Asian Journal Of Social Pharmacy

Abstract: Objective To study the related literatures on rare diseases and to provide references for economic evaluation of rare diseases. Methods Related literatures on rare diseases which were published in the Springer, Web of Science, China National Knowledge Infrastructure, Wan Fang Database and VIP Database from January 2006 to September 2016 were retrieved. Results and Conclusion A total of 101 articles were searched out and after screening, 53 articles were used for the analysis. Four aspects were classified for the research，including cost-effectiveness, economic evaluation, budget analysis and socioeconomic impact of rare diseases. In the field of rare diseases research, cost-effectiveness and rare economic evaluation were the research hotpots. The number of budget analysis and socioeconomic impact research was few. Therefore, researchers should have a comprehensive study on various aspects of rare diseases research so that they can provide references for rare diseases policy making in different countries.

Key words: rare diseases, orphan drugs, economic, cost