Seeking the Future Between Innovation and Efficiency: Insights from China's CGT Regulatory Landscape — An Exclusive Interview with a Senior Chinese Expert in Pharmaceutical Policy

The development of China’s cell and gene therapy industry has been inseparable from the rapid iteration of its regulatory environment and institutional support. In the view of the aforementioned experts, China’s drug regulatory system has undergone a “silent revolution” over the past decade.

“If you look back at the drug review and approval system before 2015, you will find that the policies at that time were predominantly characterized by ‘restriction’ and ‘prudence,’” the expert candidly stated. “However, as China elevated biomedicine to a strategic emerging industry at the national level, regulatory policies began to shift from ‘control’ to ‘guidance.’ Particularly in frontier fields such as cell and gene therapy (CGT), a virtuous cycle of interaction between regulation and innovation has gradually taken shape.”

She pointed out that China has demonstrated remarkable "late-mover advantage" in the release of regulatory guidelines for CGT.Statistics show that from 2020 to 2024, technical guidelines related to cell and gene therapy (CGT) issued by China’s National Medical Products Administration (NMPA) accounted for more than 70% of the total. Although the United States and the European Union started earlier, their update pace during the same period was relatively steady. “In approximately five years, China has rapidly caught up with, and in some areas even surpassed, the accumulation achieved by Europe and the United States over the past decade or more in terms of building a systematic framework and ensuring comprehensive content coverage for its technical guidance documents.”

Behind this “explosion of guidelines” lies the Chinese regulators’ keen grasp of industry needs and their institutional response. The aforementioned experts specifically highlighted the Draft for Comments on the Classification of Advanced Therapy Medicinal Products (ATMPs) released by the National Medical Products Administration in June 2025, regarding it as a significant milestone in aligning China’s CGT regulatory framework with international standards.

“The concept of ATMP originated in the European Union. Its introduction and localization in China signify that we are gradually aligning with global standards in classification logic, review criteria, and manufacturing requirements. This not only facilitates multinational pharmaceutical companies in conducting synchronized R&D in China but also lays the foundation for Chinese innovative products to enter international markets.”

Meanwhile, China has established a “fast-track” review mechanism with distinct local characteristics.Although China has not established a RMAT pathway exclusively for CGT like the United States, it has built an accelerated review system covering the entire product lifecycle through three policies: “Breakthrough Therapy Drug,” “Conditional Approval,” and “Priority Review.”

“Taking breakthrough therapy drugs as an example, companies can apply at the end of Phase I or the beginning of Phase II clinical trials. Once approved, they can establish a more close communication mechanism with the Center for Drug Evaluation (CDE), which greatly reduces the uncertainty of R&D.” The above expert added, “The conditional approval policy allows products that show significant efficacy in early-stage clinical trials to benefit patients earlier, while companies continue to complete confirmatory clinical trials.”

However, the “China speed” of regulation also faces dual challenges from internal resources and external expectations.The aforementioned experts revealed that the team at the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) responsible for cell and gene therapy (CGT) is severely understaffed, yet they maintain exceptionally high work efficiency, “essentially relying on a lean, highly capable team to meet the review demands of the entire industry.”

She also pointed out that, similar to Japan, China has a certain degree of “dual-track” management in the field of cell and gene therapy (CGT), whereby some products are regulated as pharmaceuticals while others are managed as medical technologies. “A dual-track system does not imply opposition; what is more important in the future is achieving mutual recognition of data and policy coordination between the two tracks, thereby facilitating the industrial translation of more research achievements.”

In the research, development, and clinical stages of cell and gene therapy (CGT), China has evolved from a former “bystander” into a key “participant.” This expert outlines China’s position in the global CGT R&D landscape with a set of data:

“By the end of 2024, there were a total of 11,923 clinical trials in cell therapy worldwide, with China accounting for approximately 20% and the United States for about 40%; in the field of gene therapy, there were 4,792 clinical trials globally, with China and the United States each accounting for 40%, truly achieving an ‘even split.’”

She further pointed out that China’s number of clinical trials in the field of gene therapy has kept pace with that of the United States, thanks on one hand to the accumulation of platform technologies by domestic enterprises in areas such as AAV vectors and gene editing, and on the other hand to China’s large patient population, diverse disease spectrum, and concentrated clinical resources.

“From 2017 to 2023, the number of clinical trial applications for CGT products in China increased by more than 15-fold. Cell therapy CTAs rose from 6 to 146, while gene therapy CTAs grew from 1 to 36. This growth trend is extremely rare on a global scale.”

In this expert's view,The focus of China’s CGT R&D is undergoing a significant shift: “In the early stages, there was a rush to develop CAR-T therapies for hematologic malignancies, given the relatively mature technology and clear development pathways. However, an increasing number of companies are now venturing into ‘deep-water’ areas such as solid tumors, autoimmune diseases, and universal cell therapies.”

