BACKGROUND:Many countries have accelerated the approval of innovative drugs through breakthrough therapy policies to meet public health needs. China needs to optimise its policy system to narrow the gap with international practices. OBJECTIVE:To identify shortcomings in China's breakthrough therapy policies and formulate optimization strategies via quantitative analysis of their dynamic evolution and cross-jurisdictional comparisons. METHODS:Adopting a quantitative design, this study employs AI technologies (LDA topic modelling + time-series deduction), combined with cross-jurisdictional policy element matrix comparison and typical drug case analysis, to systematically analyse the evolutionary path and implementation effects of China's breakthrough therapy drug policies. RESULTS:Quantitative analysis of 59 policy texts (2020-2024) reveals that China's policies have undergone a three-stage evolution: 'approval-driven (2020-2022)-collaborative transition (2023-2024)-ecological construction (exploratory scenario deduction after 2024)'. The core topics focus on 'drug review', 'industrial innovation', and 'medical services', among which 'drug review' and 'industrial innovation' form a dual-dominant pattern. However, the persistently low intensity of the 'medical services' topic reflects an ecological imbalance of 'accelerated approval-industrial innovation-lagging accessibility'. Cross-jurisdictional comparisons show gaps in early innovation incentives, international collaboration, and medical support. CONCLUSIONS:Based on the quantitative analysis of policy texts, this study proposes six optimization suggestions. These targeted strategies aim to address the imbalance of 'fast approval but slow accessibility,' support the 'Healthy China 2030' strategic goal, and provide references for developing countries in balancing resource allocation, accelerating research and development through the integration of artificial intelligence, and avoiding accessibility disparities via risk-sharing mechanisms.

Advanced therapeutic medicinal products represent a broad spectrum of biopharmaceutical innovations that prominently feature cell and gene therapy formulations. These cutting-edge therapeutic modalities are specifically engineered to intervene in and prevent illnesses that historically necessitated continuous, long-term management. To visually portray the most recent trajectories in cell and gene therapy investigations and to map out prevailing global advancements, a bibliometric assessment was conducted. This analysis meticulously sourced its data from the Web of Science Core Collection database and was significantly enhanced by analytical platforms such as VOSviewer and CiteSpace. Cell and gene therapy products demonstrate considerable promise for both foundational scientific inquiry and diverse clinical interventions, with their utility now extending far beyond cancer treatment to encompass a wide array of other pathological conditions. This paper provides a comprehensive examination of the evolving trends, collaborative endeavors, and burgeoning frontiers within the dynamic landscape of cell and gene therapy products.

Pediatric asthma is a non-communicable disease with a significant burden, and many patients cannot receive early diagnosis and effective control of the disease. Exploring the role of pharmacists in the management of pediatric asthma can provide possibilities for improving the management of pediatric asthma. The purpose of this simulation study is to quantify the 12-month disease burden reduction of pediatric asthma under pharmacist-led pharmaceutical care using QALY metrics, and to further estimate the short-term cost-effectiveness of pharmacist-led pharmaceutical care within a one-year time horizon. From a payer perspective, a Markov model was developed using cohort simulation to simulate 2 contrasting scenarios regarding to pediatric asthma management in China: the status quo scenario, and a pharmaceutical care scenario based on providing pharmaceutical care to all pediatric asthma patients. The model cycle length was one month and simulated a one-year time range. The results of the model were the direct medical expenses and quality adjusted life years associated with pediatric asthma. Both deterministic sensitivity analysis and probabilistic sensitivity analysis were conducted to test the robustness of the results. For the status quo scenario, the calculated total cost and QALYs were CNY 1300.6 and 10.8, respectively. The corresponding values for the pharmaceutical care scenario were CNY 1324.8 and 11.2. Compared with the status quo scenario, the pharmaceutical care scenario increased the cost by CNY 60.3 for every additional unit of QALY obtained. Therefore, for the WTP thresholds of CNY 89,358/QALY, the pharmaceutical care scenario was less than the thresholds and was cost-effective. Both deterministic and probabilistic sensitivity analyses demonstrated that the pharmaceutical care scenario was always more cost-effective than the status quo scenario. Expanding the scope of pharmacist services has a significant return on investment for pediatric asthma management. This study has several data and modeling limitations stemming from insufficient real-world evidence, and further comprehensive real-world research is needed to validate our findings.

