Core Insights - Approximately 100 rare disease medications have been included in the medical insurance catalog, covering 42 types of rare diseases [1] - In 2024, the medical insurance fund will pay 8.6 billion yuan for rare disease medications, accounting for 7.7% of the total payment for medications during the agreement period [1] - The establishment of a multi-layered guarantee system for rare diseases is underway, combining basic medical insurance, major illness insurance, and medical assistance [1][2] Group 1 - The National Healthcare Security Administration is actively promoting the inclusion of medications in the insurance catalog and the construction of a "dual-channel" supply mechanism to improve drug accessibility [1][2] - The number of hospitals in the national rare disease diagnosis and treatment collaboration network has increased from 324 in 2019 to 419 currently [1][2] - The national rare disease direct reporting system has registered a total of 1.64 million cases, with 1.5 million confirmed cases [2] Group 2 - Future selection criteria for rare disease medications should consider severity, intervention methods, and social impact, rather than just incidence rates [3] - The entry of rare disease medications into the catalog must prioritize clinical and patient benefits, focusing on drugs with clear biomarkers and measurable efficacy [3] - There is a need to challenge the perception that rare disease drugs must be high-priced, as some production technologies are mature and costs are controllable [3] Group 3 - Emphasis on refined management of rare disease medications is crucial due to their high cost and long-term prescription nature [3] - Collaboration among over 400 institutions in the national rare disease diagnosis and treatment collaboration network is essential for effective management practices [3][4] - The 2025 China Rare Disease Conference was co-hosted by multiple organizations, highlighting the collective effort in addressing rare diseases [4]

Core Insights - Hainan Boao Lecheng International Medical Tourism Pilot Zone is becoming a crucial link between rare disease drug companies and patients through policy iterations, insurance collaboration, and multi-dimensional empowerment [1] Policy Iteration - The policy framework for rare disease drug assurance in Lecheng has evolved through three versions: - Version 1.0 focuses on introducing licensed drugs and devices to allow some patients to access urgently needed medications [2] - Version 2.0 allows the use of real-world data from rare disease drugs for expedited domestic approval [2] - Version 3.0 expands the coverage to include special medical foods, health products, and new biomedical technologies [2] Special Drug Insurance - Lecheng has launched a special drug insurance plan to significantly reduce the financial burden on patients, with premiums starting at 49 yuan and coverage up to 2 million yuan for 120 types of global special drugs and 10 types of imported devices [3] Future Initiatives - Lecheng aims to strengthen the connection between rare disease drug companies and patients by leveraging special drug policies and introducing more special medical foods [4] - The zone has received approval for biomedical new technology policies, allowing clinical research and application transformation for technologies that have completed Phase II trials [4] - The initiative includes collaboration with the National Medical Products Administration and the National Healthcare Security Administration to expedite drug registration and insurance access [4]

Core Viewpoint - The accessibility of innovative drugs for rare disease patients is a significant barrier, prompting calls for a national-level rare disease special fund to alleviate financial burdens on patients and their families [1][2]. Group 1: Policy Recommendations - Establishing a national-level rare disease special fund is essential, with clear principles and development goals to integrate various compensation policies [1]. - A multi-faceted funding and payment mechanism involving health insurance, critical illness insurance, commercial insurance, medical assistance, and charitable funds is necessary to address the payment issues for high-value rare disease drugs [2]. Group 2: Regional Initiatives - Provinces like Jiangsu, Zhejiang, and cities like Qingdao have made significant strides in rare disease drug coverage, serving as models for others [2]. - Zhejiang Province's establishment of a rare disease drug guarantee fund in December 2019, which includes a personal payment cap, has shown stable operational effects and provides a potential framework for national implementation [2]. Group 3: Fund Management and Legislation - The rare disease special fund is viewed as an ideal payment solution, enhancing public awareness and supporting local innovation in drug development [3]. - The fund should be managed by provincial social security accounts, with diverse funding sources including government allocations, donations, and lottery funds [3]. - Emphasizing the importance of legislative support for the establishment of a sustainable payment mechanism, the fund should balance patient rights with equitable resource distribution [3].

