- 學位論文
使用Temsirolimus治療晚期腎細胞癌之成本效益分析
Cost-Effectiveness Analysis of Temsirolimus in Patients with Poor Risk Renal-Cell Carcinoma
摘要
腎細胞癌是腎臟惡性腫瘤中的一種，研究數據顯示約有四分之一的患者於初次診斷時已出現局部侵犯或為轉移性腎細胞癌，其存活率相當低。二十年來在腎細胞癌的臨床治療上以免疫治療的干擾素-α為主，但於近五年間腎細胞癌的治療方式已逐漸改變為使用分子標靶藥物。新的標靶藥物治療方式雖具有療效，但其對經濟財政所造成的衝擊仍不清楚，而在政府與社會資源有限與合理分配使用的考量中，對於藥物或健康介入應在「成本」與「效益」雙方陎的考量下進行健康照護經濟評估，藉以獲取社會與民眾的最大利益。本研究目的是以健保給付的觀點，評估分子標靶藥物Temsirolimus治療晚期腎細胞癌患者之成本效益，希望結果可作為健康照護決策者在其他晚期腎細胞癌治療藥物或相關治療方式進行成本效益分析時的參考資料。 本研究以晚期腎細胞癌患者分別使用temsirolimus或干擾素-α作為治療比較方案，經國內外之相關成本、療效及健康生活品質參數等文獻收集與彙整，並透過建構決策分析模式，進行兩種方案間的成本效益分析。研究中對成本與健康效果皆使用3%折現率，最後，對模式之參數進行單維敏感度分析與機率性敏感度分析，以了解研究結果的不確定性。 III 對晚期腎細胞癌患者而言，使用temsirolimus為治療方案與使用干擾素-α的方案作比較，經折現後，於模式模擬每位患者帄均增加0.24個品質調整存活人年數的基礎上，進一步計算出每增加一個品質調整存活人年數的ICER值為NT$ 4,775,609。同時，在每延長一個存活年的ICER值是NT$ 2,512,048。單維敏感度分析的結果，使用temsirolimus或干擾素-α治療的兩種不同方案間在整體存活危險比變化時相對於無疾病惡化存活危險比於每增加一個品質調整存活人年數之ICER值會產生較大的影響，也就是ICER值對於整體存活危險比的變化較為敏感。 依本研究之決策模式分析使用temsirolimus於治療晚期腎細胞癌之成本效益結果為每增加一個品質調整存活人年數的ICER值需要NT$4,775,609，依國際衛生組織所建議ICER值標準閾值作為比較參考，則本研究結果遠高於其建議之標準閾值，因而不具有成本效益。
並列摘要
Background: Renal cell carcinoma is a type of kidney cancer. One fourth of patients present with advanced disease, including locally invasive or metastatic RCC. The mainstay of systemic therapy for RCC has historically been immunotherapy with interferon-a in the past 20 years. The treatment of RCC has undergone an option shift over the past 5 years from immunotherapy to new targeted therapies. Evidence suggests that new target therapies offer clinical benefit; however, the financial implications of its use remain unclear. Decisions regarding health care provision are based increasingly on economic evaluations to identify treatments that provide the greatest clinical benefit at acceptable costs. The present study aimed to estimate the cost- effectiveness of temsirolimus as a treatment option in poor risk renal cell carcinoma compared with interferon-α and from the perspective of national health insurance in Taiwan. Methods: A decision-analytic model was developed to estimate the cost effectiveness of temsirolimus. Cost and the clinical effectiveness data were obtained from published literature and current reimbursed price published by Bureau of National Health Insurance. All costs and outcomes were discounted at an annual rate of 3%. The effect of parameter uncertainty on cost-effectiveness was explored through extensive one-way and probabilistic sensitivity analyses. Results: Compared with interferon-a, temsirolimus treatment resulted in an discounted incremental cost per QALY gained of NT$ 4,775,609, based on an estimated mean gain of 0.24 quality- adjusted life years (QALYs) per patient; and a cost per life-year gain of NT$ 2,512,048. Cost-effectiveness estimates were most sensitive to changes in hazard ratios for overall survival between temsirolimus and interferon-a. Conclusion: In comparison with interferon-a, the ICER (NT$4,775,609) for temsirolimus as a treatment in poor risk renal cell carcinoma, was found to be much higher than the threshold suggested by the World Health Organization. Therefore, temsirolimus could not be regarded as a cost-effective option from the National Health Insurance perspective.
參考文獻
延伸閱讀
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