ObjectiveChina released two batches of the Rare Disease Catalog in 2018 and 2023, respectively, listing a total of 207 diseases. This study aims to analyze the accessibility of medications for diseases included in the catalog and assess the impact of drug regulatory reforms on the supply of medications for rare diseases.MethodsThis study compiled five categories of information on drugs for 207 rare diseases: basic information on drug availability, the number of drug approvals and production capacity, drug development trends, marketing approval efficiency, and medical insurance coverage. Descriptive statistical analysis was employed.ResultsBy the end of 2025, out of 207 rare diseases, 98 (47.3%) had drugs approved in China, with a single company being the exclusive supplier for 37 of these diseases. Between 2015 and 2025, the NMPA approved a total of 2,007 Investigational New Drug (IND) applications and 1,134 New Drug Applications (NDAs) for rare diseases, showing an overall upward trend. Following the publication of the catalog, the median review time for NDA applications for rare disease drugs was reduced by 79 days compared to pre-publication (516 vs. 595, P < 0.001); for drugs eligible for Priority Review and Approval (PRA), the median review time was reduced by 73 days compared to non-PRA drugs (493 vs. 566, P < 0.001). The time difference between the market launches of the same drug in China and the United States has been reduced by 1.52 years (5.53 vs. 4.01, P < 0.001). Of the 263 drugs used to treat 98 rare diseases, 181 were included in the medical insurance coverage, and the time to inclusion in the insurance coverage was reduced by 1.01 years following the publication of the directory (2.09 vs. 1.07, P < 0.001).ConclusionWith the release of the rare disease catalog and the advancement of drug regulatory reforms, access to orphan drugs in China has improved. However, more than half of all rare diseases still face the dilemma of having no available treatments. It is recommended that the coordination mechanism between drug regulation and medical insurance be maintained to better meet patients’ clinical medication needs.

Purpose- With the explosive growth of e-commerce and User-Generated Content (UGC), online reviews are critical for understanding consumer preferences and product issues. Yet their multi-dimensional, colloquial, domain-specific traits challenge traditional methods, while deep learning has high costs and low interpretability. This study aims to build a framework balancing insight depth and practicality-to capture dynamic customer priorities across periods and products, and offer actionable insights for pharmaceutical e-commerce platforms. Design/methodology/approach- Using anti-infective drugs as an example and online reviews from JD Pharmacy, we integrated the Latent Dirichlet Allocation (LDA) model with multiple tools (perplexity, coherence, and pyLDAvis) to extract influencing factors with higher precision. We then constructed and refined a domain-adapted sentiment lexicon for accurate sentiment quantification. Finally, we combined a multi-attribute model with PROMETHEE-II to ensure a reliable ranking of factor importance across different dosage forms and periods. Findings- The framework successfully extracted period-specific topics and significantly improved sentiment classification accuracy by 19.38 percentage points compared to traditional methods (p < 0.05). Consumer priorities exhibited significant two-dimensional dynamics (period & times; dosage form), and the priority rankings were proven statistically robust across all independent scenarios via Bootstrap resampling and non-parametric tests (p < 0.05). Based on these findings, targeted suggestions for improving satisfaction were proposed. Originality/value- This study systematically constructs an integrated text mining framework embedded with a dual-dimensional contextual logic. It establishes a standardized, low-cost, interpretable, and decision-closed-loop paradigm for UGC analysis, achieves the accurate quantification of consumers' dynamic priorities across different contexts, and provides a reliable pathway for vertical domain insights under resource constraints.

