OBJECTIVE To review the current research progress on pharmacoeconomics evaluation related to spinal muscular atrophy （SMA）， in order to provide valuable insights for clinical treatment， screening and medical insurance payment decision- making. METHODS A computerized search was conducted across multiple databases including PubMed， Web of Science， Embase， Scopus， Cochrane Library， EBSCOhost， CNKI， VIP， CBM and Wanfang database as well as other important health technology assessment （HTA） websites， such as National Institute for Health and Care Research，International Society of Technology Assessment in Health Care， Agency for Healthcare Research and Quality， etc. The pharmacoeconomics evaluation studies related to SMA were collected from the inception to December 31st， 2023. The literature/reports were rigorously screened based on predefined inclusion and exclusion criteria by two researchers， and the essential information from the included literature/ reports was extracted using Excel 2019. The quality of the included literature/reports was evaluated by Consolidated Health Economic Evaluation Reporting Standards 2022. RESULTS Finally， 9 articles and 15 HTA reports were included， with overall good quality of literature， but poor quality of HTA reports. There were a total of 24 studies on the pharmacoeconomics evaluation of SMA， including treatment options such as nusinersen sodium， sovaprevir， risperidone， and best supportive therapy.The review results showed that nusinersen sodium was not cost-effective in the treatment of SMA； there was no consensus on the economic viability of treatment options such as risperidone and sovaprevir； newborn/prenatal screening combined withmedication therapy was cost-effective. CONCLUSIONS newborn/prenatal screening combined with SMA medication therapy demonstrates economic advantages. It is suggested to further investigate the cost-effectiveness of new SMA drugs and SMA screening in China， taking localization parameters and medical insurance prices into account， and gradually incorporate SMA screening into the scope of neonatal genetic disease detection， in order to alleviate the financial burden of patients’ families and healthcare systems.

Purpose To design PCR combined probes u-sing TaqMan technology to detect the expression of major driver genes in a variety of soft tissue sarcomas at one time,and to dis-cuss whether the combined probes can better assist clinicopatho-logical diagnosis based on histological features and FISH results.Methods Our research group designed 32 pairs of fusion gene probes related to soft tissue sarcoma based on TaqMan tech-nique,involving 10 types of sarcoma.The histopathological specimens of 70 patients with common fusion gene soft tissue sarcoma in our hospital were examined by fusion gene combina-tion,and the histopathological specimens of 30 patients with oth-er soft tissue sarcoma without fusion gene were set as controls.Individual common sarcoma types were analyzed with FISH probe detection.At the same time,the detection performance of the combined probe was evaluated by various methods.Results The soft tissue sarcoma-related fusion gene probe designed by our research group was used to detect the confirmed soft tissue sarcomas,and the results showed that the highest sensitivity was 100％.Among the three types of tumors,protuberant dermatofi-brosarcoma,synovial sarcoma and mucinous liposarcoma were verified by FISH,and the coincidence rate of the two methods was high,with no statistical significance(P＞0.05).The re-sults of interlot and intralot reproducibility of protuberous derma-tofibrosarcoma,mucinous liposarcoma and synovial sarcoma were consistent.Three different concentration limits were used to de-tect the positive plasmid of all the fused gene RNA,and 25 cop-ies/μL was the lowest concentration limit.Conclusion Com-bined with the pathological diagnosis results,TaqMan technology can be used to design PCR combined probes for soft tissue sarco-ma,which have high sensitivity and high specificity and good methodological performance,and meet the needs of primary medical institutions for one-time and rapid auxiliary pathological diagnosis of common soft tissue sarcoma.It provides a rapid and reliable method for the detection of multiple fusion genes in clin-ical soft tissue sarcoma.

Pharmacoeconomic evaluation is the essential supporting information for the inclusion of rare disease drugs into medical insurance in China. The accurate measurement of the health state utility of rare diseas is of practical significance to the development of rare disease pharmacoeconomic evaluation. Based on the review of pharmacoeconomic evaluation requirements for rare diseases in some countries/regions, we systematically retrieved the published studies on the measurement of health state utility for 121 rare diseases in China and other countries and regions. We identified 17 591 papers in the initial review, and later selected 230 after screening. We also made a comprehensive analysis of the quality of literature, evaluation content and use of tools for measuring health state utility in rare diseases in China. Finally, we analyzed the challenges in measurement in terms of population, instruments use, and application of results and made recommendations based on analysis, hoping to provide reference for the development of rare disease health state utility measurement studies in China.

We have analyzed the current status of recognization and qualification of orphan drugs in China and abroad, looking at the aspects of the authority institutions, identification and qualification process, and the number of orphan drugs identified and available in the market. By comparing and analyzing horizontally the differences in orphan drugs identification between representative developed countries vs. some developing countries, we discuss the inadequacy of orphan drugs supervision in China. We introduce the advanced experience from the developed countries and some developing countries to provide suggestions for the identification and management of orphan drugs, hoping to speed up the process of development and market availability of orphan drugs and to maximize patient's accessibility to treatment in China.

Objective:To verify the reliability, accuracy and stability of the machine performance check (MPC) system of Varian′s TrueBeam series accelerator.Methods:After the installation was completed, the acceptance data were compared with the results of the first MPC operation to verify the reliability of the MPC results; the accuracy of the MPC results in the image-guided radiotherapy (IGRT) application was verified by the method of artificially introducing errors. The mechanical performance results of 60 groups of MPC from March 2019 to March 2020 were collected to calculate the mean value and standard deviation and compare the stability differences of each index.Results:The equipment error parameters obtained by MPC were all lower than those obtained by acceptance. The maximum difference between two sets of data was only 2mm and the threshold ratio was less than 20%. MPC had a high sensing accuracy for radiation and imaging position shift, and the r values were more than 90%. The vertical direction of the couch had a poor sense of displacement accuracy, and the r value was less than 73.7%. For this verification project, the angle of the treatment bed was the most stable, and the standard deviation did not exceed 0.01. The stability of other indexes did not significantly differ.Conclusions:MPC can be used as a verification tool for routine quality assurance of IGRT. The verification accuracy of radiation and imaging centers are better than the mechanical accuracy of gantry, collimator and couch.

