英國監管機構批准用於治療罕見遺傳疾病的新療法
UK Regulators Approve New Treatment for Rare Genetic Condition
英國在醫療保健上的作法正在轉變，英國藥品及保健產品管理局正為罕見疾病引入一套具變革性的框架。
The UK's approach to healthcare is changing as the Medicines and Healthcare products Regulatory Agency (MHRA) introduces a transformative framework for rare diseases.
由於目前只有不到5%的罕見疾病有核准的治療方法，英國正從「一體適用」的模式轉向客製化、科技驅動的系統。
With fewer than 5% of rare conditions currently having approved treatments, the UK is moving away from a one-size-fits-all model toward a bespoke, tech-driven system.
這個新時代旨在減少許多患者所面臨、令人痛苦長達五年的「診斷奧德賽」(即求醫歷程)。
This new era aims to reduce the agonizing five-year 'diagnostic odyssey' that many patients face.
儘管維護科學完整性與安全性仍是首要任務，MHRA正利用「創新護照」(Innovation Passports)等工具來快速推動具潛力的藥物。
While maintaining scientific integrity and safety remains the top priority, the MHRA is utilizing tools like 'Innovation Passports' to fast-track promising medicines.
透過運用人工智慧、基因組學研究與真實世界數據，英國正將自身定位為全球醫療創新的領導者。
By leveraging AI, genomic research, and real-world data, the UK is positioning itself as a global leader in medical innovation.