Chronic spontaneous urticaria (CSU) is a common inflammatory immune skin disease. It has a serious impact on the patients’ quality of life and imposes a serious financial burden on patients. The standard therapy for CSU (using antihistamines), while less costly, has limited efficacy and no longer adequately meets the clinical needs of CSU patients. Omalizumab is the world’s first and currently the only biologic approved for the treatment of CSU. Several studies have confirmed the efficacy and safety of omalizumab for CSU. However, the economics of omalizumab treatment relative to standard therapies remains unknown in China. The purpose of this study was to analyze the cost-effectiveness of omalizumab versus standard treatments for CSU after omalizumab’s inclusion in the National Drug Insurance List from the patient’s perspective. We developed a Markov model based on the Urticaria Activity Score over 7 days (UAS7). The modeling period was 4 weeks. The time horizon was 10 years. The willingness-to-pay threshold (WTP) of 1–3 times gross domestic product (GDP) per capita was selected. Incremental cost-effectiveness ratio (ICER) was calculated from the base-case analysis, and one-way sensitivity analysis and probabilistic sensitivity analysis were performed. The ICER of omalizumab treatment relative to standard treatment was ¥160,411/QALY, which was between 1 and 3 times GDP per capita. Sensitivity analyses illustrated that the direct cost of omalizumab had a significant effect on the ICER and demonstrated the stability of the results. Omalizumab treatment is a cost-effective regimen compared with standard therapy under certain circumstances. This demonstrates the important role that health insurance policies play in reducing the burden on CSU patients. However, the limitations of applying foreign clinical data in this paper and the uncertainty of cost-effectiveness at a low WTP threshold are two aspects that cannot be ignored, and subsequent related studies are needed. This study will help patients to make decisions about treatment options, and will be a reference to relevant healthcare organizations.

Background:Gaucher disease (GD) is an autosomal recessive disorder with a wide range of clinical symptoms that cause abnormal function of parenchymal organs such as liver and spleen in patients. Enzyme replacement therapy, represented by imiglucerase, is a common approach for GD treatment. However, limited efficacy and high cost are important factors restricting its use. Ambroxol has gradually attracted attention due to its ease of administration, safety, and efficacy. However, there is no pharmacoeconomic evaluation of ambroxol for the treatment of GD in China. The cost-effectiveness profile of ambroxol combined with imiglucerase therapy for the treatment of GD, as opposed to imiglucerase monotherapy, needs to be investigated. Objective:This study aimed to analyze the cost-effectiveness of ambroxol for GD in China from the perspective of the Chinese healthcare system. Methods:We constructed an eight-state Markov model based on the disease characteristics of GD. The Markov cycle was 1 month. The time horizon was 6 years. The willingness-to-pay threshold was chosen to be 1-3 times the gross national product (GDP) per capita. The incremental cost-effectiveness ratio (ICER) was calculated from the base-case analysis, and one-way sensitivity analyses and probabilistic sensitivity analyses were performed. Results:The ICER value was ¥223,726.70, which was between 1 and 3 times GDP per capita. Sensitivity analysis showed that the cost of imiglucerase had a significant effect on ICER as well as demonstrating the stability of the results. Conclusion:Ambroxol combination therapy is a cost-effective regimen compared with imiglucerase monotherapy.

BACKGROUND:As a rare disease with the largest number of cases in China, spinal muscular atrophy (SMA) causes severe motor deficits as well as multi-system organ damage. Some potent drugs for SMA, such as Nusinersen (NU) and Risdiplam (RI) represented by targeted survival motor neuron gene therapy, have become a new trend in the treatment of SMA. RI has gradually attracted attention due to its ease of administration, safety, and efficacy. OBJECTIVE:The study was to analyze the cost-effectiveness of RI and NU for the treatment of SMA in China from the perspective of the healthcare system. METHODS:We constructed a five-state Markov model based on the different stages of SMA. The willingness-to-pay threshold was chosen to be 1-3 times the gross domestic product (GDP) per capita. The base-case analysis was used to calculate the incremental cost-effectiveness ratio (ICER), and one-way and probabilistic sensitivity analyses were performed. RESULTS:The ICER value was ¥132,402.10/QALY, which was in the range of 1-3 times GDP per capita. Sensitivity analysis results showed the cost of RI and NU had a significant effect on ICER as well as proving the stability of results. CONCLUSIONS:RI is a cost-effective option compared to NU in SMA treatment.

