方案发布：北京市罕见病药品保障先行区建设全面实施

2023年8月7日，北京市药监局、北京市卫健委、天竺综合保税区管理委员会（天竺管委会）等多部门联合发布了《北京市推动罕见病药品保障先行区建设工作实施方案（试行）》（以下简称“实施方案”）征求意见稿。公开征集意见时间为：2024年8月7日至2024年8月13日。

2023年11月，国务院首次提出在北京天竺开展罕见病药品保障先行区建设，引进临床急需境外已上市、境内未上市的罕见病药品。自此，政府及各方启动了一系列准备工作。今年3月，位于天竺的北京罕盟诊所正式开业，成为北京罕见病药品保障试点工作的重要里程碑。4月，北京市宣布，今年推动10个临床急需品种进口。

政策亮点

实施方案重点强调了三个定期动态调整的白名单，分别针对医疗机构、罕见病临床急需药品和药品进口企业，以高效引进临床急需的境外已上市药品，满足患者需求。

北京市卫健委：医疗机构白名单

北京市卫健委将遴选和认定纳入名单的医疗机构。纳入“白名单”的医院将在北京市卫健委指导下，直接向国家药监局申请进口罕见病临床急需药品。入选资格强调服务罕见病患者和采购进口药品的能力，且优先考虑三类医院：北京市罕见病诊疗协作网成员，国家医学中心的主体单位和北京市国际医疗试点。北京市卫健委将在10个工作日内完成申请评估，相关部门将在3个工作日内进行集体审议并做出最终决定。

北京市药监局：罕见病临床急需药品白名单

北京市卫健委在收集“白名单”医院的需求并指导其向国家药监局申请后，国家药监局将在咨询北京市卫健委意见的基础上，批准符合要求的申请，并最终由北京市药监局以“白名单”形式发布。入选 “白名单”的药品将在北京天竺口岸一次通关多次进口，并豁免进口检验，从而为进口企业节省大量资金。因为罕见病患者少而药品单价高，某些药品抽样比例高达40%甚至超过50%，为企业带了极大的经济负担。

天竺管委会：药品进口企业白名单

天竺管委会将遴选和认定药品进口企业，并负责管理相关“白名单”。入选白名单的进口企业将有资格开展储存、进口和配送等高效业务。医院可以委托“白名单”企业办理通关文件，也可以自行办理。

展望

在满足患者需求方面，实施方案的发布彰显了北京市政府在多渠道联动、增强罕见病患者福祉领域的努力。北京市鼓励医疗机构、药品进口企业、保险机构通过商业保险提供医责险、罕见病用药风险补偿等保险服务。同时，北京将探索建立罕见病基金专项制度，发挥社会多元力量的作用。

在产业发展方面，北京致力于在天竺综合保税区打造罕见病医药成果转化示范基地，并支持境外罕见病药品在京注册上市。

7月下旬，基誉传播和来访的国际客户有幸与北京市卫健委会面，讨论了这一尚未公开发布的文件及北京市卫健委的重要作用。随着公开征求意见的结束和正式文件的最终发布，我们预计罕见病临床急需药品白名单将随后发布，惠及众多患者。

On August 7, the Beijing Municipal Medical Products Administration (BMMPA), the Beijing Municipal Health Commission (BMHC), the Tianzhu Comprehensive Bonded Zone Management Committee (TMC), and other municipal authorities jointly released the draft Implementation Plan for the Development of the Beijing Rare Disease Drug Security Pilot Zone (“Implementation Plan”) for public consultation. Call for comments ended on August 13.

In November 2023, the State Council proposed a pilot program in Beijing Tianzhu to address the growing demand for life-saving therapies among rare disease patients. Since then, a series of initiatives were launched, with the opening of HanMeng Clinic in Tianzhou this March marking a key milestone in preparation for the pilot zone. Meanwhile, Beijing aims to introduce ten innovative drugs via an accelerated pathway made possible by the Tianzhu pilot zone this year.

Highlights of the Implementation Plan

The Implementation Plan features three whitelists — for hospitals, drugs, and importers—that will be updated regularly. These lists will form part of a program to address patients’ immediate needs for urgent drugs not yet available in China.

BMHC's whitelist of medical institutions

BMHC will select a list of hospitals that can apply directly to the National Medical Products Administration (NMPA) for the importation of urgently needed drugs.

While qualifications for enlisting highlight the capability to serve rare disease patients and procure imported medicines, BMHC will prioritize three categories of hospitals. These include 29 rare disease hospitals announced by BMHC, hospitals that host national medical centers, and the 17 pilot institutions designated for Beijing’s international medical services. BMHC will review the applications submitted by hospitals seeking inclusion on the list in ten workdays. All involved authorities must then make a final decision within three workdays.

BMMPA's whitelist of urgently needed drugs

BMHC will first assess whitelisted hospitals’ needs and will then direct these hospitals to submit their importation requests to NMPA. Upon receiving the greenlight from NMPA, BMMPA will release a whitelist of medicines approved for import.

Imported drugs can be processed with the same documentations for multiple customs clearances without requiring port inspections, which usually test a large portion of the shipment. This will result in significant time and cost savings for importers.

TMC's whitelist of importers

TMC will select a list of qualified importers who will benefit from efficient storage, importation, and distribution services. Hospitals may either work with these suppliers to import needed drugs or import directly.

Outlook

The Implementation Plan is one of several strategies by Beijing municipal authorities to enhance patient wellbeing. Market forces are encouraged to develop additional insurance options, including liability insurance and medical risk coverage. Additionally, the deputy director of BMHC announced plans to set up a special fund for rare disease drugs in the long term. 

In terms of industry development, Beijing intends to strengthen the industry base by developing a demonstration project for medical research transformation. Additionally, the city will support the registration and release of imported drugs in Beijing.   

Recently, Sandpiper and its client had the privilege of meeting with BMHC to discuss some of the above policies and better understand BMHC's important role in the healthcare policy ecosystem. With a short call-for-comment period and the Implementation Plan currently being formulated, we anticipate a swift release of the imported drugs whitelist.