Economic evaluation of ombitasvir/paritaprevir/ritonavir and dasabuvir for the treatment of chronic genotype 1 hepatitis c virus infection

Objectives: To estimate clinical outcomes and cost-effectiveness of ombitasvir/paritaprevir/ritonavir and dasabuvir?±?ribavirin (OMB/PTV/r?+?DSV?±?RBV) compared with treatment regimens including pegylated interferon (PegIFN) for patients with chronic genotype 1 hepatitis C virus (HCV) infection.

Methods: An Excel spreadsheet Markov model tracking progression through stages of liver disease was developed. Costs and patient utilities for liver disease stages were taken from published studies. Rates of disease progression were based on studies of untreated HCV infection and long-term follow-up of those achieving sustained virologic response (SVR) after drug treatment. Impact of OMB/PTV/r?+?DSV?±?RBV and other drug regimens on progression was estimated through SVR rates from clinical trials. Analyses were performed for treatment-naive and treatment-experienced patients. Impact of alternative scenarios and input parameter uncertainty on the results were tested.

Results: For genotype 1 treatment-naive HCV patients, for OMB/PTV/r?+?DSV?±?RBV, PegIFN?+?ribavirin (PegIFN/RBV), sofosbuvir?+?PegIFN/RBV, telaprevir?+?PegIFN/RBV, boceprevir?+?PegIFN/RBV, lifetime risk of decompensated liver disease was 5.6%, 18.9%, 7.4%, 11.7%, and 14.9%; hepatocellular carcinoma was 5.4%, 9.2%, 5.7%, 7.0%, and 7.4%; and death from liver disease was 8.7%, 22.2%, 10.4%, 14.8%, and 17.6%, respectively. Estimates of the cost-effectiveness of OMB/PTV/r?+?DSV?±?RBV for treatment-naive and treatment-experienced patients indicated that it dominated all other regimens except PegIFN/RBV. Compared with PegIFN/RBV, the incremental cost-effectiveness ratios were £13,864 and £10,258 per quality-adjusted life-year (QALY) for treatment-naive and treatment-experienced patients, respectively. The results were similar for alternative scenarios and uncertainty analyses.

Limitations: A mixed-treatment comparison for SVR rates for the different treatment regimens was not feasible, because many regimens did not have comparator arms; instead SVR rates were based on those from recent trials.

Conclusions: OMB/PTV/r?+?DSV?±?RBV is a cost-effective oral treatment regimen for chronic genotype 1 HCV infection compared with standard treatment regimens and is estimated to reduce the lifetime risks of advanced liver disease.     

艾尔巴韦/格拉瑞韦治疗慢性丙型病毒性肝炎的价值评估

目的全面总结评估艾尔巴韦/格拉瑞韦的临床价值、经济价值、病人价值、社会价值,为临床合理用药,减轻患者疾病负担提供参考。方法检索PubMed、Embase、中国期刊全文数据库、中文期刊全文数据库和万方数据知识服务平台等数据库,回顾性分析艾尔巴韦/格拉瑞韦治疗慢性丙型肝炎(CHC)的临床试验研究、真实世界疗效研究以及药物经济学相关研究等资料,总结艾尔巴韦/格拉瑞韦治疗基因1、4型CHC的临床价值、经济价值、病人价值和社会价值。结果在临床价值方面,艾尔巴韦/格拉瑞韦对基因1型或4型CHC患者,以及合并人类免疫缺陷病毒(HIV)、慢性肾脏疾病(CKD)等患者,均具有较高的持续病毒学应答率(SVR)。在经济价值方面,国内外药物经学评价结果显示,艾尔巴韦/格拉瑞韦在治疗CHC基因1(1a,1b)型,以及合并CKD患者中,均具有经济学优势。同时,艾尔巴韦/格拉瑞韦有助于提高病人价值和社会价值,可改善患者身体功能、生理职能、总体健康、情感职能、心理健康等,同时因其可治愈CHC而有助于节约社会生产力,减少疾病传播,具有良好的社会效益。结论艾尔巴韦/格拉瑞韦疗效好,有助于消除丙肝目标的实现。     

Budesonide with multi-matrix technology as second-line treatment for ulcerative colitis: evaluation of long-term cost-effectiveness in the Netherlands

Aims: Budesonide with multi-matrix technology (MMX) is an oral corticosteroid, shown to have high topical activity against ulcerative colitis (UC) while maintaining low systemic bioavailability with few adverse events. The aim of this study was to evaluate the cost-effectiveness of budesonide MMX versus commonly used corticosteroids, in the second-line treatment of active mild-to-moderate UC in the Netherlands.

