Key Takeaways on China's Regulations on the Clinical Study and Clinical Translation and Application of New Biomedical Technologies – A New Era for IITs and Commercialization in Cell and Gene Therapy

Authors: Aaron GU丨 Cathy ZHENG丨Matt ZHANG丨Franky YU丨Naifang ZHANG丨Shuwen SUN丨Krystal ZHAO丨Lingyu SHENG^[1]

In recent years, cutting-edge biomedical technologies, such as gene editing, CAR-T/NK therapies, stem cell therapies, and assisted reproductive technologies have developed rapidly, demonstrating broad potential and attracting numerous enterprises and other market players to actively engage in such field.  Consequently, following the issuance of the Administrative Measures for Stem Cell Clinical Study (Trial) in 2015 and the Administrative Regulations on the Clinical Application of New Biomedical Technologies (Draft for Comment) in 2019 (hereinafter referred to as the "Draft"), a dual-track regulatory framework governing this field, covering both "drugs" and "technologies", has gradually taken shape.  Over the past six years, however, while drug regulations have been continuously refined, technology regulations have lagged behind.  There is increasing anticipation that legislation on new biomedical technologies will provide clearer regulatory pathways for innovation and development, and further standardize clinical study and clinical translation and application of such technologies to prevent a regulatory vacuum in related translation and application activities.  Eventually, on October 10, 2025, China's State Council officially released the Regulations on the Administration of Clinical Study and Clinical Translation and Application of New Biomedical Technologies (hereinafter referred to as the "Regulations"), which will come into effect on May 1, 2026.

This article will focus on the scope of application of the Regulations and its coverage of clinical study and clinical translation and application, and will analyze its impact on relevant stakeholders for the industry's reference and discussion.

Scope of application

The Regulations define "New Biomedical Technologies" as medical techniques and measures that are intended to assess health status, prevent or treat diseases, or promote health, which apply biological principles, act at the cellular or molecular level of the human body, and have not yet been clinically applied within China.

First, the three core characteristics and determining factors are "applying biological principles," "acting at the cellular or molecular level of the human body," and "not yet clinically applied within China."  Compared to the Draft, the newly added condition of "not yet clinically applied within China" in the Regulations reflects its innovation-oriented approach.

Second, it is worth emphasizing that the Regulations stipulate that clinical trials for the development of drugs and medical devices shall continue to be governed by laws and administrative regulations such as the Drug Administration Law and the Regulations on the Supervision and Administration of Medical Devices.  The National Health Commission (the "NHC") and the National Medical Products Administration will formulate and adjust guiding principles for distinguishing biomedical technologies from drugs and medical devices based on scientific and technological advancements.  We understand the rationale behind this provision is to both clearly reflect the distinctions between the two categories and allow flexibility for timely adjustments to the relevant boundaries.

Third, it is notable that New Biomedical Technologies are also subject to other applicable medical technology regulations.  For example, the Measures for the Administration of Clinical Application of Medical Technologies in 2018 established a list of prohibited medical technologies.  To date, technologies such as surrogacy technology, cloning therapy, and other biomedical technologies that raise significant ethical concerns remain prohibited from clinical application.

Interpretation of core systems

The Regulations primarily focus on two core activities concerning New Biomedical Technologies: clinical study (involving studies and trials to evaluate safety, efficacy, scope of application, operational procedures, and technical requirements) and clinical translation and application (referring to the translation and application in clinical practice after the completion of studies).

I.   Clinical study

The clinical study on New Biomedical Technologies adopts a filing-based approach.  The scope of clinical study on New Biomedical Technologies is clearly defined and includes studies that (a) directly operates on the human body; (b) manipulates ex vivo cells, tissues, or organs that are subsequently implanted or introduced into the human body; (c) manipulates human germ cells, zygotes, or embryos that are later implanted into the human body for development; and other methods as specified by the NHC.

