Current Status of Studies Measuring Health State Utility for 121 Rare Diseases
10.12376/j.issn.2097-0501.2023.03.019
- VernacularTitle:121种罕见病健康效用值测量研究现况
- Author:
Junchao FENG
1
;
Shunping LI
1
;
Jingdan CHEN
2
,
3
;
Shiyao XIE
1
;
Yue ZHANG
1
;
Shijing JIANG
1
Author Information
1. Centre for Health Management and Policy Research, School of Public Health, Cheeloo College of Medicine, Shandong University, Key Lab of Health Economics and Policy Research of National Health Commission, Center for Health Preference Research, Shandong University, Jinan 250012, China.
2. Centre for Health Management and Policy Research, School of Public Health, Cheeloo College of Medicine, Shandong University, Key Lab of Health Economics and Policy Research of National Health Commission, Center for Health Preference Research, Shandong University, Jinan 250012, China.Peking Union Medical College Hospital, Chinese Academy of Medical Sciences &
3. Peking Union Medical College, Beijing 100730, China.
- Publication Type:Journal Article
- Keywords:
rare diseases;
China′s First List of Rare Diseases;
health state utility
- From:
JOURNAL OF RARE DISEASES
2023;2(3):455-462
- CountryChina
- Language:Chinese
-
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.
