Validation of Models That Estimate the Cost-Effectiveness of Improving Patient Adherence

This note suggests a test for internal validation of models that estimate the costs and effects of improving patient adherence. We apply the validation test to two published cost-effectiveness models on adherence improvement.     

Tuberculosis patient adherence to direct observation: results of a social study in Pakistan

A randomized controlled trial was carried out in Pakistan in 1999 to establish the effectiveness of the direct observation component of DOTS programmes. It found no significant differences in cure rates for patients directly observed by health facility workers, community health workers or by family members, as compared with the control group who had self-administered treatment. This paper reports on the social studies which were carried out during and after this trial, to explain these results. They consisted of a survey of all patients (64% response rate); in-depth interviews with a smaller sample of different types of patients; and focus group discussions with patients and providers. One finding was that of the 32 in-depth interview patients, 13 (mainly from the health facility observation group) failed to comply with their allocated DOT approach during the trial, citing the inconvenience of the method of observation. Another finding was that while patients found the overall TB care approach efficient and economical in general, they faced numerous barriers to regular attendance for the direct observation of drug-taking (most especially, time, travel costs, ill health and need to pursue their occupation). This may be one of the reasons why there was no overall benefit from direct observation in the trial. Provider attitudes were also poor: health facility workers expressed cynical and uncaring views; community health workers were more positive, but still arranged direct observation to suit their, rather than patients', schedules. The article concludes that direct observation, if used, should be flexible and convenient, whether at a health facility close to the patient's home or in the community. The emphasis should shift in practice from tablet watching towards treatment support, together with education and other adherence measures.     

Impact of Non-Adherence and Flare Resolution on the Cost-Effectiveness of Treatments for Gout: Application of a Linked Pharmacometric/Pharmacoeconomic Model

Background

Dual urate-lowering therapy (ULT) with lesinurad in combination with either allopurinol or febuxostat is an option for patients with gout unsuccessfully treated on either monotherapy. Treatment failure is often a result of poor medication adherence. Imperfect adherence in clinical trials may lead to biased estimates of treatment effect and confound the results of cost-effectiveness analyses.

Toward a Broader Concept of Value: Identifying and Defining Elements for an Expanded Cost-Effectiveness Analysis

This commentary identifies and defines potentially useful expansions to traditional cost-effectiveness analysis as often used in health technology assessment. Since the seminal 1977 article by Weinstein and Stason, the recommended approach has been the use of the incremental cost-effectiveness ratio based on the metric of the cost per quality-adjusted life-year gained, allowing comparisons across different technologies. An expanded framework, incorporating a wider range of the elements of value, is proposed. In addition to the core value drivers of health gain and other health system cost savings (if any), we propose adding other less recognized elements related to the value of knowing and informational externalities. We describe each of five factors related to the value of knowing: 1) a reduction in uncertainty, reflecting the benefit of a companion diagnostic increasing the certainty of a patient?s response to a medicine; 2) insurance value related to greater peace of mind due to protection against catastrophic health and financial loss; 3) the value of hope for a "cure," leading individuals to become risk seekers in some circumstances; 4) real option value due to life extension opening possibilities for individuals to benefit from future innovation; and 5) spillovers or externalities arising from benefits of scientific advances that cannot be entirely appropriated by those making the advances. Further thought and research are needed on how best to measure and integrate these elements into an incremental value framework and on coverage and pricing decisions.     

Emergence of Increased Resistance and Extensively Drug-Resistant Tuberculosis Despite Treatment Adherence, South Africa

We investigated the emergence and evolution of drug-resistant tuberculosis (TB) in an HIV co-infected population at a South African gold mine with a well-functioning TB control program. Of 128 patients with drug-resistant TB diagnosed during January 2003–November 2005, a total of 77 had multidrug-resistant (MDR) TB, 26 had pre–extensively drug-resistant TB (XDR TB), and 5 had XDR TB. Genotyping suggested ongoing transmission of drug-resistant TB, and contact tracing among case-patients in the largest cluster demonstrated multiple possible points of contact. Phylogenetic analysis demonstrated stepwise evolution of drug resistance, despite stringent treatment adherence. These findings suggested that existing TB control measures were inadequate to control the spread of drug-resistant TB in this HIV co-infected population. Diagnosis delay and inappropriate therapy facilitated disease transmission and drug-resistance. These data call for improved infection control measures, implementation of rapid diagnostics, enhanced active screening strategies, and pharmacokinetic studies to determine optimal dosages and treatment regimens.     

