美国食品和药物管理局提出了对罕见疾病的快速基因疗法, FDA proposes fast-tracking gene therapies for rare diseases with strong scientific basis.

美国林业发展局提出了一条新的监管途径,以快速批准针对稀有疾病的定制基因疗法,允许根据可信的生物机制和针对该疾病根源的证据(即使患者数据有限)进行批准。 The U.S. FDA has proposed a new regulatory pathway to fast-track approval of customized gene therapies for rare diseases, allowing approval based on a plausible biological mechanism and evidence of targeting the disease’s root cause, even with limited patient data. 这一行动旨在加快获得治疗的机会,如在传统大规模审判不切实际的极端恶劣条件下,采用基于CRISPR的治疗疗法。 The move aims to accelerate access to treatments like CRISPR-based therapies for ultra-rare conditions where traditional large-scale trials are impractical. 它使核准的治疗方法商业化——这与目前的同情性使用规则不同——并遵循最近的变化,以减少标准审判要求。 It enables commercialization of approved therapies—unlike current compassionate use rules—and follows recent changes to reduce standard trial requirements. 该指南草案开放60天供公众评论,只适用于有坚实科学依据的众所周知的遗传病,不适用于常见疾病。 The draft guidance, open for public comment for 60 days, applies only to well-understood genetic disorders with strong scientific rationale and is not intended for common diseases.