中新网济南2月21日电 题：全国政协委员彭军：从罕见病到就医体验 心系"急难愁盼" 作者 李明芮 祁建月 "怎样真正帮到罕见病患者？"这是全国政协委员、民盟中央委员、山东大学齐鲁医院副院长彭军履职过 程中，始终萦绕心头的问题。 患做检查。受访者供图 今年全国两会前夕接受中新网专访，他再次提到这一群体。"罕见病虽称'罕见'，关乎每一个患者家庭 的完整与希望。"作为血液科医生，彭军坚守临床30余年，接诊过很多罕见病患者。这个群体尤其是贫 困家庭患儿，是他履职路上放不下的牵挂。 此外，罕见病患者在生活中还面临诸多不便，在入学、就业等多环节仍会遭遇阻碍，成为需重点关注的 弱势群体。"这些孩子、这些家庭，值得被世界温柔以待。"彭军说。 在各界共同努力下，彭军的提案逐步落地，罕见病保障工作迎来积极变化。国家卫健委、国家医保局等 部门多次主动与他电话沟通，征求关于罕见病诊疗与保障工作的意见建议。 彭军为病 生交流探讨。李明芮 摄 "2024年，有15种罕见病用药被新纳入国家医保目录。这对罕见病家庭来说，是实实在在的好消息。"彭 军举例说，治疗阵发性睡眠性血红蛋白尿的特效药依库珠单抗，在纳入医保前，患者每年自付费用达 300 ...

Core Insights - AstraZeneca achieved a record revenue of $6.664 billion in China for 2025, marking a significant milestone in its 30-year history in the market [1] - The company plans to invest $15 billion in China by 2030 and has established a $18.5 billion collaboration with CSPC Pharmaceutical Group [1] Revenue Performance - AstraZeneca is the second-largest market for the company, with 2024 revenues reaching $54.073 billion, and China contributing $6.413 billion, a year-on-year increase of 11% [1] - In 2025, total revenue reached $58.739 billion, with China contributing $6.654 billion, accounting for 11% of total revenue, maintaining the top position among multinational pharmaceutical companies in China [1][3] Product Portfolio - Since entering the Chinese market in 1993, AstraZeneca has introduced over 40 innovative drugs, including key products in oncology and chronic disease management [3] - The inhaled budesonide suspension is the highest-selling drug in China, although its sales have declined due to policy price adjustments [3] - Osimertinib is expected to become a new flagship product in China, potentially replacing budesonide due to the large market for non-small cell lung cancer patients [3][4] Strategic Focus - AstraZeneca is focusing on a comprehensive "innovation offensive" in China, particularly in oncology, cardiovascular, renal, and metabolic disease areas [6] - The company has a robust pipeline in oncology, with several promising drugs set to launch in China, including novel AKT inhibitors and TROP2 ADCs for breast cancer treatment [6] - The pipeline also includes treatments for chronic diseases and rare diseases, addressing the diverse needs of the Chinese market [7][8] Investment Plans - AstraZeneca's $15 billion investment will focus on expanding drug manufacturing and R&D capabilities, particularly in cell therapy and radiolabeled drug conjugates [12][13] - The company has made 23 collaborations in China since 2021, totaling over $40 billion, with a significant focus on oncology and chronic disease treatments [9][12] Market Positioning - AstraZeneca aims to leverage China's scientific and manufacturing strengths to provide cutting-edge treatment solutions, positioning itself as a leader in cell therapy capabilities [13] - The company has established global strategic R&D centers in Beijing and Shanghai and operates multiple production bases across China [13][14] Conclusion - AstraZeneca's deep integration into the Chinese market signifies a shift from merely selling products to becoming a core participant in China's healthcare ecosystem, evolving into a global innovation center rooted in China [15]

