To evaluate the uncertainty of the determination of polysaccharide content in Haemophilus influenzae type b (Hib) conjugate vaccine by ultraviolet-visible (UV) spectrophotometry.

According to the General Rule 3421 of the Chinese Pharmacopoeia, the polysaccharide content in Haemophilus influenzae type b conjugate vaccine was determined. Based on the experimental process, the Evaluation and Representation of Measurement Uncertainty, and the Guidelines for the Evaluation of Uncertainty in Chemical Analysis, the uncertainties of the measurement results were systematically evaluated.

The sources of uncertainty mainly included the preparation of standard solution u(P), sample processing u(S), standard curve fitting u(C), measurement repeatability u(rep), UV spectrophotometry u(I), and method bias u(B). The combined standard uncertainty (u_c) was 1.109 0 μg·mL^-1, and the expanded uncertainty (U) was 2.218 0 μg·mL^-1 (coverage factor k=2, 95% confidence probability). [HJ]

The dominant contributors to measurement uncertainty were standard curve fitting, sample processing, and method bias. The fitting of the control standard curve significantly reduces measurement uncertainty, with this effect being particularly pronounced. The uncertainty introduced during sample processing primarily stems from pipette operations, which necessitate rigorous metrological verification. While standard curve preparation is time-consuming, optimized workflow efficiency can be achieved by employing pipettes for sample processing volumes of 1 mL or greater and utilizing micropipettes for standard curve preparation when the volumes are 1 mL or less. Method bias-related uncertainty requires strict compliance with standardized testing protocols, with particular attention to meticulous control of experimental parameters. Furthermore, maintaining appropriate laboratory temperature conditions, implementing regular instrument calibration, and performing systematic equipment maintenance all contribute substantially to uncertainty mitigation. This study can provide references for uncertainty assessment of drug quality control based on UV spectrophotometry.

To conduct a comprehensive analysis of proprotein convertase subtilisin kexin 9 (PCSK9) monoclonal antibodies based on a multi-dimensional evaluation method, providing a reference for clinical medication selection and drug selection in medical institutions.

Referring to the evaluation system in the Quick Guide for Drug Evaluation and Selection in Chinese Medical Institutions (the 2nd edition), with appropriate adjustments to some scoring criteria, a quantitative assessment was conducted on six PCSK9 monoclonal antibodies available in the domestic market from five aspects: pharmaceutical characteristics, efficacy, safety, economy, and other attributes. The recommendation grades were determined based on the scoring results.

The quantitative scoring results, ranked from high to low, were as follows: evolocumab (79.31 points), alirocumab (74.85 points), tolersimab (73.86 points), recaticimab (71.95 points), ongericimab (70.9 points), and ebronucimab (70.88 points).

All six PCSK9 monoclonal antibodies demonstrated good lipid-lowering efficacy. Among them, evolocumab, alirocumab, and tolersimab are strongly recommended, while the others are weakly recommended.

To analyze the efficacy and safety of sugemalimab as first-line treatment for non-small cell lung cancer (NSCLC) based on real-world data and provide guidance for its rational clinical application.

The clinical data of NSCLC patients hospitalized at a specialized cancer hospital from June 2022 to December 2024 and receiving first-line sugemalimab treatment were retrospectively collected. Statistical analyses were performed on efficacy, safety profiles, and influencing factors.

Among 81 NSCLC patients treated with sugemalimab, the median progression-free survival (PFS) was 13.6 months (10.6-NA, 95%CI), with an objective response rate (ORR) of 58.0% and a disease control rate (DCR) of 95.0%. Patients with lung adenocarcinoma exhibited longer PFS compared to those with squamous cell carcinoma (17.2 vs 11.3 months, P=0.036). The incidence of all-grade adverse drug reactions (ADRs) was 33.3%, with grade 3~4 ADRs occurring in 7.6% of patients. No grade 5 ADRs were observed. Myelosuppression was the most common ADR across all grades and severe cases. COX regression analysis identified pathological type, metastatic sites, and pre-treatment albumin levels as potential prognostic factors.

Sugemalimab demonstrates favorable efficacy and safety as a first-line treatment for NSCLC in real-world clinical practice.

To evaluate the economy of different doses of tirzepatide and semaglutide in the treatment of adult patients with type 2 diabetes mellitus (T2DM) in China, thus to provide a reference for medical insurance decision-making.