Materials and methods: An eight-state Markov model with an 8 week cycle length captured remission, four distinct therapy stages, hospitalization, possible colectomy and mortality. Remission probability for budesonide MMX was based on the CORE-II study. Population characteristics were derived from the Dutch Inflammatory Bowel Disease South Limburg cohort (n?=?598) and included patients with proctitis (39%), left-sided (42%) and extensive disease (19%). Comparators (topical budesonide foam and enema, oral budesonide and prednisolone) were selected based on current Dutch clinical practice. Treatment effects were evaluated by network meta-analysis using a Bayesian framework. Cost-effectiveness analysis was performed over a 5 year time horizon from a societal perspective, with costs, health-state and adverse event utilities derived from published sources. Outcomes were weighted by disease extent distribution and corresponding comparators.

Results: Budesonide MMX was associated with comparable quality-adjusted life year (QALY) gain versus foam and oral formulations (+0.01 QALYs) in the total UC population, whilst being cost-saving (EUR 366 per patient). Probabilistic sensitivity analysis evaluated an 86.6% probability of budesonide MMX being dominant (cost-saving with QALY gain) versus these comparators. Exploratory analysis showed similar findings versus prednisolone.

Limitations: Differing definitions of trial end-points and remission across trials meant indirect comparison was not ideal. However, in the absence of head-to-head clinical data, these comparisons are reasonable alternatives and currently offer the only comparison of second-line UC treatments.

Conclusions: In the present analysis, budesonide MMX was shown to be cost-effective versus comparators in the total UC population, for the second-line treatment of active mild-to-moderate UC in the Netherlands.     

中药内服配合敷脐治疗慢性结肠炎患者的临床疗效

目的：探讨中药内服配合敷脐治疗慢性结肠炎患者的临床疗效。方法选取2014年1月至2015年1月在辽阳市中医医院肛肠科接受治疗的114例慢性结肠炎患者,按随机数字表法将其分为试验组与对照组,各57例,试验组患者采用中药内服配合敷脐治疗,对照组患者应用糖皮质激素、免疫抑制剂药物治疗。比较两组患者的临床效果。结果试验组患者总有效率为94.7%,明显高于对照组的84.2%,差异有统计学意义（P＜0.05）。结论中药内服配合敷脐用于慢性结肠炎的疗效确切,有助于肠道功能的恢复。     

A cost-effectiveness analysis of MMX mesalazine compared with mesalazine in the treatment of mild-to-moderate ulcerative colitis from a UK perspective

Abstract

Objectives: To perform a cost-utility analysis of a new formulation of mesalazine (Mezavant XL, MMX mesalazine) versus an existing oral mesalazine (Asacol; mesalazine) from the UK National Health Service perspective.

Methods: A 5-year Markov cohort model was developed. Costs were obtained from the literature and utilities from an independent study. Uncertainty was evaluated using one-way and probabilistic sensitivity analyses (PSA). The potential effect of dosing frequency on adherence and possible long-term effects of remission maintenance on colorectal cancer (CRC) rates were also investigated.

Results: The model suggested that 5-year therapy with MMX mesalazine was likely to generate gains when compared with mesalazine, including a gain of 0.011 QALYs per patient, 19 more remission days, and 12% fewer hospitalizations and surgical episodes. These gains came at an increase in total NHS direct cost of £8, resulting in an incremental cost-effectiveness ratio (ICER) of £749. The PSA suggested that MMX mesalazine had a 62% chance of resulting in cost savings, and a 74% chance of being cost effective (£20,000 threshold). Extended analysis including adherence and CRC effects suggested further incremental benefit of MMX mesalazine over mesalazine could be expected. Limitations include uncertainty in extrapolation to a 5-year time horizon and impact of adherence and drug acquisition costs on outcomes.

Conclusion: The pharmacoeconomic analysis suggested that MMX mesalazine is likely to produce small, but worthwhile, increases in total NHS direct cost while increasing time in remission and associated quality of life, when compared with mesalazine. Advantages in adherence to treatment with MMX mesalazine relative to mesalazine suggested that further health gains and cost savings can be obtained. Overall, these results suggest that MMX mesalazine is a cost-effective treatment for UC.     

Economic evaluation of sevelamer for the treatment of hyperphosphatemia in chronic kidney disease patients not on dialysis in the United Kingdom

Abstract

Objectives:

To determine the cost effectiveness of sevelamer vs calcium carbonate in patients with chronic kidney disease and not on dialysis (CKD-ND) from the perspective of the National Health Service (NHS) in the UK.