BackgroundAn increasing number of countries worldwide, including China, have adopted Health Technology Assessment (HTA) and pharmacoeconomic (PE) principles, either comprehensively or partially, to inform drug reimbursement decisions. While China has integrated the annual cost of the drug (ACD) as a key economic factor considered in decision-making, implicitly establishing a price ceiling for medical insurance coverage. However, the current approach lacks a robust theoretical foundation and quantitative evidence.ObjectiveThis study aims to explore the rationale for incorporating ACD as a constraint in reimbursement decision-making framework, and to estimate a practical ACD threshold for China’s basic medical insurance (BMI) system.MethodsBinary logistic regression was employed to analyze the impact of ACD on patients’ financial burden. The outcome variable was the occurrence of financial barriers, with ACD serving as the primary independent variable. Covariates are factors of reimbursement benefits, including reimbursement caps and reimbursement rates. Average marginal effect analysis was performed to quantify the relationship between ACD and the likelihood of encountering barriers, suggesting a ACD threshold with practical implications for reimbursement decisions. Multicollinearity among variables was assessed using the Variance Inflation Factor (VIF). The model’s goodness of fit was assessed using the likelihood-ratio test and the Hosmer-Lemeshow test. Additionally, model performance was evaluated using the Receiver Operating Characteristic (ROC) curve.FindingsIn China, patients face significant challenges in affording high-priced medications under BMI system. Failure to consider the payment capacity of the general population in drug reimbursement decision-making can result in an inequitable allocation of basic medical insurance funds. Logistic regression analysis revealed that for each 10,000 CNY (approximately 1,431 USD) increase in the ACD, the odds of outcome occurring increased by a factor of 1.1681 (95% CI: 1.1365–1.2006, p < 0.001). The highest average marginal effect was observed at a ACD value of 400,000 CNY (0.0228; 95% CI: 0.0199–0.0256, p < 0.0001). Furthermore, when ACD exceeded 440,000–450,000 CNY, the predicted probability of financial barriers surpassed 50% (p < 0.001).ConclusionIncorporating the evaluation of ACD into the appraisal process is crucial for informing reimbursement decisions, especially in health insurance systems without robust safety net mechanisms for patients. This study innovatively estimated the value of ACD threshold, addressing the research gap and providing a methodological reference for quantitative research. Despite adopting maximized reimbursement benefits of BMI, the estimated ACD threshold appears unable to support innovative medications priced comparably to those in global markets. In the future, China should establish a risk-sharing mechanism and improve the level of medical reimbursement benefits to mitigate financial barriers for patients’ access to high-value medications.

China has implemented accelerated drug marketing registration procedures (ADMRPs) since 2016, including priority review and approval (PRA), conditional approval (CA), and breakthrough therapy drugs (BTDs). This study analyzed the characteristics of drugs approved by ADMRPs from 2016 to 2024 and explored the implementation effectiveness of these procedures. Overall, 922 drugs were approved for marketing through five ADMRPs (or procedure combinations): PRA (759, 82.3%), CA (12, 1.3%), CA + PRA (108, 11.7%), BTD + PRA (23, 2.5%), and BTD + CA + PRA (20, 2.2%). Drugs approved through the different procedures differed widely in terms of drug attributes and clinical uses, clinical trial design, and drug registration and marketing. Implementing ADMRPs has demonstrated excellent results in accelerating the development and marketing of clinically value-driven drugs.

