中国药事
中國藥事
중국약사
CHINESE PHARMACEUTICAL AFFAIRS
2014年
10期
1109-1113
,共5页
孤儿药%罕见病%药物研发%美国食品药品监管局%欧洲药品管理局
孤兒藥%罕見病%藥物研髮%美國食品藥品鑑管跼%歐洲藥品管理跼
고인약%한견병%약물연발%미국식품약품감관국%구주약품관리국
orphan drug%rare disease%drug development%US Food and Drug Administration%European Medicines Agency
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。
目的：通過對美國與歐盟對孤兒藥研髮上市相關管理政策的分析，為我國孤兒藥研髮上市管理提供藉鑒。方法分析美國和歐盟藥政管理部門公開的法規文獻和數據庫檢索數據，總結其對孤兒藥的激勵政策，分析穫得孤兒藥資格認定和批準上市的藥物特點。結果美國和歐盟對孤兒藥研髮均頒佈實施瞭諸如市場獨佔期、政府資助、審評專傢對研究方案的指導等相應的激勵政策。研髮機構應採取及早申請孤兒藥認定、多途徑髮現孤兒藥及孤兒藥的再開髮等研髮策略。結論美國和歐盟對孤兒藥的研髮與上市激勵政策，刺激瞭製藥企業對罕見病治療藥物的研髮熱情，有效緩解瞭罕見病無藥可治的現狀，對我國製定相關孤兒藥政策提供瞭有益的藉鑒。國內創新型製藥企業應儘早佈跼孤兒藥的研髮，應重點關註國內已經被大衆接受的罕見病和治療，以及超說明書使用的問題；重點關註生物倣製藥如單剋隆抗體的研髮動態；重點關註國際孤兒藥專業公司的研髮動態，使孤兒藥在國內相關法律法規建立健全後，立即有所響應，儘早搶佔孤兒藥研髮的領先地位和市場地位。
목적：통과대미국여구맹대고인약연발상시상관관리정책적분석，위아국고인약연발상시관리제공차감。방법분석미국화구맹약정관리부문공개적법규문헌화수거고검색수거，총결기대고인약적격려정책，분석획득고인약자격인정화비준상시적약물특점。결과미국화구맹대고인약연발균반포실시료제여시장독점기、정부자조、심평전가대연구방안적지도등상응적격려정책。연발궤구응채취급조신청고인약인정、다도경발현고인약급고인약적재개발등연발책략。결론미국화구맹대고인약적연발여상시격려정책，자격료제약기업대한견병치료약물적연발열정，유효완해료한견병무약가치적현상，대아국제정상관고인약정책제공료유익적차감。국내창신형제약기업응진조포국고인약적연발，응중점관주국내이경피대음접수적한견병화치료，이급초설명서사용적문제；중점관주생물방제약여단극륭항체적연발동태；중점관주국제고인약전업공사적연발동태，사고인약재국내상관법율법규건립건전후，립즉유소향응，진조창점고인약연발적령선지위화시장지위。
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.
