美国与欧盟孤儿药研发上市管理制度及对我国的启示

目的通过对美国与欧盟对孤儿药研发上市相关管理政策的分析,为我国孤儿药研发上市管理提供借鉴。方法分析美国和欧盟药政管理部门公开的法规文献和数据库检索数据,总结其对孤儿药的激励政策,分析获得孤儿药资格认定和批准上市的药物特点。结果美国和欧盟对孤儿药研发均颁布实施了诸如市场独占期、政府资助、审评专家对研究方案的指导等相应的激励政策。研发机构应采取及早申请孤儿药认定、多途径发现孤儿药及孤儿药的再开发等研发策略。结论美国和欧盟对孤儿药的研发与上市激励政策,刺激了制药企业对罕见病治疗药物的研发热情,有效缓解了罕见病无药可治的现状,对我国制定相关孤儿药政策提供了有益的借鉴。国内创新型制药企业应尽早布局孤儿药的研发,应重点关注国内已经被大众接受的罕见病和治疗,以及超说明书使用的问题;重点关注生物仿制药如单克隆抗体的研发动态;重点关注国际孤儿药专业公司的研发动态,使孤儿药在国内相关法律法规建立健全后,立即有所响应,尽早抢占孤儿药研发的领先地位和市场地位。

Policies on Orphan Drug Development in USA and European Union and their Implications to China

Objective To help China develop some encouraging policies on orphan drug development by analyzing the policies of orphan drug development of USA and European Union.Methods Relative orphan drug policies of US Food and Drug Administration and European Medicines Agency were searched,as well as orphan drug designations and approvals by FDA and EMA,and the encouraging policies were summarized and the characteristics of orphan drugs for qualification and marketing approval were analyzed.Results FDA and EMA have issued some policies to encourage orphan drug development such as market exclusivity,government funds,FDA‘s advising and so on.For drug researchers,some measures should be taken to promote research productivity including applying orphan drug qualification as early as possible,discovering orphan drug by multiple ways and redeveloping some orphan drugs.Conclusion Encouraging policies on orphan drug development of USA and European Union stimulated the enthusiasm of pharmaceutical companies to develop drugs for rare diseases,effectively alleviated the situation of no cure for rare diseases,and provided contributive reference for our country to develop relative policies for orphan drugs.Chinese pharmaceutical companies should focus on policy development and drug development trends especially in some well known rare diseases and their therapies including off-label use,pay close attention to development in biosimilar products,particularly monoclonal antibodies,and try to catch the development opportunities when relative laws are improved.