某职业技术学院结核病聚集性病例的调查与处置

目的 探讨某职业技术学院发生结核病聚集性病例的应急处置措施与对策.方法 采用现场流行病学方法,包括疫情报告、病例的核实诊断、传染源追查、病例个案信息收集、密切接触者筛查、患者治疗管理、感染者的预防、健康教育和现场处置等.结果 该学院物流系活动性肺结核罹患率为3 676/10万,传染源所在的电商0801、0802班罹患率为17％,感染率分别为74.4％、78.1％,感染率与同系其他班、其他系比较,P值均小于0.01,差异有统计学意义.结论 该学院物流系电商0801班、0802班局部发生结核病暴发流行,其处置及时、措施得当.     

DOTS策略下深圳市结核病疫情控制效果分析

目的探讨DOTS策略下肺结核病人管冶效果及疫情变化趋势。方法收集1993～2003年深圳市实施DOTS策略中结核病防治有关资料，以及流行病学调查资料，并进行描述性分析。结果从1993年至2003年，登记涂阳肺结核8483例，治愈率96，3％。肺结核疫情明显下降，深圳市户籍人口涂阳患病率下降55．9％，非户籍人口涂阳患病率下降38，6％。结论DOTS作为现代结核病控制策略是深圳市结核病疫情有效控制的基础。     

Cost-Effectiveness Analysis of Oseltamivir for Influenza Treatment Considering the Virus Emerging Resistant to the Drug in Japan

Aim:  The purpose of this study is to evaluate the cost-effectiveness of oseltamivir for influenza in Japan considering the complications and the emergence of oseltamivir-resistant virus.
Methods:  Study design is a cost-effectiveness analysis in decision analytic modeling based on previously published evidence. Outcome measures included costs and quality-adjusted life year (QALY).
Results and Conclusion:  In the base-case analysis, the incremental cost-effectiveness ratio (ICER) of oseltamivir during influenza and complications was JPY398,571 ($3320) per QALY without productivity loss, which implied oseltamivir is evidently cost-effective. Furthermore, considering the productivity loss, the ICER for oseltamivir turned to be negative, which means simply dominant. When the prevalence was in the low range of 10% to 38%, oseltamivir became less cost-effective than conventional treatment. Regarding potential emergence of the drug-resistant virus, we found the dominance of oseltamivir will vanish if the emerging rate becomes larger than 27%. The two-way sensitivity analysis also suggested that if the resistant virus rate becomes less and the prevalence higher, then oseltamivir becomes more advantageous. The analysis for uncertainty, using cost-effectiveness acceptability curve by Monte Carlo simulation, resulted in the estimate of about 80% chance that oseltamivir could be cost-effective at the willingness-to-pay level of JPY6,000,000 ($50,000), which is commonly accepted as an affordable threshold.     

A Transparent and Consistent Approach to Assess US Outpatient Drug Costs for Use in Cost-Effectiveness Analyses

Background

Assessment of drug costs for cost-effectiveness analyses (CEAs) in the United States is not straightforward because the prices paid for drugs are not publicly available and differ between payers. CEAs have relied on list prices that do not reflect the rebates and discounts known to be associated with these purchases.

Formal Implementation of Cost-Effectiveness Evaluations in Japan: A Unique Health Technology Assessment System

