Current Status of Studies Measuring Health State Utility for 121 Rare Diseases

FENG Junchao; LI Shunping; CHEN Jingdan; XIE Shiyao; ZHANG Yue; JIANG Shijing

doi:10.12376/j.issn.2097-0501.2023.03.019

Journal of Rare Diseases > 2023 > 2(3): 455-462. > DOI: 10.12376/j.issn.2097-0501.2023.03.019

FENG Junchao, LI Shunping, CHEN Jingdan, XIE Shiyao, ZHANG Yue, JIANG Shijing. Current Status of Studies Measuring Health State Utility for 121 Rare Diseases[J]. Journal of Rare Diseases, 2023, 2(3): 455-462. DOI: 10.12376/j.issn.2097-0501.2023.03.019

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Current Status of Studies Measuring Health State Utility for 121 Rare Diseases

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.
Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China

Funds:

National Natural Science Foundation of China 72174110

More Information

Corresponding author:
LI Shunping, E-mail: lishunping@sdu.edu.cn
Received Date: April 11, 2023
Accepted Date: May 15, 2023
Available Online: September 08, 2023

Graphical Abstract

Abstract

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.
- rare diseases,
- China′s First List of Rare Diseases,
- health state utility

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References (47)

References

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Current Status of Studies Measuring Health State Utility for 121 Rare Diseases