AI制药成热点 能改变创新药"三个十定律"吗

AI辅助研发药物正在成为医药产业的新热点。国内首款获批的AI辅助研发原创药艾普司韦，从发现到临床试验仅用时三年半，相比传统创新药十年的研发周期实现了大幅压缩1。这一进展引发了业界对AI是否能够突破创新药"三个十定律"——十年时间、十亿美元投入、不到百分之十成功率——的思考1。

AI在药物研发前端的作用正在被广泛关注。通过高通量筛选、分子优化、安全性预测等环节，AI显著提升了研发效率1。中国科学院院士陈凯先表示，"AI将从既有流程效率提升变为整个研究流程的系统重塑"1。然而，专家也指出了隐含的风险。清华大学药学院创始院长丁胜观察到，"生成的分子越多，失败的越多"，强调需要关注疾病机制认识和成药率提升1。

产业政策支持正在加速推进。工信部等十部门于9月18日印发《医药工业发展"十五五"规划》，提出到2030年创新药产业规模年均增速超过20%，首创新药全球占比要突破25%1。近期获批的新药数据也显示了产业的活跃态势，2025年新靶点、新机制药品获批11个，其中4个为国产创新药；2026年上半年获批的11个新药则全部为国产创新药1。

AI has emerged as a focal point in pharmaceutical research and development, with Apisweir—China's first original drug approved with AI-assisted development—completing its journey from discovery to clinical trials in just three and a half years, a dramatic compression compared to the traditional ten-year timeline 1. The drug received regulatory approval in September 2025 1. This acceleration challenges the pharmaceutical industry's longstanding "Rule of Threes": ten years of development time, one billion dollars in investment, and less than ten percent success rate 1.

AI enhances research efficiency across multiple stages, including high-throughput screening, molecular optimization, and safety prediction 1. However, experts caution against unbridled optimism. According to Ding Sheng, founding dean of Tsinghua University's School of Pharmaceutical Sciences, a concerning pattern has emerged: "the more molecules generated, the more failures accumulate" 1. This observation suggests that while AI accelerates certain workflow components, downstream attrition rates warrant careful attention and enhanced focus on disease mechanism understanding and improvement of drug candidacy success rates 1. Chen Kaishen, an academician of the Chinese Academy of Sciences, emphasized that AI's transformative potential lies not merely in incrementally improving existing processes, but in fundamentally restructuring the entire research workflow 1.

China's commitment to innovation drug development is reflected in ambitious targets. In 2025, regulatory authorities approved eleven drugs with new targets or novel mechanisms, including four domestic innovative treatments 1. The first half of 2026 saw approval of an additional eleven such drugs, all of which were domestic innovations 1. The government has set a goal for 2030: achieving annual growth exceeding twenty percent in the innovative drug industry scale, with homegrown first-in-class drugs accounting for more than twenty-five percent of global approvals 1. On September 18, the Ministry of Industry and Information Technology and nine other government departments issued the "Fifteenth Five-Year Plan for Pharmaceutical Industry Development" to guide sector advancement 1.