Considerations on the feasibility of using retrospective database in the study of rare diseases / 国际药学研究杂志

ABSTRACT

Retrospective database is one of the data sources of real-world study, which has the advantages of abundant sample size, large amount of information and multi-dimension, and can support many types of quantitative re-search. On the one hand, rare diseases have the characteristics of low prevalence and the pathogenesis is unclear some-times. On the other hand, the lack of epidemiological investigation of rare diseases in China poses a challenge in the de-velopment and economic evaluation of rare disease drugs. This paper briefly describes the characteristics of the retrospec-tive database, and takes the health administrative data and electronic medical record as examples. Then, we discuss the feasibility of using such data to carry out epidemiological research and economic evaluation of rare diseases. Furthermore, we clarify some specific key points worth mentioning in advance in terms of study designs and protocols. Lastly, we pro-vide a reference for the subsequent use of retrospective data in conducting practical research.