The pediatric drug market has long faced challenges such as insufficient drug types, uncomfortable dosage forms and low research and development investment, which have severely hindered the fulfillment of children's health needs. In recent years, the Chinese government has issued a series of policies aimed at improving the research and development efficiency of pediatric drugs, optimizing the review and approval processes, and improving the accessibility of drugs in the market. Based on the policy background and the current situation of the pediatric drug market in China, this paper deeply analyzes the specific role of these policies in promoting the development of clinical trials, promoting review and approval and market access, and discusses their comprehensive impact on the satisfaction of children's health needs and the development of the drug market.

This paper explores the optimization strategies for cooperative production models in blood products enterprises. From the perspective of international regulation and corporate practice, it analyzes different models of collaborative production and their importance in improving production efficiency and addressing supply shortages. The current Chinese regulations impose more restrictions on contract manufacturing of blood products compared to the United States and the European Union. However, under specific circumstances, such as addressing blood product shortages, China has shown regulatory flexibility. Through case studies of three major international blood products companies, the paper demonstrates the flexibility of modular production site setups and intermediate product transfer schemes. Drawing from international regulation and corporate practices, this paper suggests exploring routine allocation of plasma cryoprecipitate and intermediate components, optimizing blood product portfolios within groups, and reserving plasma resources for accelerating the development of new blood products. These recommendations aim to enhance domestic blood product production efficiency and diversity to meet public health needs.

This paper conducts a visual analysis of the regulatory scientific policies of drug supervision between China and the United States, comparing the policy evolution process and the change in their focal points between China and the United States. The aim is to provide reference for the development of drug regulatory science policy in China. The policy documents related to drug regulatory science between China and the United States from the establishment of the database to 2023 were searched in the databases such as U.S. Food and Drug Administration and National Medical Products Administration. Gooseeker, AntConc and Excel were used to conduct text visualization research on the annual volume and keyword frequency of policy documents between China and the United States. The findings indicate that while there has been a rapid increase in the quantity of China's pharmaceutical regulatory science policies, some issues persist, such as the lack of official definition, the imperfect top-level policy design, the macro policy content in the frontier field, the late start of internationalization and the decline of attention to traditional Chinese medicine. To promote the scientific development of China's drug regulatory science policies, it is essential for China to clarify the official definition of drug regulatory science. Additionally, more policies focusing on the top-level design should be issued; greater emphasis should be placed  on more cutting-edge and comprehensive key areas; new paradigms should be constructed for the development of Chinese medicine regulatory science policy; and efforts towards accelerating the internationalization of China's drug regulatory science policies should be intensified.

This article delves into the characteristics of the European Medicines Agency (EMA) guidelines and their implications for China. Authers analyze the EMA's scientific, transparent, and collaborative regulatory model by exploring its development process, classification system, implementation transition period, and update mechanism. The EMA guidelines represent a comprehensive and multi-level framework that ensures alignment with current laws and global standards while effectively addressing risks associated with emergencies and technological advancements. Building on this foundation, this article proposes optimization suggestions for the guidelines of drug regulation in China, in order to further enhance the impact on the scientific rigor and credibility of drug regulation in China.

This study systematically sorts out the data on innovative drugs approved for marketing entry in China and the United States from 2018 to 2023. It compares the R&D trends and differences in terms of drug types, target distributions, technological roadmaps, therapeutic areas. The findings reveal the bottlenecks and driving forces behind  China's transition from “pursuant layout” to “original innovation” in its innovative pharmaceutical sector. Data show that the gap between China and the United States in the approval number of innovative drugs is narrowing year by year, while the comprehensiveness of technologies is increasing yearly. In the future, domestic innovative drugs may achieve breakthroughs in areas like radiopharmaceuticals, small nucleic acid drugs, and innovative biological products. The approval trends and innovation analysis data show that, in recent years, the state's investment in pharmaceutical research and development has been increasing continuously, which has cultivated a large number of pharmaceutical R&D and regulatory talents, promoted the breakthrough reform of regulatory science, and the improvement of industrial innovation capabilities in the whole chain. It is expected to promote the high-quality development of China's pharmaceutical industry in an all-round way by strengthening basic research, connecting the entire process of “clinical demand-basic research-commercialization”, developing new regulatory tools, and accelerating the development of new quality productive forces.

