Investigation and analysis of the current status and challenges in importing rare disease drugs in China

WU Jingjing; SU Qinning; TAO Xueyi; YANG Yufei; MAO Ningying

doi:10.6039/j.issn.1001-0408.2025.17.03

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Investigation and analysis of the current status and challenges in importing rare disease drugs in China

更新时间：2025-09-29

- Investigation and analysis of the current status and challenges in importing rare disease drugs in China
- ZHONGGUO YAOFANG Vol. 36, Issue 17, Pages: 2097-2101(2025)
- 作者机构：
  
  1.中国药科大学国际医药商学院，南京　211198
  2.中国药科大学国家药物政策与医药产业经济研究中心，南京　211198
- 作者简介：
- 基金信息：
- DOI：10.6039/j.issn.1001-0408.2025.17.03
  CLC： R95
- Received：07 March 2025，
  
  Revised：2025-07-11，
  
  Accepted：11 July 2025，
  
  Published：15 September 2025
- 稿件说明：
移动端阅览
邬镜净,苏沁凝,陶雪怡,等.我国罕见病药品进口现状及挑战的调研分析[J].中国药房,2025,36(17):2097-2101.

WU Jingjing,SU Qinning,TAO Xueyi,et al.Investigation and analysis of the current status and challenges in importing rare disease drugs in China[J].ZHONGGUO YAOFANG,2025,36(17):2097-2101.
邬镜净,苏沁凝,陶雪怡,等.我国罕见病药品进口现状及挑战的调研分析[J].中国药房,2025,36(17):2097-2101. DOI： 10.6039/j.issn.1001-0408.2025.17.03.

WU Jingjing,SU Qinning,TAO Xueyi,et al.Investigation and analysis of the current status and challenges in importing rare disease drugs in China[J].ZHONGGUO YAOFANG,2025,36(17):2097-2101. DOI： 10.6039/j.issn.1001-0408.2025.17.03.

摘要

目的

分析我国当前罕见病药品进口的现状及所面临的挑战，为优化罕见病药品进口流程和完善相关政策提供参考。

方法

对罕见病药品进口环节涉及的政府部门、跨国制药企业、医疗机构和患者组织相关方展开问卷调研和访谈，探析各方在罕见病药品进口环节的现状及遇到的挑战，并综合专家意见提出改进建议。

结果

通过对25家罕见病领域的跨国制药企业相关负责人展开问卷调研，发现企业进口罕见病药品意愿强烈，58.33%的企业开展了多元进口模式实践，但在实际进口过程中面临制度不清晰（54.17%）、实际审批程序复杂（45.83%）、实际审批周期过长（41.67%）等系列问题，影响了企业进口的积极性。同时，对13位来自政府部门、医疗机构、制药企业和患者组织的罕见病领域专家展开访谈调研，发现目前我国在罕见病药品进口的政策设计、审批流程、抽检成本及与监管方的沟通效率等方面存在不足，医疗特区的药品进口模式也有待完善。

结论

我国罕见病药品在进口环节还面临着政策制度不完善、监管机制不灵活、沟通渠道缺乏等问题。建议从完善罕见病认定机制、优化进口激励政策和完善监管模式等方面出发，进一步优化罕见病药品进口流程并完善相关政策。

Abstract

OBJECTIVE

To analyze the current status and challenges in importing rare disease drugs in China， providing references for optimizing the import process and improving relevant policies.

METHODS

Questionnaires and interviews were conducted with stakeholders involved in rare disease drug importation， including government departments， multinational pharmaceutical enterprises， healthcare institutions， and patient organizations. This explored the current situation and challenges encountered by each party. Expert opinions were synthesized to propose improvement suggestions.

RESULTS

A questionnaire survey of representatives from 25 multinational pharmaceutical companies in the rare disease field revealed that these companies had a strong willingness to import rare disease drugs， with 58.33% of them practicing diverse import models. However， significant challenges hindered this process， including unclear regulations （54.17%）， complex approval procedures （45.83%）， and excessively long approval cycles （41.67%）， negatively impacting their motivation. Meanwhile， interviews with 13 experts from government departments， healthcare institutions， pharmaceutical enterprises， and patient organizations identified deficiencies in policy design， approval processes， sampling inspection costs， and communication efficiency with regulators. Additionally， the drug import model in special medical zones also required improvement.

