From “transaction expansion” to “certainty-driven expansion”: How can innovative Chinese drugs turn promising assets into global value?
Over the past few years, the overseas expansion of Chinese innovative drugs has undergone significant change.
From the number of deals and asset types to collaboration models, Chinese innovative drugs have gradually become part of the global innovation ecosystem. In next-generation technology platforms such as antibody drug conjugates (ADCs), bispecific/multispecific antibodies, cell therapies, oligonucleotides, and radiopharmaceuticals, Chinese biopharma companies are increasingly becoming an important source of assets in the global partnering landscape. [1][9]
The blue book A Winning Strategy and Practical Guide for Chinese Innovative Drug Going Global, co-published by Thermo Fisher Scientific and Pharmcube, points out that the central question for Chinese innovative drugs going global is shifting from “closing a deal” to “delivering value,” and from “competing on opportunity” to “competing on certainty.” [1]
This transition is especially important for Chinese biopharma companies. For many companies, going global is not only a business development event or a source of capital; it is also a validation of global value and an upgrade of the research and development (R&D) system. In the next stage of global competition, the focus is no longer simply on selling the product to an overseas partner, but on how a Chinese innovative asset can withstand scrutiny across global regulatory, global clinical, global quality, and global commercialization systems; secure sustained development investment; and ultimately serve patients worldwide.
Going global: From transaction opportunities to system capabilities
The overseas expansion of Chinese innovative drugs has become a new industry norm driven by both internal and external factors. [1]
Internally, China’s innovation capabilities continue to advance, but the domestic market still faces structural challenges such as intense competition, reimbursement constraints, and increasing concentration in popular targets and indications. As a result, many innovative drugs, even those with strong clinical potential, may struggle to fully realize their R&D investment and clinical value in the domestic market. [1][9]
Externally, the U.S. and Europe remain critical markets for value realization in innovative drugs, while multinational pharmaceutical companies continue to face pipeline-refresh pressure and patent cliffs. Chinese innovative biopharma companies have built differentiated capabilities in engineering innovation, R&D efficiency, and certain platform technologies, making them an important source of assets within the global business development ecosystem. [1][10]
But this does not mean that having an asset automatically leads to success overseas. Over the past few years, the industry has seen cases where deals underperformed after signing, rights were returned, overseas registration was blocked, or CMC issues caused review delays and even complete response letters. The blue book also notes that CMC and supply-chain compliance, overseas clinical execution, regulatory barriers, and commercialization capabilities have become systemic challenges that Chinese companies must confront in the process of going global. [1]
Defining global value as the starting point of global strategy
When many innovative drug companies begin BD discussions, the first questions they ask are often:
- Who will buy it?
- What is the valuation?
- How much is the upfront payment?
These questions are certainly important, but from a strategic perspective, a more fundamental question should come first: What is the global value proposition of this asset?
In other words, what unmet clinical need does it address, and for which patient population? Compared with the current standard of care in target markets, what incremental benefit can it deliver? Can that benefit be understood by regulators, clinical experts, payers and potential partners alike?
This requires companies to establish a clear target product profile (TPP) early in development. The TPP should not be merely an internal R&D document; it should serve as a shared language connecting clinical development, regulatory strategy, medical strategy, commercialization assumptions, and business development narrative.
The Center for Drug Evaluation (CDE) of China’s NMPA emphasizes in its Technical Guideline for Clinical Evaluation in Drug Clinical Trial Applications that drug clinical development should comprehensively determine target indications based on disease mechanism, the investigational drug’s mechanism of action, pharmacology, toxicity, and other characteristics, while focusing on clinical needs. Key design elements of clinical trial protocols should also be supported by prior nonclinical and/or clinical data. [2]
From this logic, three common misconceptions should be avoided.
- Do not equate China’s clinical advantages with global registration advantages. China has already established important strengths in patient enrollment efficiency, investigator resources and clinical execution. However, global development still requires addressing consistency of benefit-risk in multi-regional and multi-ethnic populations, while fully accounting for differences across countries and regions in standard of care, clinical practice, endpoint acceptance, companion diagnostic access, and regulatory review logic. Only by combining China’s clinical advantages with global evidence design can an asset better support international registration and global value realization.
