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上市和报销，在某种程度上打造报销机构、企业和患者                                praising+ultra-orphan+drugs&cmd=Search%21&wf=
          等各利益相关方多方共赢的局面。                                         4BFF&s=R&db=&de=.
              总的来说，在罕见病用药评估与报销过程中，通过                         [11]  National Institute for Health and Care Excellence（NICE）.
          纳入新的药物准入评价体系，再加上一定的政策倾斜，                                Interim  process  and  methods  of  the  Highly  Specialised
          将对我国罕见病用药可及性的提高起到促进作用。在                                 Technologies  programme[EB/OL].  [2022-09-12].  https：//
                                                                  www.nice.org.uk/Media/Default/About/what-we-do/NICE-
          当前罕见病用药的研发成本高、销售量少的大环境下，
                                                                  guidance/NICE-highly-specialised-technologies-guidance/
          中国罕见病用药准入政策的完善可参考英国等国家，依
                                                                  HST-interim-methods-process-guide-may-17.pdf.
          然需要监管部门和制药企业共同努力推进罕见病用药
                                                             [12]  National Institute for Health and Care Excellence（NICE）.
          在中国的使用。我国在帮助各方利益相关者更好地了
                                                                  Guidance  and  advice  list[EB/OL].  [2022-09-15].  https：//
          解罕见病用药需求的基础上，建立单独的评估路径是解                                www.nice.org.uk/guidance/published?type=hst.
          决我国罕见病患者用药可及性的关键手段。                                [13]  National  Health  Service.  Specialized  services[EB/OL].
          参考文献                                                    [2022-09-15]. https：//www.england.nhs.uk/commission‐
          [ 1 ]  RHEE T G. Policymaking for orphan drugs and its chal‐  ing/spec-services/key-docs/#scog.
              lenges[J]. AMA J Ethics，2015，17（8）：776-779.    [14]  National Health Service. Spotlight on specialised services
          [ 2 ]  Scottish  Medicines.  PACE （Patient  &  Clinician  Engage‐  [EB/OL]. [2022-09-18]. https：//www.england.nhs.uk/wp-
              ment）  overview  document[EB/OL].  [2022-08-22]. https：//  content/uploads/2018/09/spotlight-on-specialised-services-
              www.scottishmedicines.org.uk/media/2782/pace-overview-  second-edition.pdf.
              document.pdf.                                  [15]  NICOD E，ANNEMANS L，BUCSICS A，et al. HTA pro‐
          [ 3 ]  Organisation  for  Economic  Co-operation  and  Develop‐  gramme response to the challenges of dealing with orphan
              ment （OECD）.  Health  expenditure  and  financing[EB/  medicinal  products：process  evaluation  in  selected  Euro‐
              OL]. [2022-08-24]. https：//stats.oecd.org/Index.aspx?Da‐  pean countries[J] Health Policy，2019，123（2）：140-151.
              tasetCode=HEALTH_STAT.                         [16]  Scottish  Medicines.  Patient  and  Clinician  Engagement
          [ 4 ]  National statistics. Healthcare expenditure，UK Health[EB/  （PACE）  process[EB/OL].  [2022-09-18].  https：//www.
              OL]. [2022-08-25]. https：//www.ons.gov.uk/peoplepopula‐  scottishmedicines.org.uk/how-we-decide/pace/.
              tionandcommunity/healthandsocialcare/healthcaresystem/  [17]  Scottish Government. Review of access to new medicines
              bulletins/ukhealthaccounts/2017#how-healthcare-in-the-uk-  [EB/OL]. [2022-09-22]. https：//www.gov.scot/publications/
              is-financed.                                        review-access-new-medicines/pages/11/.
          [ 5 ]  Department of Health.The NHS constitution for England  [18]  Scottish  Government. Treatments for  rare  conditions[EB/
              [EB/OL]. [2022-08-26]. https：//www.gov.uk/government/  OL].[2022-09-22]. https：//news.gov.scot/news/treatments-
              publications/thenhs-constitution-for-england/the-nhs-  for-rare-conditions.
              constitution-for-england.                      [19]  Scottish  Medicines.  Revised  process  ultra-orphan  medi‐
          [ 6 ]  National Statistics. Prescriptions dispensed in the commu‐  cines for extremely rare conditions[EB/OL]. [2022-09-25].
              nity[EB/OL]. [2022-09-10]. https：//files.digital.nhs.uk/  https：//www.scottishmedicines.org.uk/how-we-decide/
              publication/s/o/pres-disp-com-eng-2006-16-rep.pdf.  revised-process-ultra-orphan-medicines-for-extremely-rare-
          [ 7 ]  袁妮，田婷婷，张海军，等. 英国罕见病用药纳入报销体                       conditions/.
              系的主要途径及对我国的启示[J]. 中国药科大学学报，                    [20]  Scottish Medicines. Guidance to submitting companies for
              2019，50（1）：113-119.                                 completion  of  New  Product Assessment  Form （NPAF）：
          [ 8 ]  张海军，梁云，袁妮 . 英国 NICE 罕见病用药卫生技术评                   supplement  for  medicines  for  extremely  rare  conditions
              估流程介绍及其启示[J]. 中国新药杂志，2018，27（14）：                  （ultra-orphan medicines）[EB/OL]. [2022-09-25]. https：//
              1587-1594.                                          www.scottishmedicines.org.uk/media/4155/guidance-
          [ 9 ]  宣建伟，孙巧 . 中国罕见病药物经济学评估适用模型与                       supplement-ultra-orphan.pdf.
              支付阈值参考标准探讨[J].国际药学研究杂志，2019，46                 [21]  李乐乐，何晓彤，陈湘妤，等. 我国罕见病用药保障现状
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          [10]  ADRIAN T，MARTINA G. Appraising ultra-orphan drugs：  [22]  袁帅，吴应宇. 我国罕见病用药的医疗保险准入评价指
              is  Cost-Per-QALY  appropriate?  a  review  of  the  evidence  标体系构建[J]. 中国卫生资源，2021，24（6）：646-649.
              [EB/OL].[2022-08-26]. https：//ideas.repec.org/cgi-bin/ht‐     （收稿日期：2022-12-12  修回日期：2023-05-14）
              search?form=extended&wm=wrd&dt=range&ul=&q=ap‐                                      （编辑：刘明伟）







          中国药房  2023年第34卷第13期                                              China Pharmacy  2023 Vol. 34  No. 13    · 1561 ·
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