中国工程院院士曹雪涛：新一轮“创新药国家重大专项”有四大转变

Core Insights - The new round of the "National Major Special Project for Innovative Drugs" aims to address significant unmet clinical needs in China, focusing on major chronic diseases, infectious diseases, pediatric medications, rare diseases, and special scenario medications [1][3] Group 1: Current Challenges in China's Innovative Drug Development - There are six major challenges facing China's innovative drug development, including urgent medication needs for chronic and infectious diseases, imbalances in disease spectrum matching, weak original innovation capabilities, concentrated research efforts, insufficient international clinical research layout, and the need for improved collaboration among national strategic scientific forces [1] - The "two major and three special" categories refer to major chronic diseases (such as cardiovascular diseases, tumors, metabolic diseases, and neurodegenerative diseases) and specific medication needs for children and rare diseases [1][2] - There are currently 85 rare diseases globally without available treatments, and 62 drugs that exist abroad but are lacking in China, indicating a need for targeted research and development [2] Group 2: Key Transformations in the New Project - The new project will shift from a product-oriented approach to a capability-oriented approach, focusing on the integrated development of varieties, talent, and platforms to enhance sustainable research and development capabilities [3] - There will be a transition from emphasizing the mid-to-lower reaches of the industrial chain to focusing on the upstream innovation chain, prioritizing new targets, mechanisms, and theories to develop original research drugs [3] - The project will no longer support generic drug development and will instead concentrate on original innovative drug research, aligning with unmet clinical needs [3] Group 3: Historical Context and Future Aspirations - The journey of innovative drug development in China has been gradual, driven by continuous national strategic support, evolving from a focus on generic drugs to leading original drug creation [4] - The goal is to overcome significant core technological challenges and produce high-level innovative drugs, ultimately cultivating a group of internationally influential pharmaceutical companies and establishing China as a global center for new drug creation and a hub for the biopharmaceutical industry [4]