Large language models (LLMs) have gained increasing attention for their potential to facilitate scientific discoveries across various disciplines, significantly influencing the field of drug development. The types of LLMs related to drug development are diverse, including general-purpose LLMs, protein LLMs, and molecular LLMs. These models hold great promise for applications in target discovery and identification, protein structure and function prediction, drug molecule design and optimization, drug property prediction, drug repurposing and combination therapies, and automated experimentation. Currently, LLMs are in the early stages of rapid development. However, the challenge remains to effectively integrate these models into innovative drug research and development practices in order to fully realize their potential value over time.

The echinocandin class of antibiotics is widely recognized for its unique antibacterial mechanism, offering a good balance of safety and efficacy. These agents are widely used in the treatment of invasive fungal infections, and are considered as a new generation of antibiotics for this purpose. Compared with other antifungal drugs, echinocandins excert their fungicidal effect by inhibiting the synthesis of fungal cell walls, ensuring their efficacy while reducing their toxicity to humans. This review introduces the characteristics, mechanism of action, advantages and limitations of different types of antifungal drugs, and summarizes the antibacterial principle, resistance mechanism and clinical application of echinocandin antibiotics.

Chemotherapy is an important method for the treatment for colorectal cancer; however, the therapeutic effect of chemotherapy drugs remains unsatisfactory, accompanied by many adverse reactions and individual differences. Researchers have found that chemotherapy sensitizers are of great significance for colorectal cancer. These agents not only improve the therapeutic effect of chemotherapy drugs, but also reduce their adverse reactions. The combination of fluorouracil (5-FU) and levo-folic acid is an important treatment scheme for digestive tract tumor patients. 5-FU is a classic drug that needs to be used with chemosensitization, but the current effect is less than satisfactory. Levofolinic acid is a new sensitizer, a left-handed isomer of folinic acid, serving as the active ingredient (the right-handed isomer cannot be utilized in the body). Levofolinic acid includes both levofolinic acid sodium and levofolinic acid calcium. To better guide and standardize the application of a chemosensitizer (levofolinic acid) in digestive tract tumors, the Professional Committee of the Chinese Anti-Cancer Association for Tumor Whole Assessment has organized experts to integrate the evidence-based medicine and expert clinical practice experience of levofolinic acid, as a chemosensitizer, and jointly formulated this consensus.

The prognosis of patients with advanced cholangiocarcinoma (CCA) is exceedingly poor, and the efficacy of conventional chemotherapy remains limited. Therefore, there is an urgent need to explore new therapeutic strategies. Envafolimab recognized as the world's first subcutaneously administered programmed cell death ligand 1 (PD-L1) inhibitor, has shown promising antitumor activity and safety in the treatment of various solid tumors. This article reports a case of advanced biliary tract cancer with telomerase reverse transcriptase (TERT) mutation and multiple intrahepatic metastases, which achieved significant clinical benefit following treatment with envafolimab combined with capecitabine. Through this case analysis, we aim to provide further reference for clinical practice.

Imperatae rhizoma is a traditional medicinal and edible homologous Chinese medicinal material, with a long history of application, rich chemical composition and a wide range of pharmacological effects. Its main components include triterpenes, phenylpropanoids, organic acids, flavonoids, steroids, volatile substances, polysaccharides and other classes of chemical compositions. These components are recognized for their diverse effects, including hemostatic properties, antitumor activity, enhancement of kidney function, anti-inflammatory effects, anthelmintic action, antimicrobial properties, antioxidant capabilities as well as hypoglycemic and hypotensive effects. By summarizing the chemical constituents and pharmacological effects of Imperatae rhizoma, the predictive analysis of its quality markers (Q-Marker) showed that chlorogenic acid, arundoin, cylindrene, imperanene, p-coumaric acid, luteolinidin chloride, bifendate, 5-hydroxymethylfurfural, triterpene, and polysaccharides could be used as its potential Q-Marker. These Q-Markers can be used as reference for the improvement of the quality evaluation system of Imperatae rhizoma.