Core Viewpoint - Heng Rui Medicine's subsidiary Chengdu Shengdi Pharmaceutical has received a notice of acceptance from the National Medical Products Administration for the marketing authorization application of HRS-5965 capsules, a treatment for Paroxysmal Nocturnal Hemoglobinuria (PNH) [1][2] Group 1: Company Developments - The application for HRS-5965's market approval is based on a pivotal study involving PNH patients who had not previously received complement inhibitor treatment [1] - The clinical study (HRS-5965-301) was a Phase III trial comparing HRS-5965 capsules with Eculizumab in treating PNH patients, led by prominent researchers from the Chinese Academy of Medical Sciences [1] - The study included 76 patients across 13 centers nationwide, showing that HRS-5965 significantly improved hemoglobin levels, reduced the need for blood transfusions, and enhanced quality of life compared to Eculizumab [1] Group 2: Industry Context - PNH is a rare acquired hemolytic disease characterized by the deficiency of CD55 and CD59, leading to complement-mediated intravascular hemolysis, and has been included in the national rare disease directory due to its low incidence/prevalence [2] - HRS-5965 capsules act as a complement factor B inhibitor, which can suppress complement-mediated hemolysis and improve hemoglobin levels [2] - Currently, the only other drug targeting this indication is Novartis's Iptacopan (Fabhalta), which is projected to generate global sales of approximately $129 million in 2024 [2] - The cumulative R&D investment for HRS-5965 capsules has reached approximately 218 million yuan [2]

Core Viewpoint - Heng Rui Medicine's subsidiary Chengdu Shengdi Pharmaceutical has received a notice of acceptance from the National Medical Products Administration for the market approval application of HRS-5965 capsules, a treatment for Paroxysmal Nocturnal Hemoglobinuria (PNH) [1][2] Group 1: Product Development - The application for HRS-5965 is based on a pivotal study involving PNH patients who had not previously received complement inhibitor treatment [1] - The study, HRS-5965-301, is a Phase III clinical trial comparing HRS-5965 capsules with Eculizumab in treating PNH patients, involving 76 patients across 13 centers in China [1] - Results indicate that HRS-5965 significantly improves hemoglobin levels, reduces the need for blood transfusions, and alleviates fatigue compared to Eculizumab, enhancing patients' quality of life [1] Group 2: Market Context - PNH is a rare acquired hemolytic disease characterized by the deficiency of CD55 and CD59, leading to complement-mediated intravascular hemolysis [2] - HRS-5965 is a complement factor B inhibitor that can suppress complement-mediated hemolytic reactions and improve hemoglobin levels [2] - Currently, the only approved drug targeting this indication is Novartis's Iptacopan (Fabhalta), with projected global sales of approximately $129 million in 2024 [2] - The cumulative R&D investment for HRS-5965 has reached approximately 218 million yuan [2]

Core Insights - A 7-year-old boy, referred to as Youyou, was admitted to a children's hospital due to severe symptoms including swelling and dark urine, indicating acute kidney failure [1][2] - The medical team diagnosed him with secondary IgA nephropathy and atypical hemolytic uremic syndrome (aHUS), a rare condition that can rapidly progress to kidney failure [2] Group 1: Medical Condition and Diagnosis - Initial symptoms included eyelid and leg swelling, dark urine, reduced urine output, and fatigue, leading to a diagnosis of acute kidney failure [1] - After stabilization, a kidney biopsy confirmed the presence of aHUS and IgA nephropathy, necessitating targeted treatment [2] Group 2: Treatment and Recovery - The treatment involved the use of the targeted drug Eculizumab to inhibit abnormal complement activation, along with corticosteroids and cyclophosphamide to control inflammation and protect kidney function [2] - Following 52 days of intensive care and treatment, the boy's condition improved, leading to his eventual recovery and discharge from the hospital [2] Group 3: Medical Advice - Medical professionals emphasize the importance of early diagnosis and treatment for children showing symptoms such as unexplained swelling, dark urine, and fatigue to prevent acute kidney failure and identify rare diseases [2]