OBJECTIVE：To provide reference for the clinical treatment and pharmacoeconomics research of type 2 diabetes patients. METHODS ：Using“Discrete choice ”“Discrete ranking ”as Chinese keywords ，“Discrete choice ”“Discrete ranking ” “Conjoint analysis ”“Diabetes mellitus ”“Type 2”“Type 2 diabetes mellitus ”“Non-insulin-dependent diabetes mellitus ”as English keywords，Chinese and English literatures were retrieved from domestic and foreign databases as CNKI ，Wanfang database ， PubMed，Web of Science during the inception to Dec. 2019. The application status of discrete choice experiment （DCE）was analyzed and summarized from the aspect of attributes and levels ，DCE choice sets ，DCE data quality ，sample size ，econometrics analysis and the preference results. RESULTS & CONCLUSIONS ：A total of 295 related literatures were retrieved ，involving 30 valid literatures. The attributes as drug administration ，glucose control and hypoglycemic events were included more frequently. D-efficient/ D-optimal and orthogonal experiment designs were used more frequently to create the DCE choice sets. DCE data quality could be checked by the internal validity tests. The rules of thumb was usually used to calculate the sample size. Conditional Logit model ，multinomial Logit model or mixed Logit model were used more frequently to analyze the preference data. ZH187） Compared with mild hypoglycemic events ，patients’treatment E-mail：19111020032@fudan.edu.cn choices were more likely to be affected by blood glucose control. However ， when hypoglycemic events occurred at：yychen@shmu.edu.cn night or the degree of hypoglycemia was serious ， the ·treatment preference of patients would change. Although most studies included the drug administration related attributes ，they were not major factors influencing patients ’treatment preferences and were closely related to patients ’previous medication history. DCE had been widely used in the study of type 2 diabetes in foreign countries ，but there were few relevant studies in China. The data quality of DCE was difficult to control. Although the trend of building complex econometric models was gradually rising ，most studies had not fully introduced the design details such as sample size determination method ，option set design principle and quality control option. In addition ，there were some deficiencies such as too many attributes ，too large or too small horizontal spacing. It is suggested that the ten criteria of related research in ISPOR report by Bridges JF and other soholars can be used for reference in DCE design to improve the rigor of design and ensure the credibility of preference results.

Objective:To observe the effect of mirror visual feedback (MVF) on the motor functioning and balance of stroke survivors, and to observe any morphological and structural changes in the tibialis anterior (TA) after MVF training.Methods:Thirty-six stroke survivors were divided into a mirror group ( n=18) and a control group ( n=18). Both groups received conventional drugs and routine rehabilitation treatment, while the mirror group was additionally provided with MVF training 5 days a week for 4 weeks. Before and after the treatment, both groups were evaluated using the Fugl-Meyer assessment for the lower extremities (FMA-LE) and the Berg balance scale (BBS). Moreover, the pennation angle (PA), the muscle thickness (MT) and the fascicle length (FL) of the tibialis anterior were measured at rest and in maximum isometric voluntary contraction (MVC) using ultrasonic imaging. Results:After the treatment the average PA of the affected side at rest and the MVC of the TA increased significantly in both groups compared with before the treatment, with the average improvement in the mirror group significantly greater than in the control group. The average TA thickness at rest and the MVC were both significantly greater in the mirror group than among the control group after the treatment. The average FMA-LE and BBS scores of both groups had improved significantly, but the mirror group′s average scores were significantly better than those of the control group.Conclusion:MVF can improve the motor function and balance ability of stroke survivors. Moreover, ultrasound can be used to evaluate the changes of TA morphology in such patients quantitatively, providing an objective foundation for tracking clinical efficacy.

Different levels of screening and treatment planning for many kinds of cancers were designed in many countries,but the screening rate was unreasonable.It reviewed the studies about analyzing stated preference for cancer screening by discrete choice experiments from January 2005 to July 2016.It introduced the background and method of DCE,identified the main factors affecting the attendance of cancer screening and provided theoretical foundation for further development of cancer screening in China.

Postoperative adjuvant chemotherapy for breast cancers was one of the ways to decrease the tumor recurrencerate of patients and improve the overall survival.It reviewed related articles about cost-utility analysis of postoperative adjuvant chemotherapy regimens for breast cancers before 2016,which could provide references for selecting economic and effective treatment for clinicalpractice.

Objective To determinate the heavy metal cadmium content in rice to ensure food safety.Methods Several brands and batch numbers of rice were collected and divided into groups A and B.Group A contained 24 pieces of rice from the canteens,and group B involved in 22 pieces from the farm product markets.Cadmium content in rice was detected quantitatively with X-ray fluorescence spectrometer,and then evaluated according to GB 2762-2012 which determined rice was not qualified in case cadmium content was more than 0.2 mg/kg.Results Group A had cadmium content between 0.00 and 0.477 mg/kg,the times of ultra standard being 2.385 and the disqualification rate being 29.2％ (7/24),and group B had cadmium content between 0.065 and 0.619 mg/kg,the times of ultra standard being 3.095 and the disqualification rate being 68.2％ (15/22).Excessive cadmium content in rice occurred in both canteens and markets,while the canteens was better than the markets.Conclusion X-ray fluorescence spectrometer detects cadmium content in rice rapidly and simply,and is worthy promoting in elementary facilities.