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研究生：	楊珺婷 Yang, Chun-Ting
論文名稱：	應用真實世界數據於成本效益評估之方法學建議：以第二型糖尿病藥品sodium glucose cotransporter-2 inhibitors相較dipeptidyl peptidase-4 inhibitors之成本效益分析為例 Establishment of recommendations on conducting cost-effectiveness analyses using real-world data – illustrated by a model-based cost-effectiveness analysis of sodium glucose cotransporter-2 inhibitors versus dipeptidyl peptidase-4 inhibitors in type 2 diabetes
指導教授：	歐凰姿 Ou, Huang-Tz
共同指導教授:	郭士禎 Kuo, Shih-Chen
學位類別：	博士 Doctor
系所名稱：	醫學院 - 臨床藥學與藥物科技研究所 Institute of Clinical Pharmacy and Pharmaceutical sciences
論文出版年：	2023
畢業學年度：	111
語文別：	英文
論文頁數：	158
中文關鍵詞：	真實世界數據、成本效益分析、方法學建議、第二型糖尿病、SGLT-2抑制劑、DPP-4抑制劑
外文關鍵詞：	real-world data, cost-effectiveness analysis, methodological recommendations, type 2 diabetes, SGLT-2 inhibitors, DPP-4 inhibitors
相關次數：	點閱：123 下載：0
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研究背景
近年來，真實世界數據於醫療與健康政策決策中逐漸扮演重要角色，而以真實世界數據為主之成本效益分析研究亦隨之增加。然而，儘管現存文獻陸續提出此類研究之方法學挑戰，目前仍缺乏相關建議與指引供研究者參考。因此，本研究旨在提供以應用真實世界數據於執行成本效益分析時之方法學建議 (第一章)，並以案例研究 (第二、三章) 說明如何實際運用所提出之建議。

研究方法
第一章以敘述性綜論方式執行。首先搜尋以應用真實世界數據於成本效益分析之相關方法學文章，經初步篩選納入適當文章後，與專家針對欲於方法學建議中所傳遞之資訊達成共識。而後，將各文章資訊萃取與統整於方法學建議中，統整邏輯依序為：整合執行成本效益分析時之方法學建議、提出以應用真實世界數據於成本效益分析時之方法學挑戰、提出上述方法學挑戰之解決方案。
後續以案例研究「台灣第二型糖尿病人使用sodium glucose co-transporter-2 inhibitors (SGLT2is) 相較於dipeptidyl peptidase-4 inhibitors (DPP4is) 之成本效益分析」為例，說明如何實際運用第一章所提出之方法學建議。第二章著重於探討第二型糖尿病人之心血管風險預測式於此成本效益分析族群之適用性，包括區別能力與校正能力。第三章中，將真實世界數據所生成之臨床療效、成本、以及健康效用參數，套入心血管風險預測式與模擬模型中，執行成本效益分析。此研究之資料來源為國立成功大學醫學院附設醫院及國立台灣大學醫學院附設醫院之電子病歷。

研究結果
第一章建立一方法學建議清單，當中包含十三項與成本效益分析研究設計相關之考量面向：真實世界數據資料來源、研究觀點、研究族群、治療對照組、分析時間長度、折現率、療效結果指標之選擇、療效之測量、健康效用之測量、成本之測量、成本效益模擬模型之效度、異質性之探討、以及研究結果不確定性之評估。上述建議已考量於後續案例研究中。
第二章中，Chinese Hong Kong Integrated Modeling and Evaluation模型中之心肌梗塞、中風、與心衰竭風險預測式皆於成大醫院與台大醫院之第二型糖尿病病人中顯示可被接受之模型區別能力 (area under the receiver operating characteristics curve介於0.7078 與0.7907之間)，然而模型校正能力仍有改善空間 (校正斜率介於0.3666與0.7982之間、校正截距介於-0.0363與-0.0007之間)。經再校正後，風險預測式之校正能力改善，且皆通過Greenwood-Nam-D'Agostino測試 (p值>0.05)；經再校正之風險預測式後續套用於第三章之成本效益分析模擬模型中。
第三章分析結果顯示，於真實世界之台灣第二型糖尿病病人中，SGLT2is相較於DPP4is為具成本效益之治療選項，此健康經濟好處於不同基本臨床特質病人中皆有顯示 (每多獲得一生活品質校正存活年所需之成本介於美金2,295與20,939元之間)。SGLT2i相關之經濟效益於年齡65歲以上 (每多獲得一生活品質校正存活年所需之成本美金：2,295元) 與60≤eGFR<90 ml/min/1.73m2 (每多獲得一生活品質校正存活年所需之成本美金：5,419元) 之糖尿病病人更為顯著；相較之下，於年齡未達65歲 (每多獲得一生活品質校正存活年所需之成本美金：20,939元) 與eGFR≥90 ml/min/1.73m2 (每多獲得一生活品質校正存活年所需之成本美金：12,452元) 之糖尿病病人則效益稍低。敏感性分析結果與基礎情境分析結果一致。

