Frontiers in pharmacology · 2026
Value-based pricing of brensocatib for frequently exacerbating non-cystic fibrosis bronchiectasis in China: a China-adapted, FEV1-inclusive time-dependent markov analysis.
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Zhang Dandan, Hu Pin, Zhang Wei, Zhang Wenwen
Аннотация
OBJECTIVE: To estimate a China-specific value-based annual price (P*) for brensocatib 25 mg added to standard care (SC), compared with SC alone, in adults with frequently exacerbating non-cystic fibrosis bronchiectasis, and to assess how strongly the estimate depends on transferred assumptions linking forced expiratory volume in 1 s (FEV1) to long-term outcomes.
METHODS: A seven-state Markov cohort model with monthly cycles and a lifetime horizon was constructed from the Chinese healthcare system perspective. Chinese sources informed the target population, exacerbation burden, baseline microbiology, background mortality, resource use, costs, discounting, and decision context, while ASPEN supplied the exacerbation rate ratio (0.81) and annual FEV1 decline (24 mL with brensocatib 25 mg vs. 62 mL with placebo). In the base case, cohort-mean FEV1 was linked to utility and mortality through explicit external mappings. A prespecified structural sensitivity removed both mappings but retained the trial-observed exacerbation benefit. Costs and outcomes were discounted by 4.5% annually. The primary willingness-to-pay (WTP) threshold was CNY 200,000 per quality-adjusted life-year (QALY); uncertainty was examined with a paired 10,000-draw probabilistic sensitivity analysis (PSA).
RESULTS: At an illustrative price of CNY 120,000/year, the FEV1-inclusive base case estimated an incremental cost of CNY 551,543, gains of 0.2761 QALYs and 0.3432 discounted life-years, and 0.57 fewer lifetime exacerbations. These results corresponded to an incremental cost-effectiveness ratio of CNY 1,997,665/QALY and P* of CNY 11,692/year. Drug acquisition accounted for almost all of the incremental cost. The PSA median P* was CNY 11,611/year (95% simulation interval CNY 4,145-19,324), and none of 10,000 simulations was cost-effective at CNY 120,000/year for WTP thresholds up to CNY 300,000/QALY. Estimates changed little when China-specific demographic and exacerbation inputs were varied. In contrast, removing the FEV1-linked pathway reduced P* to CNY 990/year, while assuming persistent efficacy after attrition increased it to CNY 24,432/year.
CONCLUSION: With inputs localised wherever credible Chinese evidence was available, the FEV1-inclusive base case estimated P* at CNY 11,692/year for a WTP of CNY 200,000/QALY. The broad pricing implication was stable, although the exact estimate depended chiefly on the durability and downstream consequences of the 52-week FEV1 difference. Both the base case and the key structural sensitivity remained far below the illustrative annual price of CNY 120,000.
METHODS: A seven-state Markov cohort model with monthly cycles and a lifetime horizon was constructed from the Chinese healthcare system perspective. Chinese sources informed the target population, exacerbation burden, baseline microbiology, background mortality, resource use, costs, discounting, and decision context, while ASPEN supplied the exacerbation rate ratio (0.81) and annual FEV1 decline (24 mL with brensocatib 25 mg vs. 62 mL with placebo). In the base case, cohort-mean FEV1 was linked to utility and mortality through explicit external mappings. A prespecified structural sensitivity removed both mappings but retained the trial-observed exacerbation benefit. Costs and outcomes were discounted by 4.5% annually. The primary willingness-to-pay (WTP) threshold was CNY 200,000 per quality-adjusted life-year (QALY); uncertainty was examined with a paired 10,000-draw probabilistic sensitivity analysis (PSA).
RESULTS: At an illustrative price of CNY 120,000/year, the FEV1-inclusive base case estimated an incremental cost of CNY 551,543, gains of 0.2761 QALYs and 0.3432 discounted life-years, and 0.57 fewer lifetime exacerbations. These results corresponded to an incremental cost-effectiveness ratio of CNY 1,997,665/QALY and P* of CNY 11,692/year. Drug acquisition accounted for almost all of the incremental cost. The PSA median P* was CNY 11,611/year (95% simulation interval CNY 4,145-19,324), and none of 10,000 simulations was cost-effective at CNY 120,000/year for WTP thresholds up to CNY 300,000/QALY. Estimates changed little when China-specific demographic and exacerbation inputs were varied. In contrast, removing the FEV1-linked pathway reduced P* to CNY 990/year, while assuming persistent efficacy after attrition increased it to CNY 24,432/year.
CONCLUSION: With inputs localised wherever credible Chinese evidence was available, the FEV1-inclusive base case estimated P* at CNY 11,692/year for a WTP of CNY 200,000/QALY. The broad pricing implication was stable, although the exact estimate depended chiefly on the durability and downstream consequences of the 52-week FEV1 difference. Both the base case and the key structural sensitivity remained far below the illustrative annual price of CNY 120,000.