細胞與基因治療的技術發展與支付體系探討

Abstract

細胞與基因治療（CGT）作為當代醫療創新的關鍵技術，具備根治罕見疾病與重大疾病的潛力。然而，其研發與商業化過程仍受到臨床不確定性、法規審查與支付機制限制的挑戰。本研究採用混合研究方法，結合專利分析、臨床試驗數據、國際制度比較與專家訪談，選取腺相關病毒（AAV）、嵌合抗原受體T細胞（CAR-T）、常間回文重複序列叢集關聯蛋白（CRISPR）、間質幹細胞（MSC）、自然殺手細胞（NK cell）與誘導性多能幹細胞（iPSC）等六種代表性技術，建構細胞基因療法的發展分類與成熟度評估架構。 透過美國、歐盟與日本的制度比較，本研究揭示不同法規與支付設計在CGT產品核准的偏好及產業發展的影響。最後特別聚焦於台灣現行雙軌制制度，並提出兼顧產業誘因與發展之建議。研究認為，影響CGT普及的關鍵並非僅止於技術發展，更取決於法規彈性、支付體系成熟度與整體政策前瞻性。

Cell and gene therapies (CGTs) represent a new frontier in biomedical innovation, offering curative potential for diseases once considered intractable. However, the development and delivery of CGTs remain constrained by uncertainties in regulation, reimbursement, and clinical translation. This study establishes an integrated analytical framework combining patent analysis, clinical trial mapping, and international policy comparison to examine six representative CGT modalities: AAV, CAR-T, CRISPR, MSC, NK cell, and iPSC. The analysis reveals differentiated technology trajectories and clinical maturity levels, which are used to develop a strategic group typology of CGT development. A cross-jurisdictional comparison of the United States, the European Union, and Japan further uncovers the heterogeneous structures of approval pathways and payment mechanisms. Particular attention is paid to Taiwan’s dual-track regulatory system and its implications for local innovation. The study proposes strategic recommendations for Taiwan’s CGT governance, including regulatory alignment, HTA reform, data stewardship, and global standard integration of delivery capabilities. The findings suggest that beyond technology, institutional readiness and policy foresight are decisive in shaping the future of CGT accessibility and affordability.