The Markov model was adopted to simulate the long-term health outputs and economic benefits of the four treatment regimens. Taking quality-adjusted life years (QALYs) as the indicator of health output and three times China's per capita GDP in 2024 as the willingness to pay threshold (WTP), the health output and medical costs of patients after 30 years of treatment with four treatment regimens were simulated. The incremental cost-effectiveness ratio (ICER) was used as the evaluation index to evaluate the economy of the four treatment regimens. The key assumptions were verified through univariate and probabilistic sensitivity analyses to ensure the robustness of the model.

Compared with the 1 mg semaglutide treatment regimen, the incremental costs of the 5, 10, 15 mg tirzepatide treatment regimens were 333 108.30, 699 163.50, 968 283.70 yuan, respectively; the incremental effects were 0.063 1, 0.140 5, and 0.165 5 QALYs respectively; the ICERs were 5 278 151.00, 4 977 303.00, and 5 852 089.70 yuan·QALY^-1, respectively. When the WTP was 287 247 yuan·QALY^-1, the 5, 10, 15 mg tirzepatide treatment regimens were not economically viable. The results of the sensitivity analysis showed that the results of the basic analysis were relatively robust. The results of the situation analysis showed that at the WTP of 287 247 yuan·QALY^-1, when the unit prices of 5, 10, 15 mg tirzepatide decrease by 68.4%, 79.6%, and 77.3% respectively, the probabilities of economic viability of tirzepatide compared with semaglutide were comparable.

For T2DM patients with poor blood glucose control treated with metformin monotherapy, compared with the 1 mg semaglutide regimen, the 5, 10, 15 mg tirzepatide treatment regimens have a lower probability of being economically viable at the current price. Appropriate price reduction can make tirzepatide become a safer, more effective, and economical treatment regimen.

To analyze the characteristics of rare disease drugs approved through the priority review and approval process in China from 2018 to 2024, and to provide a reference for optimizing review policies for rare disease drugs and improving drug access.

Based on data from the National Medical Products Administration and Yaozh Data (Drug Intelligence Data) and other sources, a descriptive statistical analysis was conducted on the main features of rare disease drugs approved through priority review and approval.

A total of 86 rare disease drugs were included in this study from 2018 to 2024, with an average review and approval duration of 463.55 days. Among them, chemical drugs accounted for 58%, imported drugs accounted for 77.9%, and domestic drugs only accounted for 22.1%. A total of 44 drugs were included in the national medical insurance catalog, and only 4 were pediatric-specific.

China has established a multi-pathway accelerated approval system for rare disease drugs, significantly improving the accessibility of rare disease drugs. However, there are still issues such as insufficient supply of domestic innovative drugs, uneven coverage of therapeutic areas, and a shortage of pediatric drugs.

To provide suggestions and references for strengthening the preparation and management of labeling for conditionally approved drugs in China and for enhancing their guiding value for rational clinical drug use.

A comprehensive approach integrating policy and regulation review, current situation and problem analysis, and international experience reference was adopted. We systematically reviewed the relevant policies and regulations on labeling for conditionally approved drugs in China, conducted an in-depth analysis of the core problems existing in the writing specifications, information updates and communiation efficiency of such labeling, and then formulated specific recommendations for improving the management of labeling for conditionally approved drugs in China by drawing on the mature experience of the United States and the European Union systems.

Through the above research, we identified the gaps in China's regulatory policy system and in the information update and communication mechanisms for labeling of conditionally approved drugs, as well as international experience that can be used for reference, and proposed systematic solutions covering dedicated technical specifications, update constraint mechanisms, information communication systems and digital regulatory platforms.

By improving the preparation and management mechanism of labeling for conditionally approved drugs, we can effectively address the evidence limitations of such drugs, enhance the guiding value of labeling for clinical drug use, provide strong support for ensuring patient medication safety and improving treatment effectiveness, promote the improvement of the lifecycle management level of conditionally approved drugs, and at the same time facilitate the deepening of the reform of China's drug review and approval system and the high-quality development of the pharmaceutical industry.

To conduct a comprehensive analysis of drugs marketed through the conditional approval process in China from 2020 to 2024, along with recommendations for improving this process.

Drugs conditionally approved during the study period were identified through the National Medical Products Administration public data, the Center for Drug Evaluation public data, and the Yaozhi Database. The basic information, pre-market clinical trial studies, and post-marketing study requirements and progress were collected and analyzed.

A total of 143 approved matters were marketed through the conditional approval process. Key characteristics included: R&D resources were highly concentrated on antineoplastic drugs, a high percentage of applications in pre-market pivotal clinical trials were based on surrogate endpoints, and the conversion rate of drugs from conditional approval to full approval was low.

It is necessary to strengthen the guidance provided by drug regulatory and relevant departments, establish a system for recognizing surrogate endpoints, and improve the flexibility of the conditional approval process.