全球公共健康议题持续演进，中国罕见病领域进入结构重塑阶段。《2026中国罕见病行业趋势观察报告》围绕政策框架、诊疗体系、创新研发与支付机制展开系统研究，呈现行业由制度建设向体系协同转型的发展轨迹。
国家罕见病目录的完善与多层次保障机制的构建，推动治理结构升级。创新药物研发能力提升，本土企业参与度增强，行业驱动力由引进依赖转向创新主导。多学科诊疗体系的深化强化了能力基础，运行质量成为新的发展重点。
治疗可及性与支付效率构成当前阶段的核心变量。制度成熟度提升背景下，协同效率、创新转化速度与患者获得感成为决定行业长期稳定性的关键因素。
中国罕见病行业正形成更加系统化、结构化的发展格局，高质量发展趋势逐步确立。
The global public health landscape continues to evolve, and China’s rare disease sector is entering a phase of structural transformation. The 2026 China Rare Disease Industry Trend Observation Report provides a systematic analysis of policy frameworks, clinical systems, innovation capacity, and reimbursement mechanisms, outlining the industry’s transition from institutional establishment to coordinated system development.
The refinement of the national rare disease catalogues and the advancement of multi-layered support mechanisms are driving governance upgrades. Innovation capability is strengthening, domestic participation is expanding, and industry momentum is shifting from import reliance toward innovation leadership. The deepening of multidisciplinary clinical systems reinforces foundational capacity, placing operational quality at the center of development.
Treatment accessibility and reimbursement efficiency represent the defining variables of the current stage. As institutional maturity increases, coordination efficiency, innovation translation speed, and patient-level outcomes emerge as key determinants of long-term industry stability.
China’s rare disease sector is forming a more structured and integrated development framework, with high-quality growth increasingly established as the prevailing trajectory.
