RESUMO
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.
RESUMO
OBJECTIVE To systematically sort out and evaluate the health state utility of hemophiliac patients， and to provide reliable parameters for conducting pharmacoeconomic evaluation and health technology assessment. METHODS Retrieved from CNKI， Wanfang data， VIP， CBM， PubMed， Embase， the Cochrane Library， Scopus and Web of Science databases， relevant literature about the measurement of health state utility in hemophiliac patients was collected from the inception to February 2023. After screening literature， extracting data and evaluating the quality of literature， meta-analysis was performed for health state utility with Stata 15.1 software. RESULTS Thirty-eight papers were finally included， with the highest and lowest health utility values of 0.90 and 0.46， respectively. Those studies mostly adopted the EuroQol Five Dimensions Questionnaire （EQ-5D） （73.7%）. Results of meta-analysis showed that health state utility of global hemophiliac patients was 0.69，95% confidence interval was 0.65- 0.74； those of patients with mild， moderate and severe hemophilia were 0.79， 0.70， and 0.64， respectively； health state utility for patients with inhibitors （0.64） was lower than that of patients without inhibitors （0.69）； health state utility for the Chinese patient population was 0.55， which was higher than that of Iranian patients （0.46）， but lower than those of other developed countries. CONCLUSIONS There is some heterogeneity in the results of the studies across countries/regions， with higher health state utility in developed countries than in developing countries. As the severity of hemophilia increases， the trend of decreasing health state utility is obvious.
RESUMO
In 2018, hemophilia, one of the first ones, entered into the catalogue of Rare Disease in China. However, drug accessibility and affordability have been the challenges for hemophilia patients. Therefore, a comprehensive evaluation of the cost and efficacy of hemophilia drugs is essential to push forward the appropriate application of hemophilia drugs. In the article, we have teased out the existing process of the review and approval of hemophilia drugs; have examined the procedures for the drug entry into the medical insurance policy; have systematically searched the pharmacoeconomic evaluation studies of hemophilia in published literature in both China and overseas; and have comprehensively analyzed the quality, the content of evaluation, and challenges of pharmacoeconomic studies of hemophilia in China. At last, we discuss the prospect for the future based on the following three aspects: developing a special model of hemophilia, obtaining the health utility from various sources, and establishing the threshold of willingness to pay for rare diseases, hoping to provide reference and examples to the research into pharmacoeconomic evaluation of hemophilia in China.
RESUMO
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.
RESUMO
At present, the diagnosis, treatment, and social security of rare diseases have attracts social attention. As an important part of the multi-level health insurance system in China, commercial health insurance plays an increasingly important role in the supplement to basic medical insurance. Moreover, recently, the benefits of new healthcare reform policies have provided much room and great opportunities for the application of commercial health insurance for rare disease.This paper presents current medical insurance for patients with rare diseases and discuss the necessity and feasibility of bringing rare diseases into the coverage of commercial health insurance, and analyzes the current application and the potential problems of rare disease insurance in the commercial health insurance. This article also gives suggestions to the policies to make full use of the positive role of commercial insurance for the completion of multi-level health insurance system for rare disease.
RESUMO
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.
RESUMO
To solve the problems of long waiting time for outpatients, difficulty on implementation of one patient one room initiative, backward appointment method on examination, and imperfect instruction and guidance, a series of fine management measures have been taken place in a tertiary hospital which included dividing the patient waiting queue reasonably, establishing the patient secondary consultation system, improving the outpatient order of two-way management between doctors and patients, establishing one key appointment system for examinations, building hospital intelligent navigation system, and implementing outpatient payment and self-service drug delivery.The results showed, the waiting time of outpatients was significantly shortened ( P<0.001), and the satisfaction of patients in waiting time, hospital guidance and appointment registration was significantly improved ( P<0.05). Fine management mode combining online and offline can effectively increase the efficiency of outpatient service, improve clinical experience, and facilitate the high-quality development of medical services.
RESUMO
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.
RESUMO
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.
RESUMO
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.