The MONARCH 3 trial has demonstrated that Anastrozole combined with Abemaciclib is safe and effective for the treatment of postmenopausal hormone receptor-positive advanced breast cancer. However, its cost-effectiveness for long-term use has not been investigated yet. This study aims to evaluate the cost-effectiveness of Anastrozole combined with Abemaciclib versus Anastrozole used alone for patients in China. Based on MONARCH 3 trial data, we constructed a Markov model using Treeage Pro 2022 software. The model cycle was set at 1 month over a period of 20- year time horizon, and the annual discount rate was set at 5

Aiming at the information asymmetry between pharmaceutical enterprises' technological innovation decisions and government subsidy strategy, this paper establishes a differential game model consisting of the government and a single pharmaceutical company, proposes three different government subsidy strategies, and obtains an equilibrium solution with the help of the Hamilton-Jacobi-Bellman equation, taking into consideration of the transmission effect of the enterprise's reputation. First, the innovation decisions of pharmaceutical firms without government subsidies are analysed, and based on this, the optimal strategies with government subsidies for non-cooperative pacts and cooperation between the government and enterprises are analysed separately. In addition, the effects of different subsidy strategies on the government's investment efficiency, corporate reputation, and the choice of corporate innovation strategies are compared, and the results are verified by numerical analysis. Finally, based on the results of the study, references and suggestions are provided for the formulation of government subsidy policies as well as corporate innovation decisions. The results show that: government subsidies can effectively stimulate the innovation ability of pharmaceutical enterprises and improve their reputation; the more sensitive an enterprise's reputation is to the coefficient of technological innovation, the more it can improve the enterprise's innovation level; and the coordination contract of government-enterprise cooperation can realize the Pareto improvement of the benefits of the government and enterprises.

BackgroundFabry disease (FD) is an X-chromosome-linked genetic disorder. Currently, the main treatments for FD include disease-nonspecific and disease-specific treatments. Nonspecific treatment involves symptomatic management of organ involvement. On the other hand, disease-specific treatment is to regulate the activity of the corresponding enzymes, which is targeted. Among them, enzyme replacement therapy (ERT) is a classical therapy. Several studies have demonstrated the significant ameliorative effect of agalactosidase-α (ALTA-a) on cardiac and renal function in patients with FD. Despite the excellent clinical performance, there are limited pharmacoeconomic studies on ERT for FD worldwide.ObjectiveThe aim of this study was to analyze the cost-effectiveness of ERT for FD in China from the perspective of the healthcare system.MethodsWe constructed a five-state Markov model based on the disease characteristics of FD. The modeling period was 1 month. The time horizon was 3 years. The willingness-to-pay threshold was chosen as 1-3 times the gross national product (GDP) per capita. The incremental cost-effectiveness ratio (ICER) was calculated from the base case analysis, and one-way sensitivity analysis and probabilistic sensitivity analysis were performed.ResultsThe ICER value is ¥148071.95/QALY, which is between 1-3 times GDP per capita. The sensitivity analysis showed that the cost of ALTA-a had a significant effect on ICER and proved the stability of the results.ConclusionERT therapy is a cost-effective program compared to “No ERT” therapy.

Anti-cold drugs prove effective in alleviating cold symptoms and minimizing complications. In recent years, there has been a growing tendency among the public to purchase these drugs online, driven by the prevalence of influenza. Maintaining a high level of consumer satisfaction is crucial for online pharmaceutical platforms. This study introduces a Multi-Attribute Decision Making (MADM) method to analyze online reviews from 31,392 consumers at JD Pharmacy. Unlike previous studies, multiple analytical tools are employed jointly to identify factors influencing consumer satisfaction. Four dosage forms of anti-cold drugs (granule, tablet, syrup, and spray) were analyzed using the Latent Dirichlet Allocation Model (LDA). Three tools (topic coherence curve, topic perplexity curve, and pyLDAvis visualization) were jointly applied. Then five satisfaction factors were identified as logistics distribution, drug efficacy, online consultation service, drug prices, and packaging quality. PROMETHEE II method was applied to rank the above factors. The results revealed variations in consumer satisfaction among different drug dosage forms. We conducted a detailed analysis of these distinctions. The results from this study will provide an effective reference to improve consumer satisfaction.