研究結論
本研究針對以真實世界數據執行成本效益分析提出系列方法學建議，可以作為建立此研究類型方法學指引之起始。未來將需與各領域專家合作，並定期搜尋且更新新穎之方法學，以完善此研究所提出之方法學建議。

Background
With the advocation of utilizing real-world data (RWD) in supporting clinical and health policy decision-makings, RWD-based cost-effectiveness analyses (CEAs) have increasingly emerged dramatically. Although several methodological challenges of performing RWD-based CEAs were raised, a guidance for practical recommendations to address the methodological issues related to the use of RWD in CEAs remains lacked. Therefore, the present study aimed to provide structuralized recommendations on the methodologies of RWD-based CEAs (Chapter 1) and use case studies to illustrate how the proposed recommendations can be practically applied (Chapters 2 and 3). Accomplishment of this study is anticipated to encourage the use of RWD in CEAs that appropriately reflect real-world settings and facilitate the allocation of constrained healthcare resources.

Methods
A narrative review was conducted to identify relevant articles to the methodologies and considerations of conducting RWD-based CEAs. After the screening and selection for suitable articles, expert discussions were held to reach consensus on the key messages to be delivered in the recommendation list. Data extraction was further performed with the following process: summarizing the guidance on conducting CEAs, describing the challenges faced while using RWD in CEAs, and providing potential solutions for the methodological issues related to the use of RWD in CEAs.
A model-based CEA of sodium glucose cotransporter-2 inhibitor (SGLT2i) versus dipeptidyl peptidase-4 inhibitor (DPP4i) therapy among patients with type 2 diabetes (T2D) in Taiwan was performed to illustrate how the proposed recommendations in the Chapter 1 were applied in the analyses, including 1) adaptation of risk equations for predicting cardiovascular outcomes to the target population in CEA, namely Taiwanese T2D population (Chapter 2), and 2) utilization of effectiveness, cost, and health utility parameters generated from RWD in the CEA modeling analyses (Chapter 3). Electronic health records (EHRs) from the National Cheng Kung University Hospital (NCKUH) and the National Taiwan University Hospital (NTUH) were utilized as the data sources in the illustration studies.

Results
In the Chapter 1, a recommendation list that comprised of 13 essential components in study design of RWD-based CEAs was established, including choice of RWD source, perspectives, study population, comparators, time horizon, discount rate, selection of outcomes, measurement of outcomes, valuation of outcomes, measurement of costs, validity of the CEA models, characterizing heterogeneity, and characterizing uncertainty. These recommendations were considered in the design and conduction of the case studies in Chapters 2 and 3.
In the Chapter 2, risk equations of predicting myocardial infarction, stroke, and heart failure from the Chinese Hong Kong Integrated Modeling and Evaluation model all revealed acceptable discrimination among T2D patients identified from NCKUH and NTUH EHRs (i.e., area under the receiver operating characteristics curve ranging from 0.7078 to 0.7907) while the calibrations were unsatisfactory (calibration slopes and intercepts ranging from 0.3666 to 0.7982 and -0.0363 to -0.0007, respectively). After the recalibration procedure, the recalibrated risk equations all showed improved calibrations (calibration slopes and intercepts ranging from 0.8714 to 1.1060 and -0.0046 to 0.0063, respectively) and passed the GND tests (p value>0.05), which were considered valid for further application in the Chapter 3.
In the Chapter 3, the cost-effectiveness of SGLT2i versus DPP4i therapy was revealed across real-world T2D patients with various characteristics (incremental cost-effectiveness ratios ranging from United States dollar [USD] 2,295 to 20,939 per quality-adjusted life-year [QALY] gained). The economic benefits of SGLT2i use were more pronounced among patients aged≥65 years (USD 2,295 per QALY gained) versus <65 years (USD 20,939 per QALY gained) and those with 60≤eGFR<90 (USD 5,419 per QALY gained) versus eGFR≥90 (USD 12,452 per QALY gained) ml/min/1.73m2. Sensitivity analyses showed consistent results with the base-case analysis.

