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.

The prevention and treatment of rare diseases is an important public health issue in China.Rare diseases are characterized by diverse types,complex conditions,and heavy burdened to patients,leading to a series of issues in the urgent demand in prevention and treatment of rare diseases in terms of individuals'health and the national responsibilities.Related legislation is an effective way to promote multi-disease preven-tion and treatment of rare diseases,and patients'protection in multi-level.We tease out and analyze the legal system on national and local level related to rare diseases,and recommend the coping strategies and give sug-gestions to improve and optimize the legal system for the prevention and treatment of rare diseases from the three dimensions of completeness,normalization and feasibility of the legislation,hoping to improve the competence of China's rare disease prevention and treatment and raise the level of protection for the patients.

OBJECTIVE To evaluate the cost -effectiveness of tislelizumab in the second -line treatment of advanced or metastatic esophageal squamous cell carcinoma （ESCC）in China .METHODS A three -state Markov model was constructed to assess the cost -effectiveness of tislelizumab versus chemotherapy in the second -line treatment of advanced or metastatic ESCC and programmed death receptor 1（PD-L1）positive patients . The cycle length of the model was 1 month，and the time horizon of the model was set as 10 years. The discount rate of cost and utility was 5%. One-way sensitivity analysis ，probability sensitivity analysis and scenario analysis were used to verify the robustness of the base -case analysis results . RESULTS The results of the base-case analysis showed that compared with chemotherapy ，the incremental cost -effectiveness ratio （ICER）of tislelizumab in the second-line treatment of advanced or metastatic ESCC and PD -L1-positive patients were 26 864.01 yuan/QALY and 37 510.07 yuan/QALY，respectively，which was much lower than 1 time per capita gross domestic product （GDP）in 2021（80 976 yuan）. Results of scenario analysis showed that the ICER was less than 1 times per capita GDP ，regardless of the chemotherapy regimens（paclitaxel，docetaxel or irinotecan ）used. With the extension of the simulation time limit ，the ICER of tirelizumab regimen gradually decreased ，and the reduction rate gradually E-mail：lishunping@sdu.edu.cn decreased，but they were all less than 1 time China ’s per capita GDP in 2021. The results of the one -way sensitivity analysis showed that the 3 parameters with the most significant impact on the ICER were progression -free survival of tislelizumab group ，price of tislelizumab ，and the proportion of patients receiving follow-up treatment in the tislelizumab group . The results of the probability sensitivity analysis showed that the probability of tislelizumab with cost -effectiveness in the treatment of advanced or metastatic ESCC patients and PD -L1-positive patients were 99.09% and 99.94%，respectively，when using 3 times per capita GDP as the willingness -to-pay threshold . CONCLUSIONS Tislelizumab has economic advantages over chemotherapy alone in the second -line treatment of advanced or metastatic ESCC patients.

Objective:To develop a model of stellate ganglion block (SGB) in mice and investigate the effect of SGB on cerebral cortical blood flow.Methods:Thirty clean-grade healthy male C57BL/6 mice, aged 8-9 weeks, weighing 23-27 g, were divided into 5 groups ( n=6 each) using a random number table method: control group (group C), left SGB group (group L), left normal saline group (group SL), right SGB group (group R) and right normal saline group (group SR). Group C received no intervention.SGB was performed with 0.25% ropivacaine 0.08 ml via percutaneous posterior approach in L and R groups, while the equal volume of normal saline 0.08 ml was given instead at the location of left and right stellate ganglion in SL and SR groups, respectively.The cerebral cortical blood flow was determined using laser speckle contrast imaging system before SGB (T 0) and at 10, 30, 60, 90 and 120 min after SGB (T 1-5). Results:Mice developed ptosis on the block side, indicating that the model of SGB was successfully developed in L and R groups.There was no significant difference in cerebral cortical blood flow at each time point among C, SL and SR groups ( P>0.05), and cerebral cortical blood flow on the block side decreased at T 1, began to increase at T 2, peaked at T 3, and decreased at T 5 which was still higher than that at T 0 in group L and group R ( P<0.01). Compared with C and SL groups, the left cerebral cortical blood flow was significantly decreased at T 1, 5 and increased at T 2-4 in group L ( P<0.01). Compared with C and SR groups, the right cerebral cortical blood flow was significantly decreased at T 1, 5 and increased at T 2-4 in group R ( P<0.01). There were no significant differences in cerebral cortical blood flow at each time point between group C and group SL and between group C and group RL ( P>0.05). Conclusions:The mouse model of SGB via percutaneous posterior approach is successfully developed.Unilateral SGB can affect cerebral cortical blood flow on the block side, which shows a transitory decrease followed by a sustained significant increase.

Objective:To investigate the clinical effect of using free bilateral anterolateral thigh flaps(ALTF) in series to repair large area soft tissue defects of forearm.Methods:The clinical data of 11 patients with large soft tissue defects of forearm admitted in the Department of Plastic(Repair and Reconstruction) Surgery, Lishui Hospital of Zhejiang University from March 2014 to December 2021 were retrospectively analyzed, including 8 males and 3 females. Aged 36 to 68 years old, with an average of 48 years old. VSD treatment was performed after debridement, and until fresh removed 3 to 5 days after the operation. Until the wound was fresh. The wound was repaired with free bilateral ALTF in series until fresh. The size of the forearm wound was 18 cm×15 cm-28 cm×13 cm. The cut area of a single flap was 10 cm×8 cm-20 cm×13 cm. The series of bilateral flaps: One of the flaps was used as the proximal flap, and its vascular pedicle was anastomosed with the arteries and veins of the recipient area. The other flap was used as the distal flap, and the arteries and veins between the 2 ALTFs on both sides were anastomosed. The vascular pedicle beyond the distal flap was ligated or anastomosed to the distal end of the ulnar artery or the distal end of the radial artery. The flap and the surrounding skin of the recipient area were sutured immediately. The donor sites of the flap was closed directly. Periodic and regular outpatient follow-up was performed after operation and the clinical efficacy was analyzed.Results:All the flaps successfully survived after the surgery. The postoperative follow-up lasted for 6-18 months, 12 months in average. The flaps survived well with good soft texture, without swelling, the capillary reaction time was normal, without surface ulceration, in rosy colour and restored protective sensations. The hand function of the affected limb recovered well. The wound at donor sites healed well without complications. At the last follow-up, the hand function of the affected limb was evaluated by the Evaluation Trial Standards of Upper Limb Partial Functional of Hand Surgery of Chinese Medical Association, the result was 7 in excellent and 4 in good. The patients were satisfied with the flap and the therapeutic effect.Conclusion:The free bilateral ALTF in series can be used to repair a wound surface with large area, and the donor site can be closed at the same time. It is an effective method to repair large soft tissue defect of forearm.

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 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.

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.

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.

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.