以比較法觀點探討罕見疾病孤兒藥誘因

A Study on the Incentives of Orphan Drugs for Rare Diseases from the Angle of Comparative Law

2020年新冠肺炎肆虐之際，人們對於疫苗的需求以及藥物的近用重視程度不同於以往。罕見疾病由於病人之稀少，其治療上之地位時常被忽視，開發藥廠商常因為沒有經濟上的誘因，並不積極地開發新藥以治療罕見疾病，這樣被忽視的藥物稱之為「孤兒藥」，究竟要如何才能保障罕見疾病人的用藥權利，滿足其藥品近用（access to medicine），是孤兒藥法案所關注的議題。
自1983年美國通過世界上第一個孤兒藥法案之後，各國的立法於後便紛紛確立，包括日本、澳洲、歐盟等，我國亦不例外於2000年公布「罕見疾病防治及藥物法」，並陸續公布「罕見疾病防治及藥物法施行細則」、「罕見疾病醫療補助辦法」等，所提供之誘因包括研發租稅優惠、上市規費豁免、孤兒藥之資料專屬權（保障醫藥市場獨占地位）、政府研發補助、嘗試縮小孤兒藥的試驗規模等，本文透過考察此些制度之設計來探討其成效及有無改進之處，尤其在全民健康保險下是否影響罕見疾病病人的相關權益。

When the COVID pandemic is raging in 2020, the demand for vaccines and access to medicine are different from the past. Due to the scarcity of patients, the status of rare diseases in treatment is usually neglected. The pharmaceutical companies are not actively developing rare drugs for the treatment of rare diseases, because there are no sufficient incentives to invest research program, these drugs are called“orphan drugs”. How to protect the right of patients with rare diseases and the right of access to medicine is the issue of concern to the orphan drug bill.
Since the United States passed first orphan drug bill in 1983, legislation has been established in various countries, including Japan, Australia, and the European Union. Taiwan has also announced and passed“Rare Disease Prevention and Drug Law Enforcement Rules”,“Rare Disease Medical Subsidy Measures”, etc. The incentives provided include R&D tax concessions, listing fee exemptions, and orphan drug data exclusive rights (protecting the exclusive position of the pharmaceutical market), government R&D subsidies, furthermore, attempts to reduce the trial scale of orphan drugs. This article examines the design of these systems to explore whether their effectiveness has been improved, especially whether they have related rights and interests of rare disease patients under National Health Insurance.