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专访阿斯利康全球高级副总裁Gianluca Pirozzi:中国正成为全球罕见病研发“突破引擎” 高价值药物有望构建商业闭环

Core Insights - The article highlights the dual challenges faced by rare disease patients, including difficulties in diagnosis and lack of effective treatments, with over 90% of known rare diseases lacking any therapeutic options [2][3] - China is evolving from a participant in global rare disease research to a "breakthrough engine," leveraging its unique advantages in clinical trial efficiency, technological innovation, and disease data accumulation [2][4] Rare Disease Landscape - There are over 10,000 known rare diseases, with 90% lacking effective treatments and 80% being genetic, often manifesting in childhood, making pediatric rare disease drug development urgent yet complex [3][4] - The definition of rare diseases varies significantly across countries, complicating research and development efforts, particularly in China, where a clear regulatory list of rare diseases is still absent [3][4] China's Role in Rare Disease Research - China is recognized for its leading clinical trial speed and quality, with many Phase III trials involving Chinese participants, addressing the recruitment challenges faced in rare disease studies [4][5] - The country is at the forefront of technological innovations in cell and gene therapies, which not only accelerate local research but also provide innovative ideas for global applications [5][6] - China's healthcare system allows for efficient collection of disease registry data, crucial for understanding disease progression and setting treatment endpoints [5][6] AstraZeneca's Strategy in Rare Diseases - AstraZeneca has established partnerships with 14 Chinese biopharmaceutical companies to enhance its rare disease research, focusing on unmet clinical needs and differentiated project advantages [6][7] - The company emphasizes the importance of developing transformative therapies for rare diseases, which can create significant clinical value and support sustainable business models [7][8] Clinical Developments and Future Outlook - AstraZeneca has received approval for seven rare disease drugs globally and has ten ongoing Phase III clinical and regulatory review projects across various therapeutic areas [8][9] - The year 2025 is anticipated to be significant for AstraZeneca's rare disease research, with key Phase III study results expected to be released [9][10] - AstraZeneca is actively building a diagnostic and treatment ecosystem in China, having established 138 rare disease treatment centers to ensure standardized care [9][10] Patient Engagement and Accessibility - The company involves patients and caregivers early in the research process to ensure that clinical trials address the most critical disease improvement indicators [10] - AstraZeneca is advocating for the inclusion of rare disease drugs in national insurance programs to enhance accessibility for patients, while also calling for the establishment of special funds to alleviate patient financial burdens [10]