CDE Releases Pioneer Plan to Promote Organoid-Based Alternatives in Drug R&D

The Center for Drug Evaluation (CDE) of the National Medical Products Administration has released a draft of the 'Pioneer Plan', launching a 'pilot program to promote research and application of new methods' to advance the study and use of new alternative methods (NAMs) such as organoids and organ-on-chip in new drug R&D. The CDE noted that traditional animal testing involves long cycles, high costs, and low throughput, and that due to interspecies differences, the accuracy of extrapolating animal test results to humans is limited. Global regulators are advancing the '3R' principle (reduction, refinement, or replacement of animal testing).

Traditional CROs are quietly transforming. In its 2025 interim report, Joinn Laboratories proposed expanding its business from 'human pluripotent stem cell production' to multiple 'organoid platforms' and is committed to developing multi-organoid systems and chips. Medicilon has established a NAMs service platform integrating 'AI-driven + organoid + wet-dry fusion', connecting capabilities in organoids, organ-on-chip, AI prediction, PBPK simulation, and in vitro toxicology. A Medicilon representative said the company has received and undertaken related service inquiries and projects from research institutes and pharmaceutical companies, with demand rising significantly over the past year or two, covering evaluation of novel drug modalities, improvement of early screening efficiency, and preparation of data for international regulatory filings. AI drug developer Insilico Medicine is building an automated screening and evaluation platform centered on liver organoids for early assessment of liver toxicity.

Xie Xin, founder of Shanghai organ-on-chip company Yaosu Technology, disclosed that the company expects revenue in the tens of millions of yuan in 2026 and has over 100 million yuan in orders on hand. Collaborations with pharmaceutical companies such as Sanofi, Pfizer, and Novo Nordisk have upgraded from single projects to multi-year strategic framework agreements. Yaosu Technology completed nearly 400 million yuan in Series A financing within six months. Organoid models offer predictive advantages in liver toxicity assessment, local toxicity of topical drugs, and disease areas lacking suitable animal models (e. g. , atherosclerosis). However, organoids have limitations such as the absence of vascularized structures, complete innervation, and immune microenvironment, making them unable to simulate systemic human responses.

Currently, regulatory agencies in different countries are at varying stages in determining whether and how NAMs can be considered equivalent to traditional animal testing. Establishing a regulatory acceptance framework for NAMs data—clarifying under what circumstances NAMs data can be accepted as independent evidence or supplementary evidence—is a core challenge for the industry. Researchers from the CDE recently wrote in a publication that 'exploration of strategies to reduce or replace animal testing in nonclinical studies of new drugs' has been listed as a funded project under the Key Laboratory of Drug Regulatory Science of the National Medical Products Administration, with plans to produce a series of guidance documents or consensus within two years.