Core Viewpoint - The establishment of a national rare disease special fund is essential to address the payment challenges associated with rare disease medications, as highlighted by the increasing number of drugs included in the medical insurance catalog but facing high costs that limit accessibility [1][2]. Group 1: Current Situation and Challenges - The Chinese government has shown increasing attention to rare diseases, with the introduction of the first and second batches of rare disease catalogs in 2018 and 2023, respectively [1]. - Despite the growing number of rare disease medications approved for inclusion in medical insurance, high-cost drugs remain difficult to incorporate into the catalog, leading to a growing contradiction between the availability of drugs and payment guarantees [1][2]. Group 2: Local Initiatives and Their Impact - Zhejiang Province established a rare disease medication guarantee special fund in December 2019, serving as a model for other regions and introducing a personal payment cap to alleviate patient burdens [2]. - Jiangsu Province has proposed a clear mechanism for rare disease medication guarantees, emphasizing a multi-channel funding approach involving government, market entities, and charitable organizations [2]. Group 3: Proposed Solutions and Benefits - The establishment of a national rare disease special fund could enhance the fairness and efficiency of medical insurance coverage for rare diseases and innovative drugs, ensuring that urgently needed foreign drugs reach domestic patients [3]. - A special fund would diversify funding sources and engage social resources in supporting rare disease medication, thus not relying solely on existing medical insurance funding models [3]. - Increasing public awareness of rare diseases and supporting local innovation in drug development are additional benefits of establishing a special fund [3].

Core Insights - The current challenges in rare disease medication include low accessibility, high financial burden, insufficient diagnostic and treatment systems, regional disparities in support, conflicts between payment systems and high drug prices, and inadequate incentives for drug research and market entry [1][3]. Group 1: Current Situation - There is a growing recognition of rare diseases in China, which presents opportunities for improving medication access [3]. - The existing issues significantly impact the guarantee of medication for patients with rare diseases [1]. Group 2: Policy Recommendations - The first recommendation is to innovate the insurance access model for rare diseases by adopting international risk-sharing agreements tailored to China's context, particularly for life-threatening conditions without equivalent treatments [3][4]. - The second recommendation involves optimizing outpatient insurance policies to create a multi-tiered support system that includes outpatient chronic disease coverage, major illness insurance, and supplementary insurance [4]. - The third recommendation is to establish a dedicated fund for rare disease medication, managed at the provincial level with independent accounting and funding from both government and social sources [4].

Core Viewpoint - The current challenges in rare disease medication include low accessibility, high burden, insufficient diagnostic and treatment systems, regional disparities in support, conflicts between payment systems and high drug prices, and inadequate incentives for drug research and market development [1][2]. Group 1: Current Challenges - Rare disease medications face low accessibility and high financial burden on patients [1]. - There is a lack of a comprehensive diagnostic and treatment system, leading to insufficient support capabilities [1]. - Disparities in medication support exist across different regions [1]. - There are conflicts between the payment systems and the high prices of medications [1]. - The current drug research and market incentives are insufficient to address the needs of rare diseases [1]. Group 2: Opportunities and Recommendations - Increased attention to rare diseases in China has created opportunities for medication support [2]. - Proposed measures include optimizing the entry process for rare disease medications and conducting price negotiations through the medical insurance system [2]. - A multi-layered medical security and comprehensive assistance system led by the government is necessary to address the challenges [2]. - Recommendations include innovating the medical insurance entry model by adopting international risk-sharing agreements tailored to China's context [2][3]. - Optimizing outpatient insurance policies to create a collaborative multi-layered security system is essential [3]. - Establishing a special fund for rare disease medication with provincial-level coordination and independent accounting is recommended [3].