In April 2019, Japan formally introduced health technology assessment (HTA) and, more specifically, a cost-effectiveness analysis, to inform healthcare decision making, mainly when it comes to the pricing of new technologies. This article provides an overview of this new policy, which was implemented formally after a pilot program. In the fiscal year (FY) 2012, discussions on cost-effectiveness assessments were initiated in Japan. After 7 years of deliberations, a cost-effectiveness assessment was implemented formally in April 2019. In Japan, the cost-effectiveness analysis has been used to inform price adjustments of healthcare technologies, although it has not yet been used for decision making on insurance coverage. Selection criteria were established because not all drugs and medical devices could be evaluated owing to a shortage of experts. Exclusion criteria have also been applied to prevent access restriction. The scope of the evaluation’s price adjustment target is limited to part of the product price. If the cost per quality-adjusted life-year (QALY) threshold falls below ¥5 million per QALY, the price adjustment rate changes stepwise according to the cost per QALY. In addition to price reduction, a price-raising scheme has also been implemented for scenarios where products are evaluated to be highly cost-effective and innovative. This article describes the first formally implemented HTA system in Japan. Although it is too early to make any conclusions about its effect, the Japan-specific context makes this system unique. To fully understand the opportunities and challenges of the new system, it is vital that Japan accumulates experience with this system and develops human resources in health economic evaluation.     

Cost-Effectiveness Analysis of Rituximab Combined with CHOP for Treatment of Diffuse Large B-Cell Lymphoma

PURPOSE: To estimate the cost-effectiveness from a French payer perspective of CHOP (cyclophosphamide, doxorubicin, vincristine, and prednisone) alone compared with CHOP plus rituximab (R-CHOP) for treatment of patients with diffuse large B-cell lymphoma. METHODS: Mean patient survival, days of hospitalization, and chemotherapy costs during treatment were estimated from a Phase III trial in France, Belgium, and Switzerland. Survival during the trial was estimated using the Kaplan-Meier method; survival beyond the trial period was projected based on mortality rates from the Scottish and Newcastle Lymphoma Group database. French diagnosis-related group (DRG) payment schedules were applied to trial data to estimate cost of adverse events and drug administration. We estimated survival and cost-effectiveness [the incremental cost per quality-adjusted life-year (QALY) gained] from 4 years (median clinical trial follow-up period) to 15 years, discounted at a fixed annual rate of 3%. We used published patient preferences. We converted currency to euros, based on 2003 exchange rates. RESULTS: R-CHOP resulted in a 20.6% relative increase in complete response rate (absolute increase from 63% to 76%), and a 31% decrease in risk of death at 4 years (95% CI 8-49%). Over a 15-year time horizon, mean overall survival (OS) duration was estimated to be 6.90 years for R-CHOP and 5.74 years for CHOP, a mean increase in OS of 1.16 years (or 1.07 QALYs). Total direct medical costs were 13,170 euro higher with R-CHOP, with an incremental cost-effectiveness ratio of 12,259 euro per QALY gained. CONCLUSION: R-CHOP significantly increases mean OS up to 4 years compared with CHOP, and its cost-effectiveness ratio compares favorably with other oncology treatments in widespread use.     

自我护理教育对传染性肺结核患者治疗依从性和生活质量的影响

目的：研究自我护理教育对传染性肺结核患者治疗依从性和生活质量的影响。方法将该院于2014年1月-2015年1月收治90例的传染性肺结核患者,根据随机分组法分为观察组与对照组各45例,对照组进行常规健康教育,观察组除此之外还要实施自我护理教育,对比两组对患者治疗依从性和生活质量的影响。结果观察组治疗依从性基本做到、完全做到机率均优于对照组,差异有统计学意义(P<0.05)。观察组躯体功能、心理功能、社会功能、物质生活、总生活质量评分均高于对照组,差异有统计学意义(P<0.05)。结论自我护理教育在传染性肺结核患者中的效果显著。对患者依从性以及生活质量有着提升作用。     

Model-Based Cost-Effectiveness Analyses for the Treatment of Acute Stroke Events: A Review and Summary of Challenges

Objective:  To summarize the methodological approaches used in published decision-analytic models evaluating interventions for acute stroke treatment, to highlight key components of decision-analytic models of stroke treatment, and to discuss challenges for developing stroke decision models.
Methods:  A review of the published literature was performed using Medline, to identify studies involving mathematical decision models to evaluate interventions for acute stroke treatment. Articles were analyzed to determine key components of a stroke model and to note areas in which data are lacking.
Results:  We identified 13 published models of acute stroke treatment. These models typically possessed a short-term treatment module and a long-term post-treatment module. The following aspects of economic modeling were found to be relevant for developing a stroke model: modeling approach and health state; health state transition probabilities; estimation of short-term, long-term, and indirect costs; health state utilities; poststroke mortality; time horizon; model validation; and estimation of parameter uncertainty.
Conclusions:  Data gaps have limited the development of economic models in stroke to date. In order to more accurately assess the long-term incremental impact of a new treatment of stroke, future research is needed to address these data gaps. We recommend that the complexity of models for examining the cost-effectiveness of an acute stroke treatment be kept to a minimum such that it can incorporate the currently available data without making a large number of assumptions around the data.     