Methods:

A Markov decision analytic model was developed to estimate (1) total life years (LYs), quality-adjusted life years (QALYs), and costs for patients treated with sevelamer or calcium carbonate; and (2) incremental costs per LY gained (LYG) and per QALY gained for sevelamer vs calcium carbonate. Data informing probability transitions to all-cause death and dialysis inception in CKD-ND patients were taken directly from the INDEPENDENT-CKD study and were extrapolated beyond the 3-year clinical trial using Weibull regression analysis. Estimates of health utility and costs (in £2011) were derived from the published literature.

Results:

Over a lifetime horizon, sevelamer treatment resulted in a gain of 2.05 LYs and 1.56 QALYs per patient, an increase of £37,282 in total costs per patient vs calcium carbonate (3.5% discount), and a per-patient cost of £18,193/LYG and £23,878/QALY gained. Results were robust to alternative assumptions in key parameters; results were most sensitive to alternative assumptions regarding the mean daily dose of sevelamer, impact of sevelamer on dialysis initiation, cost of dialysis, and health utility estimates. The probabilistic sensitivity analysis showed that sevelamer was cost-effective vs calcium carbonate in 93% of simulations at a willingness-to-pay threshold of £30,000/QALY gained.

Limitations:

While the model simulated a real-world clinical setting, this analysis was subject to limitations common to all decision analytic models, in that it used a mix of data sources and relied on several assumptions. Not all variables that impact real-world outcomes and costs were included in this model.

Conclusions:

Sevelamer is a cost-effective option compared to calcium carbonate for the first-line treatment of hyperphosphatemia in CKD-ND patients in the UK.     

Congregate meal sites participants: Can they manage their diets?

Many individuals attending congregate meal sites have chronic conditions that can be impacted by their diet. This study found that congregate meal site participants visit meal sites primarily for social purposes but consume meals while they satisfy their social agenda. Also, individuals that suffer from diabetes, cancer, and heart disease were not aware of what foods were beneficial in prevention and/or maintenance. Specifically, individuals with diabetes, cancer, and heart disease were not able to determine any better than those without those conditions which food types were recommended by nutrition experts, nor which food types were more beneficial in the maintenance or prevention of their conditions.     

An economic evaluation of tofacitinib for the treatment of moderately-to-severely active ulcerative colitis: modeling the cost of treatment strategies in the United States

Abstract

Aims: To evaluate the cost differences between a treatment strategy including tofacitinib (TOFA) vs treatment strategies including adalimumab (ADA), golimumab (GOL), infliximab (IFX), and vedolizumab (VEDO) among all patients with moderate-to-severe ulcerative colitis (UC) (further stratified by patients naïve/exposed to tumor necrosis factor inhibitors [TNFis]).

Materials and methods: An Excel-based decision-analytic model was developed to evaluate costs from the perspective of a third-party US payer over 2 years. Efficacy and safety parameters were taken from prescribing information and published trials. All patients started induction therapy on the first treatment in the strategy and continued if efficacy criteria were met and no major adverse event occurred (in which cases they proceeded to the next treatment in the strategy).

Results: The cost per member per month (PMPM) of the TOFA–>IFX–>VEDO–>GOL strategy ($1.11) was lower than that of the ADA–>IFX–>VEDO–>GOL strategy ($1.34; Δ = $?0.23) among the TNFi-naïve population (n?=?204 patients out of a plan of one million members). Similarly, the use of TOFA before ADA (i.e. TOFA–>ADA–>IFX–> VEDO) was also associated with lower PMPM costs than the use of ADA before TOFA (i.e. ADA–>TOFA–>IFX–>VEDO): $1.15 vs $1.25 (Δ = $?0.10). Similar, and often larger, differences were observed in both the overall moderate-to-severe population and the TNFi-exposed population. Sensitivity analyses resulted in the same conclusions.

Limitations: Our model relied on efficacy data from prescribing information and published trials, which were not head-to-head and slightly differed with respect to methods. Additionally, our model used representative minor and major ADRs (and the associated costs) to represent toxicity management, which was a simplifying assumption.

Conclusions: This analysis, the first of its kind to evaluate TOFA vis-à-vis other advanced therapies in the US, suggests the early use of TOFA among both TNFi-naïve and TNFi-failure patients results in lower PMPM costs compared with other treatment alternatives.