Department of Pharmaceutical Outcomes & Policy, College of Pharmacy, University of Florida, Gainesville.
Center for Drug Evaluation and Safety, College of Pharmacy, University of Florida, Gainesville.
J Manag Care Spec Pharm. 2023 Aug;29(8):884-895. doi: 10.18553/jmcp.2023.29.8.884.
Paliperidone is among the most cost-effective antipsychotics in adults with schizophrenia, and it has different formulations, including oral paliperidone extended-release (ER) and long-acting injectable (LAI) paliperidone formulations administered every month (PP1M), 3 months (PP3M), or 6 months (PP6M). However, cost-effectiveness analyses comparing different paliperidone formulations were limited. To compare the cost-effectiveness across different paliperidone formulations. A Markov model was developed to simulate 1,000 adults aged 40 years with stable schizophrenia transitioning among stable disease-medication adherent, stable disease-medication nonadherent, relapse with hospitalization, relapse with ambulatory care, and death states every 3 months for 5 years. Drug costs were estimated using the prices listed in the Veterans Affairs Federal Supply Schedule, and costs for treating complications were estimated from published studies. All costs were estimated from the US health care system perspective and standardized to 2022 US dollars using the Consumer Price Index Inflation Calculator. Quality-adjusted life-years (QALYs) were estimated using relapse rates from randomized clinical trials and health-related quality of life scores from observational studies. The estimated future costs and QALYs were discounted at 3%. We reported incremental net monetary benefits between alternative formulations at the $50,000 willingness-to-pay (WTP) threshold with a positive value indicating cost-effectiveness. The impact of parameter uncertainty on study outcomes was assessed using 1-way deterministic and probabilistic sensitivity analyses. In adults with schizophrenia stabilized with paliperidone ER, switching to LAI formulations was associated with increased QALY (PP1M = 0.05, PP3M = 0.14, PP6M = 0.15) and increased cost (PP1M = 49,433, PP3M = 26,698, PP6M = 26,147), leading to a negative incremental net monetary benefit (PP1M = -$46,804, PP3M = -$19,508, PP6M = -$18,886) compared with continuing ER. Among LAI formulations, PP6M was cost-saving with the most QALYs gained (cost = $63,277, QALY = 3.731), followed by PP3M (cost = $63,828, QALY = 3.729) and PP1M (cost = $86,563, QALY = 3.638). At the $50,000 WTP threshold, the probabilities for PP1M, PP3M, and PP6M being cost-effective compared with paliperidone ER were 0.4%, 10.2%, and 9.8%, respectively. The probability of PP6M being cost-effective was 92.6% for the PP6M-PP1M pair and 55.2% for the PP6M-PP3M pair, and 91.1% of PP3M use was cost-effective in the PP3M-PP1M pair. The results were generally robust in the sensitivity analyses, even at the $190,000 WTP threshold. For patients with schizophrenia stabilized with paliperidone ER, switching to LAI formulations was not cost-effective, suggesting the high drug costs for LAI may not justify the improved quality of life within 5 years. Among LAI formulations, PP6M was cost-effective over PP1M and PP3M.
帕利哌酮是精神分裂症成人患者中最具成本效益的抗精神病药物之一,它有不同的剂型,包括口服帕利哌酮延长释放(ER)和长效注射(LAI)帕利哌酮制剂,每月(PP1M)、每 3 个月(PP3M)或每 6 个月(PP6M)给药。然而,比较不同帕利哌酮制剂的成本效益分析有限。为了比较不同帕利哌酮制剂的成本效益,我们开发了一个马尔可夫模型,以模拟 1000 名年龄在 40 岁的稳定精神分裂症患者,这些患者在稳定的疾病-药物依从性、稳定的疾病-药物不依从性、住院复发、门诊护理复发和死亡状态之间转换,每 3 个月转换一次,持续 5 年。药物成本使用退伍军人事务部联邦供应时间表中列出的价格进行估计,并发症治疗成本使用已发表的研究进行估计。所有成本均从美国医疗保健系统的角度进行估计,并使用消费者价格指数通胀计算器标准化为 2022 年的美元。使用随机临床试验中的复发率和观察性研究中的健康相关生活质量评分估计质量调整生命年(QALY)。使用替代制剂的增量净货币效益在 50000 美元的支付意愿(WTP)阈值下进行报告,正值表示成本效益。使用 1 种确定性和概率敏感性分析评估参数不确定性对研究结果的影响。在稳定使用帕利哌酮 ER 的精神分裂症成年患者中,转换为 LAI 制剂与增加 QALY(PP1M=0.05,PP3M=0.14,PP6M=0.15)和增加成本(PP1M=49433 美元,PP3M=26698 美元,PP6M=26147 美元)相关,与继续使用 ER 相比,导致增量净货币效益为负(PP1M=-46804 美元,PP3M=-19508 美元,PP6M=-18886 美元)。在 LAI 制剂中,PP6M 成本效益最高,获得的 QALY 最多(成本=63277 美元,QALY=3.731),其次是 PP3M(成本=63828 美元,QALY=3.729)和 PP1M(成本=86563 美元,QALY=3.638)。在 50000 美元的 WTP 阈值下,PP1M、PP3M 和 PP6M 与帕利哌酮 ER 相比具有成本效益的概率分别为 0.4%、10.2%和 9.8%。对于 PP6M-PP1M 对和 PP6M-PP3M 对,PP6M 具有成本效益的概率分别为 92.6%和 55.2%,而在 PP3M-PP1M 对中,91.1%的 PP3M 使用具有成本效益。即使在 190000 美元的 WTP 阈值下,结果在敏感性分析中也是稳健的。对于稳定使用帕利哌酮 ER 的精神分裂症患者,转换为 LAI 制剂并不具有成本效益,这表明 LAI 的高药物成本可能无法在 5 年内证明提高生活质量是合理的。在 LAI 制剂中,PP6M 比 PP1M 和 PP3M 更具成本效益。