- Do not mistake differentiated messaging for differentiated evidence. True differentiation comes not only from a mechanism story, but from verifiable data, including efficacy, safety, tolerability, convenience of administration, quality of life, combination potential, and health-economic value.
- Do not mistake closing a deal for delivering value. The true measure of value is whether an overseas partner continues to invest in the asset, advances it through clinical development, secures regulatory approval and brings it successfully to market.
Design global evidence from the early stages
For assets intended for simultaneous development or registration in major markets such as the U.S. and Europe, relying solely on China data and adding bridging data later is becoming a less predictable model. Multi-regional clinical trials (MRCTs) should enter global development strategy discussions earlier. [1]
This aligns with ICH E17 and the CDE’s regulatory logic regarding MRCTs. An MRCT is not simply adding a few more countries; it is a clinical trial conducted under a single protocol and in a harmonized manner across multiple regions, generating a data package that can be understood and assessed by regulators in multiple regions. [3][5]
From a sponsor perspective, an MRCT strategy must answer at least three questions.
- What regional differences matter? Epidemiology, diagnostic and treatment pathways, standard of care, ethnic or genetic background, PK/PD, prescribing habits, access to companion diagnostics, and endpoint assessment practices may all affect treatment effects. The CDE MRCT guideline also explicitly states that MRCTs should focus on intrinsic and extrinsic factors that may affect treatment effects across regions. [3]
- Which early China data can inform the global development plan? Early clinical studies are not only for confirming safety and preliminary efficacy; they should also serve early dose selection, exposure-response relationships, target population definition, biomarker strategy, and regional difference assessment. [3]
- Are sample size, regional allocation, and data quality sufficient to support regulatory interpretation? An MRCT is not globalization in form alone; it must ensure that efficacy and safety data from major regions are interpretable and can withstand regulatory review and partner due diligence. [3]
In other words, global clinical development should not wait until Phase III to become global. From first-in-human (FIH), proof of concept (POC), and even the pre-IND strategy design stage, sponsors should already be considering target markets, evidence pathways, and regulatory acceptability.
Quality is built into the design: Making global evidence withstand scrutiny
ICH E6(R3) emphasizes that GCP is the international ethical, scientific, and quality standard for protecting the rights, safety, and well-being of trial participants and for ensuring the reliability of clinical trial results. [4]
Compared with earlier interpretations that focused more on process compliance, E6(R3) further emphasizes that clinical trial quality should be built into scientific and operational design rather than relying on post hoc remediation. The principles of quality by design, proportionate risk management, critical-to-quality factors, data integrity, and sponsor oversight have very practical implications for Chinese innovative drugs going global. [4][6]
For sponsors, this means that even if the clinical trial is outsourced to a contract research organization (CRO), the sponsor cannot outsource final responsibility. CROs can execute, support, and manage clinical operations, but the sponsor must still maintain sufficient oversight of participant rights, safety, data reliability, and trial quality. [4]
Therefore, when selecting global clinical partners, sponsors should not only compare pricing, enrollment speed, and project manager responsiveness, but also assess whether the partner understands target-market regulatory requirements, has multi-regional clinical operations experience, can help proactively identify critical quality factors, and can integrate protocol science, operational feasibility, data integrity, and regulatory communication.
This is also the deeper meaning of the blue book’s point that the CRO’s role is shifting “from cost outsourcing to strategic symbiosis.” [1] In the overseas expansion process, the value of a CRO is not only to finish the trial, but to enable the sponsor to transform the asset into a globally acceptable, auditable, reproducible and interpretable evidence package.
From efficacy signals to benefit-risk evidence
In recent years, regulatory trends represented by FDA Project Optimus and ICH E6(R3) sent a clear signal: beyond efficacy evidence, regulators are paying increasing attention to whether dose selection, population definition, and dosing regimens are sufficiently justified and whether they support a better benefit-risk balance. [4][7][8]
This does not mean regulators are weakening efficacy requirements. On the contrary, it means that on the basis of proving efficacy, companies must also explain more systematically why a particular dose, patient population and dosing regimen were chosen; whether those choices have been adequately validated; and whether the resulting benefit-risk balance can support subsequent registration and clinical use.
