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别嘌醇与非布司他治疗慢性痛风患者的成本效果分析:美国支付者视角。

Cost-effectiveness analysis of allopurinol versus febuxostat in chronic gout patients: a U.S. payer perspective.

机构信息

South College School of Pharmacy, 400 Goodys Ln., Knoxville, TN 37922.

出版信息

J Manag Care Spec Pharm. 2015 Feb;21(2):165-75. doi: 10.18553/jmcp.2015.21.2.165.

Abstract

BACKGROUND

Gout is a chronic inflammatory condition associated with poor urate metabolism. Xanthine oxidase inhibitors such as allopurinol and febuxostat are recommended to reduce uric acid levels and to prevent gout attacks in adult patients. Under budget-driven constraints, health care payers are faced with the broader challenge of assessing the economic value of these agents for formulary placement. However, the economic value of allopurinol versus febuxostat has not be assessed in patients with gout over a 5-year time period in the United States.

OBJECTIVE

To evaluate the cost-effectiveness of allopurinol versus febuxostat in adult patients with gout over a 5-year time period from a U.S. health care payer's perspective.

METHODS

A Markov model was developed to compare the total direct costs and success of serum uric acid (sUA) level reduction associated with allopurinol and febuxostat. Treatment success was defined as patient achievement of a sUA level less than  6 mg/dL (0.36 mmol/L) at 6 months. Event probabilities were based on published phase III randomized clinical trials and included long-term sequelae from open-label extension studies. A hypothetical cohort of 1,000 adult gout patients with sUA levels of ≥ 8 mg/dL (0.48 mmol/L) who had received either allopurinol 300 mg or febuxostat 80 mg at model entry transitioned among the 4 health states defined by treatment success, treatment failure and switch, treatment dropout, and death. The length of each Markov cycle was 6 months. Costs were gathered from the RED BOOK, Medicare fee schedules, Healthcare Cost and Utilization Project's Nationwide Inpatient Sample, and for a limited number of inputs, expert consultation. Direct costs included treatment drug costs, costs for prophylaxis drugs, diagnostic laboratory tests, and the treatment and management of acute gout flare. Resource utilization was based on clinical evidence and expert consultation. All costs were inflated to 2014 U.S. dollars and were discounted at 3% in the base case. One-way sensitivity analysis and probabilistic sensitivity analyses (PSAs) were performed to assess the robustness of the results.

RESULTS

The total per patient cost incurred over 5 years was $50,295 for febuxostat and $48,413 for allopurinol, with an incremental total cost of $1,882. The expected percentage of treatment success during the 5-year period was 72 for febuxostat and 42 for allopurinol, resulting in an incremental percentage of treatment success of 30. The estimated incremental cost-effectiveness ratio for febuxostat compared with allopurinol was $6,322 per treatment success over a 5-year time period. The one-way sensitivity analysis indicated that the results were sensitive to probability of treatment success for allopurinol, probability of treatment dropouts for both allopurinol and febuxostat, and the probability of failure and switch to allopurinol. PSAs demonstrated that at a willingness-to-pay threshold of $50,000 per treatment success, febuxostat was cost-effective compared with allopurinol.

CONCLUSIONS

Febuxostat was found to be a cost-effective option compared with allopurinol based on a U.S. payer perspective.

摘要

背景

痛风是一种与尿酸代谢不良有关的慢性炎症性疾病。黄嘌呤氧化酶抑制剂,如别嘌醇和非布司他,被推荐用于降低尿酸水平,并预防成年患者的痛风发作。在预算驱动的限制下,医疗保健支付者面临着更广泛的挑战,即评估这些药物在定价中的经济价值。然而,在美国,尚未在痛风患者中评估别嘌醇与非布司他在 5 年内的经济学价值。

目的

从美国医疗保健支付者的角度,评估 5 年内别嘌醇与非布司他治疗痛风患者的成本效益。

方法

开发了一个 Markov 模型,比较了别嘌醇与非布司他在 5 年内治疗痛风患者的总直接成本和血清尿酸(sUA)水平降低的成功率。治疗成功定义为患者在 6 个月时达到 sUA 水平<6mg/dL(0.36mmol/L)。基于已发表的 III 期随机临床试验和开放标签扩展研究的长期后遗症,计算了事件概率。一个包含 1000 名血尿酸水平≥8mg/dL(0.48mmol/L)的成年痛风患者的假设队列,在模型进入时接受别嘌醇 300mg 或非布司他 80mg,在治疗成功、治疗失败和转换、治疗脱落和死亡定义的 4 个健康状态之间转换。每个 Markov 周期的长度为 6 个月。成本来自 RED BOOK、医疗保险费用表、医疗保健成本和利用项目全国住院患者样本,以及有限数量的投入,通过专家咨询获得。直接成本包括治疗药物成本、预防药物成本、诊断实验室测试以及急性痛风发作的治疗和管理。资源利用基于临床证据和专家咨询。所有成本均按 2014 年美元进行了通胀调整,并在基础情况下按 3%贴现。进行了单因素敏感性分析和概率敏感性分析(PSA),以评估结果的稳健性。

结果

5 年内,非布司他的每位患者总成本为 50295 美元,别嘌醇为 48413 美元,增量总成本为 1882 美元。5 年内预期的治疗成功率为非布司他 72%,别嘌醇 42%,治疗成功率提高 30%。非布司他与别嘌醇相比,5 年内每例治疗成功的增量成本效益比为 6322 美元。单因素敏感性分析表明,结果对别嘌醇治疗成功率、别嘌醇和非布司他治疗脱落率以及非布司他和别嘌醇治疗失败和转换的概率敏感。PSA 表明,在每例治疗成功 50000 美元的意愿支付阈值下,非布司他与别嘌醇相比具有成本效益。

结论

从美国支付者的角度来看,非布司他被认为是一种比别嘌醇更具成本效益的选择。

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