CONCLUSIONS

The importation of rare disease drugs in China faces challenges such as incomplete policies， inflexible regulatory mechanisms， and insufficient communication channels. It is recommended to enhance the rare disease definition criteria， optimize import incentive policies， and refine regulatory models， so as to further optimize the import process of rare disease drugs and improve relevant policies.

关键词

Keywords

references

ABOZAID G M ， KERR K ， MCKNIGHT A ， et al . Criteria to define rare diseases and orphan drugs：a systematic review protocol [J ] . BMJ Open ， 2022 ， 12 （ 7 ）： e062126 .

EDVARD SMITH C I ， BERGMAN P ， HAGEY D W . Estimating the number of diseases：the concept of rare，ultra-rare，and hyper-rare [J ] . iScience ， 2022 ， 25 （ 8 ）： 104698 .

RAJASIMHA H K ， SHIROL P B ， RAMAMOORTHY P ， et al . Organization for Rare Diseases India （ORDI）： addressing the challenges and opportunities for the Indian rare diseases’ community [J ] . Genet Res （Camb）， 2014 ， 96 ： e009 .

金敏，马爱霞 . 江苏省罕见病保障政策与孤儿药供应保障模式研究 [J ] . 中国药物经济学， 2019 ， 14 （ 3 ）： 93 - 101 .

过其祥，刘云鹏，朱珈震，等 . 基于Orphanet数据库的中国罕见病界定及累积时点患病率测算 [J ] . 中国新药杂志， 2025 ， 34 （ 1 ）： 6 - 11 .

任淑月，孙岚，杜冠华 . 重定位策略在罕见病治疗药物中的应用研究进展 [J ] . 江苏大学学报（医学版）， 2021 ， 31 （ 1 ）： 17 - 22 .

中国罕见病联盟 . 中华人民共和国中央人民政府国务院常务会议 [EB/OL ] .（ 2019-02-12 ）[ 2024-11-13 ] . https：//www.chard.org.cn/list/news/15/28 https://www.chard.org.cn/list/news/15/28 .

第一财经 . 30 种罕见病“境外有药，境内无药”，进博如何提速罕见病用药保障 [EB/OL ] . （ 2024-11-09 ）[ 2025-03-25 ] . https：//m.yicai.com/news/102351030.html https://m.yicai.com/news/102351030.html .

药智网 . 第二批罕见病目录：18种罕见病“境外有药，境内无药”、10+种罕见病新药国内将上市 [EB/OL ] . （ 2023-10-17 ）[ 2024-10-18 ] . https：//mp.weixin.qq.com/s/OLvPUVwcGsqLbptA_UFTHQ https://mp.weixin.qq.com/s/OLvPUVwcGsqLbptA_UFTHQ .

FELLOWS G K ， HOLLIS A . Funding innovation for treatment for rare diseases：adopting a cost-based yardstick approach [J ] . Orphanet J Rare Dis ， 2013 ， 8 ： 180 .

黄哲，曲若暄，兰毅鹏，等 . 罕见病药品进口流程与监管建议研究 [J/OL ] . 沈阳药科大学学报， 2023 ： 1 - 7 （ 2023-11-09 ）[ 2025-03-25 ] . https：//link.cnki.net/doi/10.14066/j.cnki.cn21-1349/r.2023.0295 https://link.cnki.net/doi/10.14066/j.cnki.cn21-1349/r.2023.0295 .

World Health Organization . Anatomical therapeutic chemical （ATC） classification [EB/OL ] . [ 2025-06-08 ] . https：//www.who.int/tools/atc-ddd-toolkit/atc-classification https://www.who.int/tools/atc-ddd-toolkit/atc-classification .

邬镜净，袁惠琴，田梦妍，等 . 美国罕见病药物研发激励政策实践及启示：基于药品全生命周期视角 [J ] . 中国新药杂志， 2024 ， 33 （ 20 ）： 2081 - 2087 .

European Medicines Agency . Mutual recognition agreements （MRA） [EB/OL ] . （ 2025-07-05 ）[ 2025-07-11 ] . https：//www.ema.europa.eu/en/human-regulatory-overview/research-development/compliance-research-development/good-manufacturing-practice/mutual-recognition-agree- ments-mra https://www.ema.europa.eu/en/human-regulatory-overview/research-development/compliance-research-development/good-manufacturing-practice/mutual-recognition-agree-ments-mra .

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