She particularly emphasized the bridging role of “Investigator-Initiated Trials (IITs)” in the development of cell and gene therapy (CGT) in China. “Cell therapy products originate from innovations and advancements in medical technology, possessing attributes of both pharmaceuticals and medical technologies. Over the past decade, Chinese physicians have accumulated extensive practical experience through numerous IITs, including therapeutic practices, patient management, risk management and control, and combination therapies. These experiences have significantly facilitated the translation of cell therapy from clinical techniques into commercial products.”

Citing the collaboration between Legend Biotech and Johnson & Johnson as an example, the aforementioned experts highlighted the value of Investigator-Initiated Trial (IIT) data: “Even though deaths occurred in the early-stage IITs, Johnson & Johnson was still able to recognize the technological potential from the data. This demonstrates that high-quality IITs serve not only as a platform for technical validation but also as a catalyst for product translation.”

She further pointed out that investigator-initiated trials (IITs) have bridged the gap between physicians and the industry, enabling clinical needs to be fed back to the R&D side more rapidly. “Physicians in China are not merely executors of treatment, but also participants and drivers of innovation.”

However, the expert also cautioned that a surge in the number of clinical trials does not equate to a commensurate improvement in quality. “There remains a gap between China and Europe and the United States in areas such as clinical trial design, endpoint selection, and data management. In particular, more experience needs to be accumulated in the design and conduct of multicenter, randomized controlled trials.”

She urged companies to place greater emphasis on data standardization and international comparability when advancing clinical trials. “Only high-quality data can support international product registration and market access.”

Despite significant progress in research and development as well as regulatory review within China’s cell and gene therapy (CGT) industry, the expert explicitly pointed out that the key to achieving sustainable development lies in overcoming the two major practical bottlenecks of “cost” and “payment.”

It is understood that the current production cost of a CAR-T product in China is approximately several hundred thousand yuan. Even with pricing at the million-yuan level, corporate profit margins remain very limited. As this expert analyzed, “Health insurance authorities seek price reductions, patients expect accessibility, and companies require returns—this is a classic trilemma.”

Regarding the market call to “reduce CAR-T costs to RMB 100,000,” the aforementioned experts expressed cautious optimism. “Cost reduction cannot be achieved by simply compressing existing processes; it must rely on fundamental transformations in technological pathways and production models.”

In this regard, the industry has also proposed four possible pathways for cost reduction:

First is production automation. "Fully enclosed, automated all-in-one production systems can significantly reduce reliance on GMP-compliant facilities and manual operations. Some domestic companies are already experimenting with such equipment, but the challenges lie in maintaining quality consistency after process changes and gaining regulatory approval for the new processes."

Second is the standardization of technology. “Off-the-shelf CAR-T is widely recognized in the industry as a key direction for cost reduction. If ‘off-the-shelf availability’ can be achieved, not only will prices drop significantly, but treatment accessibility will also be greatly enhanced.”

Third, in vivo gene editing. “If gene repair or regulation could be achieved through a single injection, it would completely eliminate the complex processes of ex vivo cell preparation, fundamentally transforming treatment paradigms and cost structures.”

Fourth, expand its application scenarios. Currently, it is limited to hematologic malignancies, which have a very small patient population. If technological breakthroughs in the future enable its application to expand into solid tumors and autoimmune diseases, production capacity will increase significantly. This will also substantially reduce the cost of upstream technologies, thereby lowering the overall product cost.

Regarding payment mechanisms, relying solely on basic medical insurance is impractical. Given the high cost of cell and gene therapy (CGT) products and the dispersed nature of the patient population, full coverage by public health insurance is unsustainable. The aforementioned experts also recommend exploring a “multi-payer system,” incorporating models such as commercial insurance, installment payments, outcome-based payment, and special funds.

“China needs to pursue greater institutional innovation in its payment mechanisms. For instance, can outcome-based payment models be established? Can exclusive commercial insurance products tailored to innovative therapies be introduced? These are avenues worth exploring.”

At the conclusion of the interview, the expert summarized that China’s CGT industry is currently in a development phase that emphasizes both “efficiency and innovation.”

“We boast a globally leading volume of R&D, a continuously optimized review and approval system, a resilient industry sector, and extensive clinical experience accumulated through investigator-initiated trials (IITs). These are our strengths,” she said. “However, we still face practical challenges in terms of cost, reimbursement, and international recognition.”

She believes that future breakthroughs in the industry will focus on three key areas: first, achieving cost reduction and efficiency improvement through technological innovation; second, demonstrating the irreplaceability of products by proving their clinical value; and third, enhancing global influence through international cooperation. “Cell and gene therapy is not only a technological revolution but also a redefinition of the value of life.”