Objective: A qualitative and quantitative analysis method for perilla leaf and white perilla leaf was established using ultra-performance convergence chromatography (UPC²). Methods: The samples were extracted by methanol. The separations were performed on Waters Torus^TM DIOL (100 mm×3.0 mm，1.8 μm) and Waters Viridis HSS C₁₈ SB (100 mm×3.0 mm，1.7 μm) columns using CO₂ （100%） and CO₂ (A)-95% methanol containing 0.1% trifluoroacetic acid (B) (80∶20) as the mobile phase, respectively. The flow rate was 0.8 mL·min^－1, the column temperature was maintained at 43 ℃ and the system back pressure was 1 800 psi. The injection volume was 1 μL. Cluster analysis was conducted on the chromatograms of perilla leaf with both surfaces purple (PP), perilla leaf with upper surface green and the lower surface purple (GP), and white perilla leaf with both surfaces green (GG). And the contents of perillaldehyde and rosmarinic acid in these samples were determined. Results: Different characteristics were discovered in the chromatograms of PP, GP, and GG. Chemical composition of perilla leaf with both surfaces purple and white perilla leaf with both surfaces green showed greater difference. Perillaldehyde and rosmarinic acid had a good linear relationship within their respective ranges. Relative standard deviations for precision, repeatability and stability tests were all below 3%. The average recoveries were 98.20% and 97.82%, respectively. Conclusion: Using UPC² as single instrument, this method successfully achieved both qualitative and quantitative analyses of non-volatile and volatile components within 4 min through coordinating columns with different polarities. The method is fast, organic solvent saving, and environmental friendly. This study can provide scientific basis for the use and quality control of perilla leaves, as well as new idea and reference for the application of UPC² in analysis of traditional Chinese medicine.

Objective: Our research group previously utilized Descurainia sophia seeds (DS) as a model due to their characteristic sinking-descending properties. Through pharmacological studies in traditional Chinese medicine, we identified the oligosaccharide (Oli), flavonoid glycoside (FG), and fatty oil (FO) components as the active constituents of the herbal medicine. Building upon the holistic principles of traditional Chinese medicine, our study aimed to further investigate the metabolic regulation of DS and its sinking-descending components in an asthma model using a system biology-metabolomics approach, thereby providing a comprehensive scientific explanation for the sinking-descending properties of DS. Methods: Male SD rats were divided into the control group (CON), asthma model group (M), dexamethasone group (Dex, 0.075 mg·kg^－1), DS group (2.334 g of crude herb·kg^-1), DS-Oli group (30.48 mg·kg^－1), DS-FG group (6.75 mg·kg^－1), and DS-FO group (672.19 mg·kg^－1). The allergic asthma model was established by ovalbumin combined with aluminum hydroxide gel. The effect of DS and its sinking-descending components on lung tissue was observed by HE staining. Urine and serum samples were detected using ultra-high-performance liquid chromatography coupled with quadrupole time-of-flight mass spectrometry. Changes in the overall metabolic profile were distinguished using principal component analysis; potential differential metabolites were identified by orthogonal partial least squares discriminant analysis, and relevant metabolic pathways were enriched using MetaboAnalyst 6.0. Results: DS and its sinking-descending components effectively alleviated pulmonary inflammatory infiltration and reversed the perturbed urine and serum metabolic profiles toward the normal state. A total of 33 urine biomarkers and 40 serum biomarkers were identified. Eight significant metabolic pathways associated with asthma and administration were enriched, including histidine metabolism, tryptophan metabolism, arginine and proline metabolism, etc. Conclusion: DS and its sinking-descending components demonstrate significant anti-asthmatic effects by regulating metabolic pathways associated with inflammation and immunity. The study investigated the regulatory effects of sinking-descending components from DS on an asthma model through metabolomics. In conjunction with previous pharmacology research, the comprehensive experimental data provide valuable insights into the sinking-descending properties of DS.

