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孤儿药的中文命名,源于它的英文——Orphan Drug,它是所有罕见病治疗药物和治疗手段的统称。由于罕见病患病群体小、药品市场需求少、研发成本高,因此孤儿药异常稀少。这样的命名极为形象,也分外悲伤。“孤儿”一词,通常意味着某个不知晓其父母的小孩。其实,研发孤儿药也需要确认许多扑朔迷离的罕见病致病机理,需要进行多学科、跨领域联合研究,需要更加紧密的患者信息收集、疾病诊断、研发政策扶持和审批机制。此外,孤儿药由于研发成本高昂,导致其售价昂贵,也需要良好的商业保险政策来支付天价的治疗费用。
The orphan drug’s Chinese name, derived from its English - Orphan Drug, which is a generic term for all rare medical and therapeutic treatments. Due to the small population of rare diseases, low drug market demand and high research and development costs, orphan medicines are extremely rare. This name is very image, but also exceptionally sad. The word “orphan” usually means a child who does not know his or her parents. In fact, the development of orphan drugs also need to confirm the many complicated and pathogenic mechanisms of rare diseases, the need for multidisciplinary and cross-cutting joint research, the need for closer patient information collection, disease diagnosis, R & D policy support and approval mechanism. In addition, orphan drugs are costly due to high R & D costs and also require good commercial insurance policies to cover the high cost of treatment.