BackgroundWith the introduction of the accelerated drug review policy in China, the clinical research and development time and the review and approval time of drugs have been shortened accordingly. Especially under the influence of the COVID-19 pandemic, the vaccine formulations released through the accelerated review policy are springing up, and the question of how the accelerated review policy affects the investment portfolio of vaccine enterprises has also attracted more and more attention.Aims and methodsThe article uses mixed-integer linear programming to develop a new model on portfolio planning for vaccine companies based on the accelerated review policy context. The model is constructed using the Gurobi extension class of .NET, and the investment decision is made and simulated by the Gurobi solver to investigate the portfolio planning decision of a vaccine company maximizing the net present value of its vaccine production portfolio with the increase of available capital over a 20-year time horizon.ResultsThe NPV under the accelerated review policy is significantly higher than the net present value under the standard review policy when the available capital exceeds RMB 900 million. And the difference between the two of them peaks at RMB 1.87 billion when the available capital is RMB 1.9 billion; break-even occurs about 1.3 years earlier in the accelerated review policy than in the standard review; and when the available capital is the same, firms in the accelerated review policy choose to produce four products earlier and make the decision to invest in facility construction earlier; scenarios in the accelerated review policy are not as sensitive to changes in model parameters as they are in the standard review.ConclusionThe accelerated review policy is effective in providing incentives for commercialisation. The results of this study will provide an effective reference for vaccine companies to make scientific portfolio planning under the accelerated review policy.

BACKGROUND:In November 2015, China launched a pilot program for its Drug Marketing Authorization Holder (MAH) system, aiming to integrate production and research and development resources to spur drug innovation. While many scholars have examined the theoretical relationship between the MAH system and pharmaceutical manufacturing innovation, empirical studies have been scarce. METHODS:This study uses inter-provincial panel data on China's pharmaceutical manufacturing industry from 2009 to 2019, along with synthetic control methods, to offer a robust analysis of the impact of the MAH paradigm on innovation quality in pilot provinces. We treat the 2015 MAH system implementation in ten provinces as a natural experiment and account for the mediating effects of R&D investments, allowing us to discern the underlying mechanisms. RESULTS:Our findings demonstrate a significant positive effect of China's MAH system on innovation quality in the pharmaceutical manufacturing industry. Specifically, the system is most effective in Jiangsu, Guangdong, and Shandong provinces. We also identify R&D investments as playing a mediating role in facilitating the favorable impact of the MAH system on innovation quality. CONCLUSION:Our study highlights the crucial importance of the MAH system in promoting innovation quality in China's pharmaceutical manufacturing industry, providing vital empirical support for its adoption and further enhancement. This research also offers practical insights for policymakers and R&D decision-makers in the pharmaceutical sector.

China has initiated drug regulatory reforms since 2015. Here, we analyze the characteristics and trends of domestic innovative drugs approved for marketing in China from January 2010 to May 2024 to explore the effectiveness of drug regulatory reform. Overall, 219 drugs were approved, with growth in chemicals and therapeutic biologics post-reform. Single-arm trials as an important option for clinical trial design of antineoplastic agents increased. The time for each link from investigational new drug (IND) to new drug application (NDA) has been shortened post-reform. Moreover, the time for access to medical insurance for approved drugs has been shortened and price reductions have been increased. China’s drug regulatory reforms have made progress in improving the accessibility of domestic innovative drugs.

