大约80%的罕见病由单基因缺陷导致,约有一半的罕见病会影响儿童。很多罕见病属于严重或危及生命的病症,由于大多数罕见病没有获批的治疗用药,因此存在显著的未竟医疗需求。之前我们也在相关分析中指出,在研罕见病用药已占据超过三分之一的研发管线,但由于罕见病患者更难找到,临床试验招募难度更大。不少罕见病存在多个变异或亚型,患者临床表现和疾病进展率可能出现多样化,临床过程不可预测。因此,安全、有效罕见病用药开发存在挑战。我们期待由监管机构发布的针对具体罕见病用药临床研发的行业指南,能够有助于提高药物研发效率,为患者带来更多新药。
参考资料
[1] Fabry Disease: Developing Drugs for Treatment Guidance for Industry. AUGUST, 2019. Retrieved Aug 10, 2019 from https://www.fda.gov/regulatory-information/search-fda-guidance-documents/fabry-disease-developing-drugs-treatment-guidance-industry
[2] Human Gene Therapy for Rare Diseases. Draft Guidance for Industry. July, 2018. Retrieved Aug 10, 2019 from https://www.fda.gov/media/113807/download
[3] Galafold Approved to Treat Fabry Teens and Adults in Argentina, Amicus Announces. Retrieved Aug 12, 2019 from https://fabrydiseasenews.com/2019/08/07/galafold-approved-in-argentina-to-treat-fabry-disease-amicus-announces/
原标题:视点 | FDA发布法布里病指南草案,这几点要注意!
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