ABSTRACT
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.
ABSTRACT
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.
ABSTRACT
OBJECTIVE To provide methodology re ference for conducting health state disutility estimation and calculating health output in pharmacoeconomic evaluation. METHODS Literatures about health state disutility estimation of malignant tumor drugs-related adverse events were retrieved from 3 Chinese databases such as CNKI ，Wanfang database and VIP and 3 English database such as PubMed ，Web of Science and the Cochrane Library from inception to July 2021. After 2 researchers independently screened the literature and extracted the data ，qualitative analysis was conducted. The modified decision-making technical support documents of the National Institute for Health and Care Excellence were used for quality evaluation. The practice and existing problems of health state disutility measurement were summarized to put forward some suggestions. RESULTS A total of 77 literatures were included. The first literature was published in 1991，and the number of publications increased in a fluctuating manner. 77 literatures involved 120 kinds of adverse events ，mainly utility studies （49，63.64％），only 3 （3.90％） were investigated in China. A total of 35 literatures（45.45％）had no statement of study design type ，48（62.34％）had no statement of health status development method ，52（67.53％）had no statement of health status verification method ，46（59.74％）set and explained the basic health status ，and 20（25.97％）were ranked for the health status . The utility measurement tools used were mainly the standard game method and the European five-dimensional health scale （23 literatures each ，accounting for 29.87％）. The research objects of 45 literatures（58.44％）were patients ，and 63（81.82％）did not specify the representative test of the population. Utility report types included negative value ，non-negative value and non-negative value of consolidated basic status. A total of 45 literatures（58.44％）had no statement of statistical method of utility. Overall quality score of included studies was 0.683. CONCLUSIONS The current disutility estimation include 4 key links of basic study design ， health status confirmation， utility estimation survey and survey data E-mail：luyuqiong96@foxmail.com statistics. The deficiencies of related studies are the lack of data based on the Chinese population ，the inconsistent type ofresult reporting ，and the incomplete report of measurement- related critical items. It is recommended to standardize the basic implementation path of disutility estimation ，formulate guidelines for health status utility value estimation ，and strengthen multidisciplinary cooperation so as to improve the quality of health status utility value estimation .
ABSTRACT
OBJECTIVE To explore the merging methods and influencing factors of health state disutility values estimation. METHODS Retrieved from 6 Chinese and English databases such as CNKI and PubMed ，the literatures about disutility values of diarrhea caused by antitumor drugs were collected from the inception to July 2021. After 2 researchers independently screened the literature，extracted the data ，assessed the quality ，the Meta-analysis and regression analysis were conducted using Stata 16.0 software. RESULTS Fifteen literatures were included. The results of Meta-analysis showed that diarrhea caused by antitumor drugs had a significant impact on health utility [ MD＝－0.26，95％CI（－0.30，－0.22），P＜0.05]. The results of subgroup analysis showed that the disutility values of the 3 types of negative value ，non-negative value changed into negative value ，and non-negative value combined with basic state changed into to negative value were MD ＝－0.14，95％CI（－0.19，－0.09）；MD＝－0.46，95％CI （－0.56，－0.36）；MD＝－0.12，95％CI（－0.20，－0.05），respectively. Meta regression results showed that the year of publication ， survey country/region ，severity of adverse events ，basic state settings ，utility estimation tools ，utility report types ，and utility statistical methods significantly affected the value of diarrhea disutility （P＜0.05）. CONCLUSIONS Disutility values for treatment-related symptoms or complications should be fully considered when inputting the parameters to the economic evaluation model. In the study or application of disutility values ，the types of utility reports should be distiguished ，and the core influencing factors such as the investigation country or region ，the severity of symptoms or complications ，and whether the basic state is set should be focus on.