Core Viewpoint - The National Healthcare Security Administration (NHSA) announced the 2025 version of the National Basic Medical Insurance Drug List and the first Commercial Health Insurance Innovative Drug List, which includes significant updates to enhance drug accessibility for patients [1][2][3]. Group 1: Drug List Adjustments - The new drug list adds 114 new drugs, including 50 innovative drugs, while removing 29 drugs that are either unavailable or can be replaced by others [1][2][3]. - The total number of drugs in the list now stands at 3,253, comprising 1,857 Western medicines and 1,396 traditional Chinese medicines, with improved coverage for critical areas such as cancer, chronic diseases, mental health, rare diseases, and pediatric medications [1][2][3]. Group 2: Financial Implications - The NHSA has assessed the budget impact of new drugs on the medical insurance fund, concluding that the overall financial burden remains manageable within the national budget [3][4][12]. - The introduction of new drugs may lead to increased costs, but many are expected to replace older medications, mitigating the financial impact [3][4][12]. Group 3: Challenges in Hospital Implementation - Despite being included in the insurance list, many "nationally negotiated drugs" face challenges in being adopted by hospitals due to limited capacity in hospital drug lists, which typically accommodate only 1,500 to 2,000 drugs [4][6][8]. - The approval process for new drugs in hospitals can be lengthy, often taking from one to twelve months, which complicates timely access for patients [8][12][44]. Group 4: Dual-Channel Policy - To address the issue of drug availability, the government introduced a "dual-channel" policy, allowing patients to access "nationally negotiated drugs" through designated hospitals and retail pharmacies, ensuring equal reimbursement [14][16][50]. - As of now, 402 nationally negotiated drugs have been integrated into this dual-channel system, significantly increasing sales from under 4 billion yuan in 2020 to nearly 15 billion yuan in 2024 [16][50]. Group 5: Commercial Health Insurance Innovations - The newly established Commercial Health Insurance Innovative Drug List includes 19 drugs, selected from 121 candidates, aimed at covering high-value drugs not included in the basic insurance [27][30][67]. - This list is designed to complement the basic medical insurance system, addressing the gap for high-cost innovative treatments that are crucial for patients with severe conditions [30][32][67]. Group 6: Future Implications - The introduction of the Commercial Health Insurance Innovative Drug List is expected to enhance patient access to high-value treatments, encouraging insurance companies to cover these drugs and potentially increasing patient enrollment in commercial health plans [30][66][73]. - The dual-directory approach aims to create a more robust healthcare financing system, ensuring that patients can access necessary medications while maintaining the sustainability of the insurance funds [30][66][73].

Core Viewpoint - The article discusses the treatment challenges and advancements in therapies for generalized myasthenia gravis (gMG), emphasizing the need for targeted therapies to address the limitations of traditional treatments [1][2][8]. Treatment Challenges - Traditional non-specific immunotherapy, including corticosteroids and non-steroidal immunosuppressants, has significant limitations, including adverse effects from long-term corticosteroid use and slow onset of action for other immunosuppressants [2][3]. - Approximately 10% to 20% of patients show limited response to conventional treatments, leading to a subset of patients developing "refractory gMG" [2][3]. - The heterogeneity of MG results in varied patient responses to medications, complicating the selection of effective treatment plans [2][3]. Advancements in Targeted Therapies - Targeted therapies for gMG are categorized into several classes, including FcRn antagonists, B-cell targeted therapies, and complement inhibitors [3][4]. - FcRn antagonists, such as efgartigimod, accelerate the degradation of pathogenic antibodies, reducing their levels [4]. - B-cell targeted therapies include monoclonal antibodies like rituximab and inebilizumab, which have shown efficacy in improving clinical symptoms and reducing corticosteroid dosage [4][6]. - Complement inhibitors, such as eculizumab and ravulizumab, block the complement pathway, preventing immune damage to neuromuscular junctions [4][6]. Clinical Evidence and Guidelines - Clinical studies indicate that treatments like eculizumab significantly improve MG-ADL and QMG scores within weeks, with a notable percentage of patients able to reduce corticosteroid use [5][6]. - The 2025 guidelines emphasize achieving "minimal symptom expression" as a treatment goal, highlighting the importance of long-term maintenance therapy [3][8]. - The CHAMPION MG OLE study demonstrated a 71.3% reduction in clinical deterioration events after 60 weeks of treatment with eculizumab [5][6]. Future Directions - The article advocates for a shift from traditional stepwise treatment to early targeted interventions and personalized treatment strategies for gMG patients [7][8]. - Emphasis is placed on the need for early intervention in high-activity gMG patients to prevent irreversible damage and improve quality of life [7][8]. - A comprehensive management model that includes rapid control of symptoms, long-term maintenance, and safe tapering of corticosteroids is essential for effective gMG treatment [7][8].

