- Title
- An overview of evidence- and value-based economic evaluation for health care in chronic disease
- Creator
- Luan, Luan
- Relation
- University of Newcastle Research Higher Degree Thesis
- Resource Type
- thesis
- Date
- 2019
- Description
- Research Doctorate - Doctor of Philosophy (PhD)
- Description
- In China, the drug expenditure accounts for 40% of total health expenditure, which is much higher than the average reported by the Organisation for Economic Co-operation and Development. [1, 2] If the desired health outcomes can be achieved, high utilization of pharmaceuticals would not be a problem. However, without evidence of achieving the desired health outcomes, this high utilization of pharmaceuticals naturally leads to an interest of pharmaceutical policy makers regarding drug reimbursement and pricing. Particularly, the 2009 national healthcare reforms in China brought about an increase in the use of health economic evidence when drug companies apply for new drug approvals or drug pricing negotiation.[3] Health economic evaluations have been emphasized increasingly in the drug assessment process in China. Practically speaking, health economic evaluations focus on costs and outcomes of the intervention. Cost-effectiveness analysis (CEA) is a form of health economic analysis which is used commonly to compare the costs and the outcome or effects of two or more treatments or interventions. Whereas, cost-utility analysis (CUA), considered as a subgroup of CEA, studies the cost of treatments or interventions per quality-adjusted life year (QALY). QALY reflects both quantity and quality of life under certain treatment, which is widely used for assessing treatments in chronic diseases. [4-6] However, the evidence of outcomes and cost are not always available in China, and hence hinder the wider application of economic evaluation in facilitating cost-effective drug assessment, and more rational healthcare resource allocation. So, in our presented studies, we are going to explore how to access or explore the different aspects of CEA (CUA) in terms of efficacy, utility, and cost evidence in the evaluation process and finally how to build a CEA (CUA) model in China when such evidence is not available. Considering cardiovascular diseases (CVDs) being the major cause of disability and mortality worldwide and in China, we take CVDs as the example disease of interest. Hopefully, this would provide some insights and guidance for other researchers evaluating new or existing drug therapy for other diseases and conditions in China. In this thesis, the research studies carried out can be divided into two major parts, namely, Part One – Assessing Outcomes (effectiveness and utility) and Part Two – Assessing Costs. In Part One, to explore the application of utility in economic evaluation, we conducted a systematic review on the validation and application of health-related quality of life questionnaire (HRQL) on ischemic heart disease (IHD) in Chinese-speaking populations first to identify the gap in HRQL application on IHD patients in China. After confirming a lack of an IHD-core instrument for evaluating HRQL in Chinese IHD patients. We then conducted a study to translate and validate a Chinese version IHD-specific HRQL instrument for IHD patients in China. Considering utility scores can be derived from certain generic instruments, to avoid the use of multiple instruments in practice, we built a model to map this validated version of HeartQoL to EQ-5D so that utility scores will be available when applying only one IHD-specific instrument clinically. For application of other effectiveness measures in economic evaluation, efficacy is always an important outcome and data could be usually obtained from published randomized clinical trials and meta-analyses conducted locally or elsewhere. However, there are often no head-to head comparison within one specific class of drugs, making it difficult for clinicians and administrators to make informed decisions about ranking specific class of drugs based on their relative efficacy. So, to illustrate how to resolve this problem, we conducted a network meta-analysis for Angiotensin Receptor Blocker (ARB) for Heart Failure, which could provide a efficacy ranking among ARB drugs, and further could provide evidence to the clinicians and regulators in their decision-making clinically as well as in resource allocation. In this part, there are four chapters included: Chapter 2. A Review of Studies of Quality of Life for Chinese-Speaking Patients with Ischemic Heart Disease; Chapter 3. The psychometric evaluation of the Chinese version HeartQoL Questionnaire among patients with ischemic heart disease in China; Chapter 4. Mapping Utility Scores from HeartQoL Questionnaire into EQ-5D for Ischemic Heart Disease; and Chapter 5. Angiotensin Receptor Blocker for Heart Failure: A Network Meta-Analysis. In Part Two, to explore how to measure cost, we conducted a cross-sectional cost of illness study to estimate the cost of IHD and its three sub diagnoses –angina, myocardial infarction(MI) and heart failure(HF) - including direct, indirect and intangible costs, and analyze the factors that influence the costs in China, which is presented in Chapter 6. Cost of Ischemic Heart Disease and factors affecting the costs: A cross-sectional study in China. Finally, after exploring the feasibility of applying effectiveness, utility and cost for CEA and CUA, we conducted a CEA and CUA model of Valsartan, an ARB drug, for heart failure in China. In this study, we evaluated the additional effects of Valsartan as an add-on therapy to standard therapy, compared with the standard therapy in the Chinese population to further illustrate the feasibility of conducting CEA (CUA) in China. The results are presented in Chapter 7. The Cost Utility of Valsartan for Heart Failure in China. In conclusion, this thesis has systematically explored feasibility of conducting CEA and CUA for cardiovascular disease in China. We provided solutions to access the evidence when it is unavailable, new perspectives of analyzing the available evidence and an application of CEA and CUA in economic evaluations for cardiovascular diseases in China. With the newly validated IHD-specific instrument, and the mapping algorithm, we are able to assess patient’ HRQL and derive utility values without generic instruments; With the network meta-analysis of assessing efficacy, we are able to assess not only the efficacy of individual drugs in meta-analysis, but also the various drugs’ ranking based on their relative efficacy. With the real-world data from cost of illness, we gain deeper insight on how the different components of costs influencing the total cost of illness; and by building a CUA and CEA model, we demonstrated how to use the data of utility, efficacy and cost form domestic and international source to contribute for the gap in health economic evaluations on Chinese drug reimbursement and pricing assessment process. Furthermore, those information and applications would not only assist in healthcare policy administrations, but also are beneficial for clinician in clinical decision-making.
- Subject
- chronic diseases; ischemic heart diseases; quality of life; thesis by publication; angina; heart failure; patient reported outcomes; network meta analysis; ARB drugs; cost of illness; cost effectiveness modelling; China
- Identifier
- http://hdl.handle.net/1959.13/1402941
- Identifier
- uon:35082
- Rights
- Copyright 2019 Luan Luan
- Language
- eng
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| View Details Download | ATTACHMENT01 | Thesis | 10 MB | Adobe Acrobat PDF | View Details Download | ||
| View Details Download | ATTACHMENT02 | Abstract | 267 KB | Adobe Acrobat PDF | View Details Download |