Current challenges and future perspectives of drug discovery in China

China’s pharmaceutical industry, which is historically centered around generic medicines, has largely transformed from imitation to innovation over the past three decades. Despite unprecedented progress, critical challenges remain such as insufficient indigenous research funding, underdeveloped academia-industry relationships, and significant barriers to market access. This perspective examines the evolving pharmaceutical landscape of China, focusing on its participation in global clinical trials and the resultant new drug approvals. Data for this analysis was sourced from several databases (e.g. PharmCube, NextPharma, and PharmaGO), academic reports, and published literature, covering data up to 2024 (unless otherwise specified). This perspective highlights ongoing regulatory challenges, such as inconsistencies in product standards, and the approval processes relative to the U.S.A. and the European Union. There is also an urgent demand for sustained international investment and recognition, partially due to the recent changes in the geopolitical environment. This perspective also discusses China’s efforts to implement accelerated approval pathways and foster multilateral development collaborations. China must align its regulatory policies more closely to the international norm to generate robust trial data that will be readily acceptable to the FDA and EMA. Continued investment in biologics as well as cell and gene therapy and artificial intelligence will drive innovation and enhance competitiveness. Additionally, strengthening the academia-industry collaboration is crucial to obtaining new leads through translational research. Ultimately, structural reforms are required to solidify the country’s goal of becoming a major player in the global pharmaceutical market.

中国医药产业历来以仿制药为核心，近三十年来已基本完成从仿制向创新的转型。尽管取得了前所未有的进展，但仍存在诸多关键挑战：本土研发投入不足、产学研联结有待深化、市场准入壁垒显著。本视角文章聚焦中国医药产业的发展格局，重点探讨其参与全球临床试验及由此带来的新药获批情况。本分析所用数据源自多个数据库（如PharmCube、NextPharma与PharmaGO）、学术报告及已发表文献，数据覆盖至2024年（另有说明者除外）。本文还指出了当前存在的监管挑战，例如相较于美国与欧盟，中国在产品标准一致性、审批流程等方面仍存在差距。受近期地缘政治环境变化影响，持续获取国际投资与国际认可的需求尤为迫切。本视角同时探讨了中国在推行加速审批路径、推动多边研发合作方面所做出的努力。中国需进一步推动监管政策与国际规范接轨，以生成能够被美国食品药品监督管理局（FDA）与欧洲药品管理局（EMA）直接认可的高质量临床试验数据。持续加大对生物制剂、细胞与基因治疗以及人工智能（artificial intelligence）领域的投入，将助力产业创新并提升全球竞争力。此外，加强产学研合作，通过转化研究获取新的研发线索至关重要。归根结底，唯有推进结构性改革，才能夯实中国跻身全球医药市场核心参与者行列的目标。