This shift will directly affect early development design. Companies need to place greater emphasis earlier on clinical pharmacology and evidence generation, including rational dose exploration, exposure-response analysis, biomarker strategy, translational medicine research, and long-term safety assessment.
This is especially true in oncology innovation areas with complex mechanisms, such as ADCs, bispecific antibodies, and T-cell engagers, where regulators and potential partners are no longer focused solely on early efficacy signals, but increasingly on the overall benefit-risk profile of the product. For example, is the current dose sufficiently optimized? Is safety manageable in the long term? Is the target patient population clearly defined? Does the asset have development potential to support future combinations and indication expansion?
From the perspective of business development and global development, an asset with a clear dose rationale, robust translational evidence, and predictable safety characteristics can typically provide stronger support for regulatory communication, clinical development, and commercialization decisions, while also making it easier to gain partner recognition and demonstrate long-term development value.
Turning “China efficiency” into “global trust”
China’s efficiency advantage in innovative drug development has already been recognized globally. The next question for the industry is: how can that efficiency be recognized by global regulatory systems, trusted by global partners and ultimately serve patients worldwide?
The blue book proposes “reshaping global value.” The key is not to tell a more exciting globalization story, but to build a more verifiable global capability system. [1]
The next stage of competition for Chinese innovative biopharma companies going global will no longer be simply whose asset is seen first, but who can first convert scientific advantage into global evidence advantage, regulatory communication advantage, operational execution advantage, and quality delivery advantage.
In this process, we endeavor to work with Chinese innovative biopharma companies to promote a more rational, scientific and sustainable path to globalization: designing evidence to global standards from early development; building trust through quality systems from clinical execution; safeguarding long-term value through governance mechanisms from the point of transaction; and, from the moment products go overseas, ensuring that Chinese innovation truly enters the treatment options available to patients worldwide.
The next phase of Chinese innovative drugs will be defined by certainty.
And certainty comes from science, as well as from systems.
References and Sources
[1] Thermo Fisher Scientific; Pharmcube. Reshaping Global Value: A Winning Strategy and Practical Guide for Chinese Innovative Drug Going Global [R]. 2026.
[2] Center for Drug Evaluation, NMPA. Technical Guideline for Clinical Evaluation in Drug Clinical Trial Applications [S]. Apr 2026.
[3] Center for Drug Evaluation, NMPA. Guideline on Benefit-Risk Assessment Using Multiregional Clinical Trial Data in the Global Simultaneous Development of New Drugs (Trial) [S]. Feb 2026.
[4] International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use. ICH Harmonised Guideline: Guideline for Good Clinical Practice E6(R3) [S]. Final version, adopted on 06 January 2025.
[5] International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use. ICH E17: General Principles for Planning and Design of Multi-Regional Clinical Trials [S]. Final version, adopted on 16 November 2017.
[6] International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use. ICH E8(R1): General Considerations for Clinical Studies [S]. 2021.
[7] U.S. Food and Drug Administration. Project Optimus [EB/OL]. FDA Oncology Center of Excellence.
[8] U.S. Food and Drug Administration. Optimizing the Dosage of Human Prescription Drugs and Biological Products for the Treatment of Oncologic Diseases: Guidance for Industry [S]. Aug 2024.
[9] Pharmcube. 2025 Trends in China’s Innovative Drug Development [R]. Mar 2026.
[10] Insight Database. 2020–2026Q1 Pharmaceutical Deal Report: China Pharma Deal Market from Recovery to Restructuring [R]. Apr 2026.
[11] School of Pharmaceutical Regulation Science, Tsinghua University; Pharmcube. Global Innovative Drug Clinical Trial Trends Insight (2015–2025) [R]. May 2026.
[12] Pharmcube. 2025 China Clinical Trial Data Insight [R]. 2026.
Note: This article is an industry observation and professional analysis, using public guidelines, industry reports, and regulatory materials as the basis for discussion. The transaction data, pipeline data, clinical trial trend data, and market judgments mentioned herein are primarily derived from the cited industry reports and their database methodologies. Regulatory and clinical development views are based mainly on CDE, ICH, and FDA guidance documents and official materials. This article does not constitute medical advice, clinical treatment advice, regulatory filing conclusions, commitments to a registration pathway, commercial partnership commitments, or investment advice.