A new round of technological revolution and industrial transformation in global biomedicine is accelerating, with innovative drugs becoming the most crucial strategic development direction in the biopharmaceutical industry. Since 2016, China’s innovative drug R&D has entered the fast track and gradually joined the international advanced ranks. Facing the vast overseas market for innovative drugs, an increasing number of domestic innovative pharmaceutical companies are joining the globalization trend, making product internationalization a “must-choose” option for enterprise development. During the 2024 China BioMed Innovation and Investment Conference (CBIIC), on the morning of December 1^st, New Drugs R&D and Regulation Internationalization Forum, organized by the International Regulatory Science Specialty Committee of China Pharmaceutical Innovation and Research Development Association (PhIRDA), was successfully held. The forum focused on the globalization of innovative drugs, with experts discussing hot topics such as new drug R&D transformation, simultaneous submissions to China and US’s regulatory authorities to be approved, and the NewCo model. The forum was moderated by Dr. He Ruyi, Chairman of the International Regulatory Science Specialty Committee of PhIRDA, Chief Strategy Officer of RemeGen, Chief Scientist of SDIC, and former Chief Scientist of the Center for Drug Evaluation, CFDA.

Dr. He Ruyi

Dr. Geoff Tsen

Dr. Geoff Tsen,Global Vice President and General Manager of Greater China at the United States Pharmacopeia (USP),delivered a keynote speech on “USP Quality Standards to Enable Innovative Drug R&D.”USP is an independent, science-based nonprofit organization responsible for setting standards for medicines, food ingredients, and dietary supplements produced, distributed, and used globally. Since its establishment in 1820, USP has become a crucial reference for global pharmaceutical quality standards, with its standards adopted by over 140 countries. USP’s general chapters and monographs cover all aspects of drug development and manufacturing, providing support for analytical methods, excipient standards, and technical guidelines applicable to innovative drugs, generic drugs, and biologics development. As a “quality guardian” for new drug development, USP continues to promote pharmaceutical innovation through high-quality standards, helping Chinese innovative drugs enter the international stage and contributing to global medication safety and human health welfare.

Professor Yang Jin

Professor Yang Jin from China Pharmaceutical University focused on “Fostering a Biopharmaceutical Innovation Ecosystem and Advancing the High-level Translation of Basic Research” in his keynote speech about China’s innovative drug R&D and translation. Professor Yang first reviewed China’s pharmaceutical reform process, noting that while China has made significant progress in basic research, there remains a “valley of death” in new therapy translation, mainly due to a lack of translatable targets that could directly attract investment. The success of innovative drugs depends not only on target and mechanism research but also requires discovering key early disease stages through population stratification and disease progression analysis. He emphasized the importance of technology incubation platforms and proposed the “lean proof of concept” method to determine the technical success probability of drug candidates in the shortest time and at the lowest cost, avoiding excessive investment in ineffective drugs. Professor Yang believes the key to future success in innovative drug R&D lies in “two-end force”: strengthening the depth and breadth of basic research to discover precise targets and mechanisms, while using biomarker development as a lever to improve research translation efficiency, supporting the high-quality development of China’s pharmaceutical innovation ecosystem.

Dr. Zhao Xiaobin

Dr. Zhao Xiaobin, President and Founder of Zhejiang Haichang Biotech, shared insights on “Translational Research and Regulatory Considerations of Small Nucleic Acid Drugs,” discussing key challenges and development opportunities in clinical translation and regulatory practice. Small nucleic acid drugs are characterized by protein-independent druggability, short R&D cycles, and multiple drug targets. However, clinical translation faces challenges including potential off-target effects causing toxicity, pharmacokinetic/pharmacodynamic (PK/PD) considerations, and complex clinical trial design. Dr. Zhao noted that China has not yet issued specific regulations and guidelines for small nucleic acid drugs, suggesting that companies should proactively maintain close communication with regulatory authorities to jointly promote the establishment of clinical trial and standardization systems. With rapid development in R&D technology, the small nucleic acid drug sector is poised for broader opportunities, becoming a crucial driver for biopharmaceutical innovation.

