OBJECTIVE: To construct a method of recognizing rare diseases drugs in drug clinical necessity assessment of drug selection in medical institutions. METHODS: With “rare diseases drugs”

“rare diseases” and “orphan drugs” as Chinese and English keywords

the laws

policies and interpretation documents published on relevant Chinese government websites（from the inception/establishment of the database to Feb. 2021） were retrieved. Related literature were collected from PubMed

Wanfang

CNKI

VIP databases. After screening and analyzing the retrieved literature and documents

the method for recognizing rare diseases drugs in drug selection of medical institutions was formed. RESULTS and CONCLUSIONS: Six policy documents and 1 literature were included after screening. This study has formed the method of judging the drugs to be selected as drugs for rare diseases by drugs with indications for rare diseases in the drug instructions

drugs for the treatment of rare diseases in the List of Clinically Urgent New Drugs from Overseas

and drugs for the treatment of rare diseases in the Guidelines of the Diagnosis and Treatment of Rare Diseases in China（2019 Edition）

domestic marketing of drugs of “new use of old drugs” for the treatment of rare diseases included in the Orphanet website

which can provide separate evaluation pathway and index for the evaluation of the clinical necessity of drugs for rare diseases in the Drug Selection Guidelines for Medical Institutions.