論罕見疾病藥物健保給付之現狀及改革方案

藥物是否得以認列為罕見疾病用藥、以及健保是否給付等，多取決於以科學醫療效益作為評估指標的專家審查會議，而罕病常遇到參與小型試驗人數太少、數據不完整以及療效不明確、罕病多是慢性的退化過程等困難，如未重視不同疾病族群與資源需求導致之差異而僅以單一事前審查判斷，便有可能對健康人權形成侵害。本文欲從國際上規定、英國及印度法院判決、我國司法院大法官解釋及學者意見觀察之，論述罕病患者請求健保給付為健康權所保障。其次，藥價保險支付是個複雜的政治問題，涉及一連串醫療科技、科學證據、成本和其他因素。缺乏單一及被普遍接受的健康收益閥值（health gain threshold）是我國當前評估健康介入以及衛生資源分配的一大障礙。期待我國健保支付制度改革，結合成本效益分析訂出明確閾值，也就是逐漸形成共識得到一個人年或一個健康人年(QALY)約要花費多少錢是能被全民健保制度接受的，日本學者對於高價新藥給付條件的不同觀點可供台灣學習，以兼顧健保資源有限及罕病藥物給付之權利保障。而我國二代健保規劃理念之一即為擴大社會多元化參與健保政策，如何使全民健保的政策制定更符合民主的原則，成為各界關注的問題，本文將一併介紹我國二代健保修法前後及英國NICE、加拿大CADTH有關病人參與之制度，望能提昇病人及病友團體的實質參與，能透過參與決策過程，讓病人行使其最基本權利，實踐人道主義精神。本文亦透過質性研究方法，實際深度訪談相關機構人員之內容列出我國罕見疾病藥物健保給付之現況及修改建議，望能將學術理論及實務結合，研究出更符合我國實務層面的修改建議。

關鍵字

健保給付；罕見疾病；病人參與；公民參與；健康權

並列摘要

Whether drugs are recognized as a rare disease medication, and whether health insurances reimburses the drugs, often depend on the mechanisms of scientific and medical expert review meetings. The policymaking for rare diseases is often based on insufficient evidence such as small number of small trials, incomplete data, unclear efficacy, and chronic degeneration process, etc. If we do not pay attention to the minority of disease groups and only make judgments by single factors, the decisions might violate the human rights of the minor disease groups. This article intends to protect the right to health insurance from the collection of relevant international laws , the British and Indian court judgments, the interpretation of the Judge of the Court of Justice, and the opinions of the scholars. Medicinal insurance reimbursement is a complex political issue involving a range of medical technology, scientific evidence, costs and other factors. The lack of a single and universally accepted health gain threshold is a major obstacle to the current assessment and the allocation of health resources. We hope the Taiwan's health insurance system will change to set a clear threshold based on a cost-benefit analysis approach, that is, the decision-makers will gradually achieve a consensus on the acceptable range for the yearly expense by a person or a healthy person (QALY). The views of new drugs reimbursement policy from some Japanese scholars are referential for Taiwan to make a better healthcare system which can balance the use of limited public health resources and the sufficient access to drugs for rare disease patients. One of the main ideas of the second-generation NHI is to expand the social diversity participation policy into the NHI. How to make the policies of NHI more in line with the principles of democracy is the primary concern. This research will analynize the impact of before-and-after implementation of health insurance policies in United Kingdom’s NICE, Canada’s CADTH, especially in the aspects that involving patient’s participation. In hope that it can improve patients and patient’s relatives’ experience by substantially involving them in the decision-making process, as such, patients can exercise the most of their basic human rights. This research also adopts a qualitative research method through intensive interviews with relevant institutions and staffs to understand the current situations in reimbursing rare disease drugs, and proposes suitable reformative recommendations. We hope that by combining academic theory and practice, the outcome of this research will provide significant contribution into our country’s healthcare system policy.