歐盟罕病藥物供給、研發政策法規與藥法規與藥品市場現況分析

A Review to the Provision of Orphan Drugs Supply, the Development of Orphan Medicinal Products and Markets in European Union

孤兒藥（罕病藥）為罕病患者的必要醫療供給之一，而對於歐盟27個會員國的龐大孤兒藥市場供給、研發狀況，及其政策法規發展為何是本研究所欲了解，本文主要目的是藉由資料收集與分析，探討歐盟孤兒藥的相關政策與法律及歐洲孤兒藥市場之現況，以了解歐盟孤兒藥之市場發展。研究方法為檢索國內、外之文獻，並做系統性的整理，來呈現相關資料。研究結果顯示歐盟的孤兒藥法涵蓋孤兒藥物委員會之成立、孤兒藥物查驗登記、孤兒藥市場上市程序之輔導協助、孤兒藥上市後10年的市場授權鼓勵措施，以及中小企業在孤兒藥研發時的保障規範；在孤兒藥市場上，可看到歐盟自2000年指定孤兒藥數目逐年增加，且在孤兒藥市場的成本花費上則有逐年增加之趨勢。歐盟的孤兒藥研發相關法規政策相較於台灣來得成熟許多，若台灣欲改變目前孤兒藥只能由外商供應之現況，政府仍扮演相當重要角色，我國藥物政策發展方向可試著積極經營。

Orphan drug (rare disease drug) is necessary medication for people with rare disease. This review aims to understand the orphan drugs supply, scientific development regulation and drug market in European Union(EU) based on the analysis of recent studies or reports.The results show the EU's legislation is administered by the Committee on Orphan Medicinal Products of the European Medicines Agency, and the EUenacted regulationpursuant to which pharmaceuticals developed to treat rare diseases are referred to as "orphan medicinal products." Orphan drug status granted by the European Commission gives marketing exclusivity in the EU for 10 years after approval. The type of approved orphan drugs and drug cost expenditures are increasing gradually. The scientific development and regulations are well established and provide incentives for the development of orphan drugs in the EU. The orphan drug management system can be a good reference for Taiwan's future health initiatives for patients with rare diseases.