Regarding participating entities, the Regulations distinguish between the roles of clinical study initiators and clinical study institutions.  Entities that initiate clinical study on New Biomedical Technologies should be legally established entities within China and are no longer limited to medical institutions.  This implies that the Regulations allow medical institutions to initiate such clinical studies (i.e., investigator-initiated trials, IITs) while also allowing biopharmaceutical companies to initiate such studies (i.e., industry-sponsored trials, ISTs).

Regarding the requirements clinical study institutions, the Regulations stipulate that such institutions must be Class A tertiary medical institutions.  It is important to note that these institutions must have "clinical study academic committees and ethics committees that meet the requirements".  Besides the existing ethics regulations, such as the Measures for the Ethics Review of Life Sciences and Medical Research Involving Humans, whether additional or more detailed requirements will be specified remains uncertain and should be closely monitored.

Regarding the filing procedure, clinical study institutions are required to complete the filing within five (5) working days after the academic and ethics review are concluded.  Compared with the approval-based approach set out in the Draft, the Regulations' shift to a filing-based management system simplifies the administrative procedures.  The specific filing process remains subject to further clarification in subsequent implementing rules.

The Regulations also place emphasis on post-filing supervision: the NHC has the authority to evaluate filed clinical studies.  If any technical or ethical risks are identified, the NHC may require the study to be suspended, adjusted, or terminated.  Additionally, the Regulations no longer adopt the mechanism set out in the Draft, which implemented a tiered management system at provincial and national levels by degree of risk.  Instead, the NHC assumes unified responsibility for filing management and evaluation oversight, highlighting a regulatory approach that is filing-based with post-filing oversight.

The Regulations also include specific provisions regarding preclinical studies, amendments to filings, retention periods for records and raw materials, technical support from third-party institutions, periodic research reporting, and circumstances under which clinical studies may be suspended or terminated.  Notably, if serious adverse events (SAE) occur during a clinical study on New Biomedical Technologies, the clinical study institution is required to suspend such study.  This reflects the regulatory authorities' cautious approach to ensuring the safety and stability of such new technologies while promoting innovation.

II.   Clinical translation and application

The clinical translation and application for New Biomedical Technologies have long been a major focus of the industry.  Compared with the clinical study, it allows medical institutions to charge patients in accordance with regulations, marking a crucial step toward the value-based clinical application of these technologies.

Under the Regulations, the clinical translation and application for New Biomedical Technologies operate under an approval-based framework.  The Regulations explicitly provide the approval process, time limits, and clinical application scenarios, while also introducing mechanisms such as re-evaluation and priority review.  This demonstrates support for the clinical translation and application of New Biomedical Technologies that have been proven safe and effective through clinical study and that comply with ethics principles.

Regarding the entities involved in the approval process, the applicant for clinical translation and application is the clinical study initiators (in contrast to the Draft, which required medical institutions to submit the applications).  The NHC serves as the authority responsible for accepting and approving applications, marking the elimination of the two-tier review process outlined in the Draft and simplifying procedures.  The Regulations also specify that application materials will be forwarded to a professional institution for evaluation.  Whether this professional institution is an internal body similar to China's Center for Drug Evaluation (CDE) or a third-party entity is yet to be determined.  Additionally, upon completion of a clinical study, clinical study institutions shall report their findings and recommendations for translation and application to the NHC, underscoring their key role in the process.

For the approval process, the NHC will forward materials to the professional institution for evaluation within five (5) working days of acceptance, and will make a decision within fifteen (15) working days of receiving the evaluation opinion.  The Regulations do not specify the timeframe for the professional institution's evaluation, indicating that the review may be relatively lengthy.  One of the key distinctions from drug and medical device review is that the professional institution conducts an ethics evaluation concurrently with the technical evaluation.

Upon approval, the name of the technology, the qualifications required for medical institutions and health professionals applying this technology, and operational guidelines for clinical application will be publicly disclosed.  Notably, subjects shall not be charged any fees related to the clinical studies of the New Biomedical Technologies; however, medical institutions may charge fees for clinical application in accordance with Regulations.  This may establish an additional pathway for biomedical technologies in terms of clinical implementation and commercialization beyond traditional drug and medical device's marketing authorization.