Conclusions
The present study provides an overview of practical recommendations for designing and conducting RWD-based CEA studies, which may serve as a starting point for the emergent need of the development of such guidance. Future collaborations with multi-stakeholder expertise and periodic updates that keep up with the modern methodologies are warranted to advance the current work.

中文摘要 I
Abstract III
誌謝 V
Content VI
Index of Tables IX
Index of Figures XI
Chapter 1. Establishment of recommendations on conducting cost-effectiveness evaluations using real-world data – illustrated by a model-based cost-effectiveness analysis of SGLT2is versus DPP4is 1
1	Background 2
2	Methods 4
2.1	Search strategy 4
2.2	Selection criteria and data extraction 4
3	Results 5
3.1	Choice of real-world sources for estimating study parameters 9
3.2	Study perspectives	12
3.3	Determination of study populations 12
3.4	Determination of study comparators 13
3.5	Time horizon 13
3.6	Discount rate 14
3.7	Selection of effectiveness outcomes 14
3.8	Measurement of outcomes 15
3.9	Valuation of outcomes 19
3.10	Measurement of costs 24
3.11	Validity of the CEA models 27
3.12	Characterizing patient heterogeneity 28
3.13	Characterizing uncertainty 29
4	Discussions 31
5	Conclusions 36
Chapter 2. Adaptation of risk equations of predicting cardiovascular outcomes in patients with type 2 diabetes to Taiwanese population	 37
1	Background 38
1.1	Global and local picture of diabetes 38
1.2	Computational models for disease prediction in T2D 38
1.3	Considerations of applying existing prediction models and risk equations in Taiwanese T2D populations 39
1.4	Study aim and significance 39
2	Methods	41
2.1	Identification of risk prediction equations for further adaptation in Taiwanese T2D population 41
2.2	Identification of data sources and study cohort 43
2.3	Identification of risk predictors and cardiovascular outcomes, and their operational definitions 44
2.4	Assessment of the performance of existing risk prediction equations	46
2.5	Recalibration of risk prediction equations 46
2.6	Evaluation of internal validation of the recalibrated risk prediction equations 48
3	Results 49
3.1	Patient characteristics of the T2D cohorts identified from NCKUH and NTUH 49
3.2	Observed event risks among the overall T2D cohorts 49
3.3	Patient characteristics and observed event risks of cardiovascular outcomes in training and test sets 49
3.4	Discrimination of risk prediction equations in NCKUH and NTUH populations 56
3.5	Calibration of risk prediction equations in NCKUH and NTUH populations 57
3.6	Performance of the recalibrated risk prediction equations from CHIME model 62
4	Discussions 68
4.1	Differences in the patient characteristics and event risks between patients with T2D from NCKUH and NTUH 68
4.2	Performance of risk prediction equations from the UKPDS-OM2, RECODe, and CHIME models in Taiwanese T2D population	68
4.3	Variations in the recalibration process between NCKUH and NTUH populations 72
4.4	Considerations of applying the recalibrated risk prediction equations among Taiwanese T2D population	73
4.5	Strengths and limitations of the recalibration method in the present study 74
4.6	Clinical and health policy implications 74
4.7	Study limitations 75
5	Conclusion 76
Chapter 3. Heterogeneity in health and economic outcomes of SGLT-2 inhibitors versus DPP-4 inhibitors among patients with type 2 diabetes: A model-based cost-effectiveness analysis using real-world data	77
1	Background 78
2	Methods 79
2.1	Model structure 79
2.2	Study cohorts 79
2.3	Transition probabilities between model states 80
2.4	Health utility and cost parameters	84
2.5	Base-case analysis 84
2.6	Sensitivity analyses	85
2.7	Statistical software and reporting transparency 85
3	Results 86
4	Discussions 94
4.1	Comparisons with current evidence on cost-effectiveness of SGLT2is versus DPP4is 94
4.2	Pronounced economic benefits of SGLT2i therapy among patients aged≥65 years and with 60≤eGFR<90 ml/min/min2 94
4.3	Variations in the cost-effectiveness estimates by patient baseline HbA1c levels 96
4.4	Influential drivers of ICER estimates 97
4.5	Clinical and health policy implications 97
4.6	Study limitations 98
5	Conclusion 100
References 101
Appendix 110
                                    

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