英国约有350万罕见病患者 政府将大幅改革监管框架|国产做a爱片久久毛片a片古代

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  中新社伦敦11月2日电 (记者 欧阳开宇)英国政府11月2日发布消息称,将对罕见病治疗的监管框架进行大幅改革,旨在加快从研发到患者可及的全链条进程。这一举措是英国政府推进生命科学战略的重要组成部分。

  据英国药品和医疗产品监管局介绍,英国目前约有350万罕见病患者,仅有不到5%的罕见病拥有获批治疗方案,患者平均确诊耗时5.6年,30%的患病儿童在5岁前夭折。

  英国药品和医疗产品监管局称,罕见病不仅给患者家庭带来沉重负担,也对英国经济造成每年约149亿英镑(1394亿元人民币)的经济损失。

  为此,英国政府将主导成立一个新机构—罕见病治疗联盟,该联盟成员包括患者及其代表、学术界和产业界人士等,以共同应对罕见病挑战。

  与此同时,英国药品和医疗产品监管局提出多项创新监管思路。其中最引人关注的是探索“早期单一批准”机制——即在临床证据有限但极具前景的情况下,一次性批准临床试验与上市许可,并辅以严格的实时安全监测和评估,这将大幅简化审批流程。

  英国药品和医疗产品监管局表示,这项改革还将推动英国与全球范围内的证据共享,助力英国打造全球罕见病治疗创新标杆。

  多家患者组织和行业机构对此表示欢迎。英国基因联盟首席执行官尼克·米德表示,对许多家庭来说,罕见病确诊往往意味着没有有效的治疗方法。此项改革是改变现状的关键一步,也将增强在英国罕见病治疗方面的国际竞争力。(完)

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发布于:北京市