BACKGROUND:Drug shortage is a worldwide problem that seriously threatens public health. China released the most comprehensive list of key drug shortage monitoring varieties ever in 2022. We aim to analyze the attributes and characteristics of the medicines within the list to provide a reference for improving China's supply security of shortage drugs. METHODS:We used public data to extract information on drug types, dosage forms, indications, classification of clinical uses, whether they were included in medical catalogs such as the National Essential Drugs, and the number of drug and active pharmaceutical ingredient (API) manufacturers. A descriptive statistical analysis was used. RESULTS:Of the 980 drugs on the list, 99.59% were chemicals and 92.65% were injectables. Drugs for blood and hematopoietic organs, the cardiovascular system, and the digestive tract and metabolism ranked among the top three shortages. Verification of the medical catalogs showed that 90.41% of the drugs belonged to the national essential drugs, 95.10% were medicare drugs, 2.55% were volume-based procurement drugs, and 14.70% were for rare diseases, and 42.04% were for children. In terms of drug supply capacity, 21.33% of drug approvals are less than 10, and there were even 26 drugs for exclusive production, close to 90% of manufacturers need to purchase APIs from outside. Among the 256 APIs included in the list, 152 APIs had less than 10 manufacturers, and there were even 5 APIs produced by only one enterprise nationwide. CONCLUSIONS:The situation of drug shortages in China was severe and complex, with serious shortages of medicines adapted to basic medical and healthcare needs and clinically necessary medicines, and a need to improve the production capacity of drugs and the ability to supply APIs. We recommend strengthening drug monitoring and stockpiling and accelerating the approval of shortage drugs to improve drug supply security.

Background Artificial intelligence (AI) technology has developed rapidly in recent years, leading to exponential growth in the AI medical industry. However, a comprehensive investigation of approved medical devices in China is needed. Methods We utilized a web crawler to collect data on all medical devices from the China National Medical Products Administration website since 2018. Through natural language processing techniques and manual analysis, we identified all medical devices developed by artificial intelligence medical devices (AIMD) companies and conducted a statistical analysis. Results Since 2018, the number of AI-related medical devices approved in China has significantly increased. Most devices (79%) were classified as Class II with moderate risk, whereas 21% were classified as Class III with high risk. Most devices (74.2%) were categorized as medical device software, and the most common application was medical image processing (63.2%). In terms of target body areas, devices related to the heart accounted for the highest proportion (12.8%), followed by those related to the lungs (11.3%) and brain (6.7%). Conclusion This study establishes a comprehensive database of medical devices developed by AIMD companies in China, enabling the public to gain a coherent understanding of their current development status.

Objective:Since 2016, China has successively implemented Accelerated Drug Marketing Registration Procedures (ADMRPs) for drugs, including Breakthrough Therapy Drug (BTD), Conditional Approval (CA), and Priority Review and Approval (PRA), which have played an important role in promoting the development and review of clinically urgently needed drugs. In this study, we focused on the antineoplastic and immunomodulating agents approved for marketing through ADMRPs, to provide a reference for promoting the formation of a stable and mature regulatory system for the review and approval of antineoplastic drugs and immunomodulating agents in China.Methods:Reviewed the National Medical Products Administration (NMPA) drug review reports for the years 2016–2022 and screened the antineoplastic and immunomodulating agents approved through ADMRPs. Then, with the help of the NMPA website and the Yaozhi Database, two researchers independently queried and entered the detailed information of the selected drugs, and checked with each other. The attribute classification and main characteristics of the drugs were then analyzed with descriptive statistics to obtain the trend of drug types, drug review and approval status, and timeliness.Results:A total of 206 antineoplastic and immunomodulating agents were approved for marketing through five accelerated marketing registration procedures (or procedure combinations), with the average review time shortened by about 81 days. Among them, imported drugs accounted for a larger proportion, the most drugs for treating non-small cell lung cancer and lymphoma, and the largest number of PD-1/PDL-1 inhibitors, but pediatric drugs and rare disease drugs accounted for a smaller proportion.Conclusion:ADMRPs can promote the accessibility of antineoplastic and immunomodulating agents in China and safeguard the life and health rights of more patients. Nevertheless, it is necessary to pay attention to the expansion of the types of indications for medicines and to increase the development of drugs that are urgently needed by a small number of patients.