The Regulations also establish a re-evaluation mechanism.  For approved technologies that cause severe adverse reactions or uncontrollable risks, the regulatory authorities may prohibit their clinical application if re-evaluation confirms they cannot ensure safety and efficacy, thereby providing effective post-approval supervision.

Impact of the Regulations and practical implications

The Regulations, as an administrative regulation promulgated by the State Council, integrate provisions from the Administrative Measures for Clinical Study on Stem Cells (Trial) and the Guidelines for Clinical Study on Somatic Cell (Trial), thereby implementing the framework for the clinical study and clinical translation and application of New Biomedical Technologies.  For enterprises, research institutions, and medical institutions engaged in clinical studies (e.g., IITs) on cutting-edge technologies in the fields of cell and gene therapy (CGT), including CAR-T/NK, stem cell therapies, as well as assisted reproductive technologies, etc., the Regulations offer a legal basis to support the clinical activities of such New Biomedical Technologies.

The Regulations provide a formalized pathway and stable expectations for the New Biomedical Technologies from clinical study to clinical application.  For example, under the Administrative Measures for Clinical Study on Stem Cells (Trial), completed stem cell clinical studies cannot directly proceed to clinical applications.  Therefore, in practice, relevant companies had to pursue commercialization through drug marketing authorization.  The implementation of the Regulations clarifies the preconditions for clinical application, transforming the previous prohibitive rules into a basis for practical implementation.

Considering that New Biomedical Technologies require prudent evaluation of safety, efficacy, and ethics compliance, the implementation of the Regulations presents both new opportunities and compliance requirements for relevant entities.  Key takeaways for reference include, but are not limited to:

For potential clinical study initiators of New Biomedical Technologies:

• Companies should continuously monitor legislative developments, including requirements for conducting clinical studies and applying for clinical translation and application, for preparation of compliant preclinical study, clinical activities, and translation applications after the Regulations come into effect;

• For ongoing studies on relevant technologies (e.g., stem cell and somatic cell IIT projects), given the relationship between the Regulations and existing regulations or guidance remains to be further clarified, it is still advised to review the requirements under the Regulations and prepare accordingly before the Regulations officially take effect;

• For ongoing clinical trials conducted for drug or device registration purposes, applicable laws and regulations related to clinical trials, such as Drug Administration Law and the Regulations on the Supervision and Administration of Medical Devices shall continue to apply;

• When collaborating with medical institutions on related clinical studies, it is necessary to select qualified Class A tertiary medical institutions and prepare for filing procedures in accordance with the Regulations.  When entering into clinical study agreements with relevant medical institutions, appropriate attention should be paid to the special provisions outlined in the Regulations.

For potential clinical study institutions of New Biomedical Technologies:

• Assess whether it meets relevant requirements and monitor the release of further detailed rules for clinical study institutions (if any);

• Pay attention to provisions in the Regulations that differ from drug clinical trials (e.g., retention period for records and raw materials) and update internal clinical study standard of procedures accordingly;

• Place emphasis on academic and ethics review of relevant clinical studies and improve the relevant review systems.

Conclusion

The formal promulgation of the Regulations marks a new stage in the regulatory framework for cutting-edge biomedical fields in China.  At the policy level, it sends a clear signal encouraging innovation and promoting translation and application of such cutting-edge technologies, while also providing institutional support for the stable implementation of relevant New Biomedical Technologies under the premise of ensuring safety, efficacy, and ethics compliance.  Prior to the implementation of the Regulations, additional supporting rules, such as guidelines for distinguishing biomedical technologies from drugs and medical devices, application guidelines for clinical translation and application, relevant technical requirements for technical evaluations, and ethics requirements, are expected to be gradually released, requiring ongoing attention from industry stakeholders.

[1]Jingjing Xu has contributions to this article.