21世纪是生物科学的世纪，是生物技术、生物信息学和纳米技术的时代。生物技术、生物信息学和纳米技术为生物医药的发展提供了前所未有的机遇，带来迎接创新挑战的科学基础和力量，理应对传统创新药物研发模式带来革命性变化。回顾半个多世纪的传统创新药物研发模式，不难发现世界高成本、高风险、低效率的研发传统模式，存在不利于创新发展的问题。根据近20年的进展和研发水平，估计未来10年小分子化学药物的创新仍然主导世界新药发展，小公司的前景和多渠道整合研发是世界新药不可抗拒的力量。面对挑战，采用先进监管科学技术和新方法，加强生物技术新药的研究开发和药物有效性和安全性风险评估研究十分迫切。其次，创新药物的高成本、高风险、低效率瓶颈需要提升合作效率来突破。第三，根据我国国情，注意发展分子靶向药物的研发战略和策略，重视给药系统的研究、明确创仿结合发展新药的目标，更需要合理整合研发发展模式，需要优化资源配置来提高研发效益。第四，中国特色的自主创新需要国家政策引导，政府在规划、政策、开放和实力建设的四大举措的实现，需要企业实力支持和官–产–学–研–企的多元协同创新体制的变革。

The 21st century is the century of biological sciences, biotechnology, bioinformatics, and nanotechnology era. Biotechnology, bioinformatics, and nanotechnology provided unprecedented opportunities and challenges, brought innovation to meet the scientific basis and strength for the development of biomedicines, and should be innovative to traditional drug development model which brought revolutionary change. Recalling the research and development (R&D) mode of tradition innovative drug could find that the traditional R&D mode with high-cost, high-risk, and low efficiency did not have the conducive to innovation and development in the past half-century. According to the progress and development of the new drugs in the past 20 years, the innovative small molecular drugs would still dominate the worldwide drug development, and the development prospects of small R&D companies and multi-channel integration were the irresistible power for the worldwide new drugs. To face the challenges, the use of advancing science, technology, and supervision methods is very urgent to strengthen the biotechnology in drug efficacy and safety risk assessment study for R&D of new drugs. The bottleneck of “high-cost, high-risk, low-efficiency” needs to be broken by increasing the efficiency of cooperation in innovative drugs. According to the national conditions in China, the development strategies and tactics on molecular targeting drugs and the drug delivery system should be focused, and the R&D combination of innovative drugs and generic drugs to develop new drugs needs to reasonably integrate the development mode and to optimize the allocation of R&D resources to improve the R&D efficiency. The independent innovation with Chinese characteristics needs the guidance by national policy, and in the planning, policy, openness, and efficiency construction by policy, the government-industry-academy-research diverse collaborative innovation system should be needed.