survival by 0.055 years (17.965 vs 17.910) and increased quality-adjusted life years (QALYs) by 0.779 QALYs (9.290 vs 8.511), while saving Chinese Yuan (CNY) 4,578,403 (CNY 12,060,222 vs CNY 16,638,625) compared with BAY 81-8973 in PUPs with severe hemophilia A. Results were most sensitive to the annualized bleeding rate and utility of patients free of bleeding episodes. PSA with 5000 Monte Carlo simulations estimated a 97.5% probability that rAHF-PFM was the cost-effective option.

Conclusions

Prophylactic treatment with rAHF-PFM is likely more cost effective than BAY 81-8973 for Chinese PUPs with severe hemophilia A. This finding is primarily driven by the lower incidence of inhibitor development and fewer bleeding episodes associated with rAHF-PFM in the model.