Core Insights - The National Healthcare Security Administration (NHSA) announced the 2025 version of the National Basic Medical Insurance Drug List, which includes 114 new drugs, 50 of which are innovative drugs, while 29 drugs were removed due to lack of supply or better alternatives [1][2][4] Group 1: Drug List Adjustments - The total number of drugs in the directory has increased to 3,253, with 1,857 Western medicines and 1,396 traditional Chinese medicines, significantly enhancing coverage for critical areas such as cancer, chronic diseases, mental health, rare diseases, and pediatric medications [1][4] - The new directory will be implemented nationwide starting January 1, 2026 [1] Group 2: Innovative Drug Access Challenges - Despite the inclusion of innovative drugs in the directory, there are challenges in getting these drugs into hospitals due to limited capacity in hospital drug lists, which typically accommodate only 1,500 to 2,000 drugs [6][9] - Hospitals face difficulties in deciding whether to replace existing drugs with newly added "nationally negotiated drugs," leading to a cautious approach in procurement [7][11] Group 3: Financial Implications for Healthcare Providers - The NHSA has assessed the budget impact of new drugs on the healthcare fund, indicating that the overall financial burden will remain manageable within the national healthcare budget [5] - Hospitals are under pressure to control costs, especially as high-priced innovative drugs could lead to reduced budget surpluses or even deficits [13][15] Group 4: Dual-Channel Policy for Drug Distribution - The "dual-channel" policy allows patients to access "nationally negotiated drugs" through designated hospitals and retail pharmacies, ensuring equal reimbursement rates [16][18] - This policy has successfully increased the availability of critical drugs, with sales of "nationally negotiated drugs" rising from under 4 billion yuan in 2020 to nearly 15 billion yuan in 2024 [21] Group 5: Commercial Health Insurance Innovations - The NHSA introduced the first Commercial Health Insurance Innovative Drug List, which includes 19 drugs, to complement the basic medical insurance system and address the coverage gap for high-value innovative drugs [31][34] - This new list aims to facilitate better access to expensive treatments that are not covered by basic insurance, thereby reducing the financial burden on patients [40][42]

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 [1][2] - China is evolving from a participant to a "breakthrough engine" in the global rare disease research landscape, leveraging its unique advantages in clinical trial efficiency, technological innovation, and disease data accumulation [1][3] Diagnosis Challenges - The number of known rare diseases has increased from 7,000 to over 10,000 due to advancements in gene sequencing, yet approximately 90% still lack treatment [2] - 80% of rare diseases are genetic and often manifest in childhood, making pediatric rare disease drug development a pressing and complex task [2] - Variations in the definition of rare diseases across countries complicate research efforts, with China lacking a clear regulatory list of rare diseases [2] 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 [3] - The country is at the forefront of technological innovations in cell and gene therapies, which can accelerate local research and provide global insights [3] - China's healthcare system allows for efficient disease data collection, crucial for understanding disease progression and treatment endpoints [3] Collaborations and Partnerships - AstraZeneca has established collaborations with top Chinese hospitals to create disease excellence centers focusing on rare kidney and neurological diseases [4] - The company has engaged in acquisitions and partnerships with 14 Chinese biotech firms to enhance its rare disease research capabilities [5] Strategic Focus on Rare Diseases - AstraZeneca's commitment to rare disease research is driven by the potential for transformative therapies that meet significant unmet needs, despite the high costs and small patient populations [5][6] - The company has developed drugs that can significantly reduce mortality rates in conditions like hemolytic uremic syndrome, emphasizing the clinical value of these treatments [6] Pipeline and Future Outlook - AstraZeneca has seven approved rare disease drugs and ten ongoing Phase III clinical trials across various therapeutic areas [6] - The year 2025 is anticipated to be significant for the release of numerous rare disease research outcomes [7] Patient-Centric Approach - AstraZeneca involves patients and caregivers early in the research process to ensure that clinical trials address the most critical disease improvement metrics [8] - The company collaborates with global patient associations to enhance patient engagement and communication with regulatory bodies [8] Accessibility and Affordability - AstraZeneca has three innovative rare disease drugs approved in China, with some included in the national medical insurance catalog, improving patient access [9] - The company advocates for the establishment of rare disease-specific funding to alleviate patient financial burdens, recognizing that insurance alone cannot cover all costs [9] - Optimism is expressed regarding the commercial prospects in China as policies improve and more rare disease drugs are included in insurance coverage [9]

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]