Dr. Du Xin

Dr. Du Xin, CEO of Evergreen Therapeutics, delivered a keynote speech on “Quality Considerations in Overseas Expansion and Investment Decisions for Innovative Drugs,” analyzing the core role of drug quality in international deployment and investment decisions. Quality serves not only as the fundamental guarantee for clinical efficacy and safety but also impacts investment, market prospects, regulatory approval, market access, and corporate brand reputation. In innovative drug investment decision-making, companies must comprehensively focus on product quality throughout the entire chain from R&D to production, incorporating brand quality management as a strategic priority. In the process of going global, 62% of global drug shortages are caused by quality issues, highlighting the necessity of establishing and maintaining quality management systems. Dr. Du urged companies to establish a “quality-first” concept in their international layout, continuously optimizing quality management systems to improve product quality and production efficiency, thereby leading global pharmaceutical innovation.

Dr. Howard Yuwen

Dr. Howard Yuwen,Senior Vice President and Chief  Business Officer of SDM Bioservices, shared practical experiences in the internationalization of Chinese innovative drugs through his presentation on “Regulatory Considerations of Global Submissions for Innovative Drugs – Case Studies,” providing valuable reference for pharmaceutical companies’ global strategic planning. Dr. Yu pointed out that China’s pharmaceutical R&D capabilities have continuously improved in recent years, particularly in the field of new drugs. For Chinese pharmaceutical companies, international expansion is not only a necessary step for entering global markets but also a key pathway to enhance the global competitiveness of Chinese pharmaceuticals. Companies should systematically plan their global strategy from early R&D stages, especially during clinical trials, developing scientific and differentiated strategies to meet various national regulatory requirements, thereby facilitating faster and more stable entry of Chinese innovative drugs into international markets.

Dr. He Ruyi

Dr. He Ruyi, delivered a report on “Reply of Innovation: Reflections on the NewCo Model of Licensing-out.” Dr. He reviewed the internationalization journey of Chinese pharmaceutical companies, from primarily License-in to gradually shifting towards License-out in recent years. When considering internationalization now, companies should not only focus on the US market but also seek new development paths through markets such as Hong Kong, Macao, and ASEAN. Facing the capital winter and IPO challenges, the NewCo model provides new financing and exit channels, offering new opportunities and choices for Chinese innovative pharmaceutical companies’ international development. The NewCo trend essentially reflects the gradual  international acceptance of Chinese innovative drugs, representing a two-way pursuit between innovative drugs and overseas markets. As innovative pharmaceutical companies mature and develop capabilities to compete in international markets, it also indicates that the future biopharmaceutical industry will become more specialized and align with US pharmaceutical industry development trends. Top-tier large pharmaceutical companies will become fewer, maintaining their positions through numerous mergers and acquisitions with their long histories and abundant resources. Mid-sized pharmaceutical companies will survive in niche markets with only a few leading products, while numerous biotech companies will need to focus on early-stage R&D and clinical development, with slim chances of evolving into large pharmaceutical companies. In the end, Dr. He proposed a new concept of  “Modified NewCo,” suggesting joint development and investment in Chinese innovative drugs through collaboration between Chinese funds and USD funds, providing new internationalization opportunities for Chinese pharmaceutical companies.

The panel discussion was moderated by Dr. Zhao Xiaobin, with participation from Dr.Geoff Tsen,Professor Yang Jin,Dr. Du Xin, Dr. Howard Yuwen,and William Sun,Head of Healthcare Industry Head at Citi China Commercial Banking. The panelists shared key strategies and practical experiences from different areas in the internationalization process of Chinese innovative drugs, focusing on “How to Promote the Overseas Expansion of the Chinese Biotechs from the Perspective of Regulation,” providing multi-dimensional, full-chain references for pharmaceutical companies’ global development.

Panel Discussion

The experts agreed that Chinese pharmaceutical companies need to focus on five key areas in promoting innovative drugs globally: In terms of quality standards, they should align with international standards (such as USP) to improve drug quality and global recognition while effectively reducing R&D costs. Regarding precision R&D, they should strengthen biomarker research, build innovation ecosystems, and promote efficient translation and internationalization of new drug R&D. For submission strategies, they should adopt China-US dual submission approaches to expand global market access through international clinical trials and filing systems. In global deployment, they should plan internationalization strategies early, identify potential issues, and improve overseas submission efficiency and success rates. For capital support, they should integrate global resources to enhance international competitiveness. 

The globalization of innovative drugs requires multi-party collaboration, strengthening regulatory communication, enhancing R&D innovation, and advancing the internationalization of Chinese innovative drugs through capital and global cooperation, opening a new chapter in going global and pushing the innovative drug industry to a higher level!

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