Task shifting for tuberculosis control: A qualitative study of community-based directly observed therapy in urban Uganda

This qualitative study of task shifting examined tuberculosis (TB) therapy under modified community-based directly observed treatment short-course (CB-DOTS) in Kampala, Uganda. New TB patients selected one of two strategies: home-based DOTS and clinic-based DOTS. Relevant socio-economic characteristics, treatment-seeking experiences and outcomes were assessed over eight months of follow-up. Of 107 patients recruited, 89 (83%) selected home-based DOTS. Sixty-two patients (70%) under home-based DOTS and 16 patients (89%) under clinic-based DOTS had successful outcomes following completion of tuberculosis therapy. Treatment supporters’ provision of social support beyond observing drug ingestion contributed to successful outcomes under both strategies. Home-based DOTS provides continuity of social support during therapy, strengthening the potential for treatment success. Conventional health facility-based DOTS can be modified in resource-limited urban Africa to offer a viable DOTS strategy that is sensitive to personal preference. Shifting the task of DOTS support away from only qualified health workers to include laypersons in the patients’ social-support network may contribute to meeting World Health Organization (WHO) treatment targets. We recommend an intervention evaluating this modified DOTS strategy on a larger scale in TB high-burden, resource-poor urban settings.     

西安市灞桥区2004～2008年结核病控制效果及其影响因素

目的评价西安市灞桥区2004～2008年推行现代结核病控制(DOTS)策略的效果,探讨提高涂阳肺结核患者发现率和治愈率的影响因素。方法收集2004～2008年西安市灞桥区结核病控制工作月报表、季报表、年报表资料以及各年度工作总结,统计各年度涂阳患者发现情况、治疗转归情况以及健康教育、业务培训和督导管理工作情况,采用SPSS 13.0软件对资料进行统计分析。结果西安市灞桥区从2004年起推行DOTS策略,新涂阳肺结核患者发现率逐年增加,由2004年的72.22%增加到2008年的82.82%。治愈率也逐年提高,由2004年的72.00%增加到2008年的86.93%。结核病防治知识的宣传教育、医务人员结核病防治培训与发现率之间呈正相关(P0.01),全程督导管理与治愈率之间呈正相关(P0.05)。结论通过实施DOTS策略,西安市灞桥区涂阳结核患者发现率和治愈率达到了WHO和中国国家卫生部制订的结核病控制目标。结核病防治知识的宣传教育、医务人员结核病防治培训和全程督导管理可提高涂阳肺结核患者发现率和治愈率。     

Cost and Cost-Effectiveness of the Mediterranean Diet: Results of a Systematic Review

The growing impact of chronic degenerative pathologies (such as cardiovascular disease, type 2 diabetes and Alzheimer’s disease) requires and pushes towards the development of new preventive strategies to reduce the incidence and prevalence of these diseases. Lifestyle changes, especially related to the Mediterranean diet, have the potential to modify disease outcomes and ultimately costs related to their management. The objective of the study was to perform a systematic review of the scientific literature, to gauge the economic performance and the cost-effectiveness of the adherence to the Mediterranean diet as a prevention strategy against degenerative pathologies. We investigated the monetary costs of adopting Mediterranean dietary patterns by determining cost differences between low and high adherence. Research was conducted using the PubMed and Scopus databases. Eight articles met the pre-determined inclusion criteria and were reviewed. Quality assessment and data extraction was performed. The adherence to the Mediterranean diet has been extensively reported to be associated with a favorable health outcome and a better quality of life. The implementation of a Mediterranean dietary pattern may lead to the prevention of degenerative pathologies and to an improvement in life expectancy, a net gain in health and a reduction in total lifetime costs.