Objective: To establish a high-performance liquid chromatography (HPLC) fingerprint and multi-index component content determination method for Zaoren Anshen Liquid, and to evaluate the differences of Zaoren Anshen Liquid between different manufacturers and different batches of the same manufacturer in combination with chemometrics. Methods: HPLC method was adopted with CAPCELL PAK C₁₈ column (250 mm×4.6 mm，5 μm) used with acetonitrile-0.1% phosphoric acid solution as the mobile phase for gradient elution. The flow rate was set at 1.0 mL·min^－1, and the injection volume was 10 μL. The detection wavelength was 320 nm and 250 nm, and the column temperature set at 30 ℃. The similarity evaluation was performed using the “TCM Chromatographic Fingerprint Similarity Evaluation  System” (2012 Edition). Combined with cluster analysis in SPSS 27.0 software and orthogonal partial least squares discriminant analysis in SIMCA 14.0 software, the results of sample content determination were analyzed, and the important projection values of analysis variables were used to screen the markers affecting its quality. By comparing with self-made simulated preparations, the sources of differences between different enterprises were explored. Results: The fingerprint of Zaoren Anshen Liquid was established, and 6 characteristic peaks were confirmed. The similarity of the fingerprint from 13 batches of Zaoren Anshen Liquid was above 0.85 except for the sample of A enterprise. The determination method of six characteristic components of spinosin, 6‴-feruloylspinosin, rosmarinic acid, salvianolic acid B, schisandrin and schisandrin B was investigated. Chemometric analysis showed that different batches of samples from the same production enterprise could be obviously clustered into one category, and differences appeared in Zaoren Anshen Liquid from different enterprises. The differential components of the samples were schisandrin and spinosin. Compared with the simulated preparation, it was inferred that the decrease of schisandrin B content was caused by the change of pH value during the decoction process. Conclusion: The established HPLC fingerprint and multi-index component determination method are accurate and simple. Combined with chemometric analysis, the quality of Zaoren Anshen Liquid can be comprehensively evaluated.

Objective: To conduct mining and analyzing of the adverse drug event (ADE) signals of ciltacabtagene autoleucel for its rational and safe clinical use. Methods: ADE data from the FAERS database were collected from Q1 2022 to Q2 2024 with ciltacabtagene autoleucel as primary suspect drug, and valid signals for ADEs were screened using the reporting odds ratio (ROR), proportional reporting ratio (PRR), muti-item Gamma Poisson shrinker (MGPS), and Bayesian confidence propagation neural network (BCPNN) methods. Results: A total of 1 364 ADE reports were retrieved with ciltacabtagene autoleucel as primary suspect drug, and the percentage of ADEs with serious outcomes (resulting in hospitalization or prolonged hospitalization, death, life-threatening, and disability) was 41.3%. A total of 556 preferred terms (PTs) were mined from the ADE reports involving 24 system organ class (SOCs), and after excluding signals that were not related to the adverse drug reactions, the PTs that met the four assays were collected. There were 68 positive signals, of which 14 ADEs were not included in the specification, including cytomegalovirus pneumonia, immune-mediated small bowel colitfis, large bowel perforation, squamous cell carcinoma of the skin, and basal cell carcinoma. The onset of adverse events was concentrated in the 1st month after the initiation of ciltacabtagene autoleucel treatment and occurred in 6.91% of patients after 1 year of treatment. Conclusion: In addition to paying close attention to adverse reactions documented in the insert, it is also important to be alert to the occurrence of potential adverse events when treating patients with relapsed refractory multiple myeloma with ciltacabtagene autoleucel, and long-term follow-up is recommended for patients using ciltacabtagene autoleucel.