Current landscape of innovative drug development and regulatory support in China

Abstract

The global pharmaceutical landscape remains dynamic and competitive, shaped by advancements in first-in-class therapies and breakthrough technologies. The United States has maintained its leadership in first-in-class therapies and breakthrough technologies, driven by advanced regulatory pathways, significant multinational corporation investments, a robust Research and Development (R&D) workforce, and continuous technological innovation. Additionally, global impact of the Food and Drug Administration (FDA) is further amplified through collaborations like Project Orbis, which facilitates simultaneous reviews of cancer treatments by multiple regulatory authorities worldwide. Europe, while historically strong, faces growing challenges in maintaining its competitive edge, particularly due to protracted regulatory timelines and complex coordination among its member states. In this competitive global environment, China has rapidly transformed from a generics-dominated market to a key player in innovative drug development. This article reviews China's progress in innovative drug R&D from 2019 to 2023, emphasizing regulatory modernization, clinical trial advancements, and the emergence of novel therapies. By comparing China's developments with above global counterparts, this review highlights the country's achievements in regulatory efficiency, clinical trial progress, and the development of innovative therapies such as biologics and cell and gene therapies. Through this comparative analysis, the article underscores how China's evolving policy-driven innovation ecosystem has positioned it as a growing leader in global drug development. The review examines how enhanced regulatory efficiency, clinical trial progress, manufacturing capabilities, and international collaboration have bolstered China's growing influence, while also discussing the future opportunities and challenges it faces in shaping global pharmaceutical innovation and development.

Fig. 1

Overview of IND applications and approvals for innovative drugs in China from 2019 to 2023. a Number of innovative drug IND applications and approvals from 2019 to 2023, along with their respective growth rates. b Number and proportion of innovative drug varieties within the total drug varieties in IND applications of 2023. The groups are classified as: all drugs, chemical drugs, preventive biologics (vaccine), therapeutic biologics (TB), and TCM. c Number and proportion of innovative drug varieties within the total drug varieties in IND approvals of 2023. d Number of chemical drug, vaccine, TB and TCM among innovative drug IND applications from 2019 to 2023. e Number of chemical drug, vaccine, TB, and TCM among approved innovative drug INDs from 2019 to 2023. Data for this figure were derived from 2019 - 2023 Annual Drug Evaluation Reports published by CDE, NMPA.^–

Fig. 2

Overview of NDA applications and approvals for innovative drugs in China from 2019 to 2023. a Number of innovative drug NDA applications and approvals from 2019 to 2023, along with their respective growth rates. b Number of NDA applications for chemical drugs, biological products (BP), and TCM among innovative drug NDA applications from 2019 to 2023, along with their respective growth rates. c Number of drug varieties of chemical drugs, BP, and TCM among innovative drug NDAs approved from 2019 to 2023, along with their respective growth rates. The total number of approved drug varieties each year is indicated by yellow-marked symbols and numbers. d Number and growth rate of out-licensing deals of Chinese innovative drugs. Data for this figure were derived from 2020 - 2023 Annual Report on the Progress of Clinical Trials for New Drug Registration in China published by CDE, NMPA.^–

Fig. 3

R&D investment, new technologies, and new targets in China’s innovative drugs. a Internal R&D expenditure in China’s pharmaceutical manufacturing industry from 2019 to 2021, along with the year-on-year growth rates. b Comparison of the number and growth rates of First-in-Class (FIC), Fast-Follower (FF), Me-Too and all drugs under development in China in 2021 and 2024. c Number and proportion of various drug types under development in China in 2021 (inner ring) and 2024 (outer ring). d Number of drug pipelines in China and globally from 2019 to 2023, along with annual growth rates.^,

Fig. 4

Clinical trials for new drug registration in China. a Number and growth rate of new drug clinical trials (NDCT) and bioequivalence (BE) trials in China from 2019 to 2023. b Proportion of Category 1 innovative drugs in NDCT in 2023 (left pie chart) and the proportion of chemical drugs, biological products, and TCM among Category 1 innovative drugs (right pie chart). c Time to initiate subject recruitment in NDCT from 2021 to 2023. d Time required for market approval for innovative drugs. The time to market is analyzed based on the date of the first clinical trial approval to the date of the market approval. The inner ring and outer ring of the combined ring chart represent the time to market of innovative drugs that approved for market release in 2022 and 2023, respectively. Data for this figure were derived from 2020 - 2023 Annual Report on the Progress of Clinical Trials for New Drug Registration in China published by CDE, NMPA.^–

Fig. 5

Overview of accelerated review and approval process for innovative drugs in China from 2020 to 2023. a Number of cases agreed or disagreed for inclusion under the Breakthrough Therapy program from 2020 to 2023. b Distribution of indication areas for drugs agreed for inclusion under Breakthrough Therapy program in the year of 2023. c Number of drugs conditionally approved and transitioned to regular approval from 2020 to 2023. d Distribution of indications for conditionally approved drugs from 2020 to 2023. e Number of drug registration applications under the Prioritized Evaluation and Approval procedure from 2020 to 2023. f Distribution of indications for drug registration applications under the Prioritized Evaluation and Approval procedure from 2020 to 2023.^–

Fig. 6

a Clinical trials of Chinese innovative drugs conducted in the U.S. from 2007 to 2023. b Number and growth rate of out-licensing deals of Chinese innovative drugs.