RESUMO
OBJECTIVE To provide reference for the access to medical insurance for rare diseases in China based on the existing access pathway and framework by analyzing the access policy of medical insurance for rare diseases in the United Kingdom （UK）. METHODS After collecting relevant guidelines and policy documents related to drug use for rare diseases in the UK， content analysis method was used to analyze the evaluation mechanism of drug use for rare diseases， reimbursement decision- making standards， stakeholder participation， coping strategies for dealing with uncertainties and risks， and policy implementation effects， and extract the key points of medical insurance access for drug use for rare diseases in the UK， to provide some suggestions for the establishment of medical insurance access system for rare diseases in China. RESULTS & CONCLUSIONS From the perspective of access， the UK had adopted a separate approach and clear criteria to assess and reimburse drugs for rare diseases. From the perspective of evaluation mechanism， multi-stakeholders such as doctors， patients and applicants participated in the decision-making process in the UK. The UK addressed uncertainty and risk by gathering better clinical evidence and using the patient access programme. After the implementation of the policy related to drug use for rare diseases， the UK had achieved remarkable results in terms of funding for drug use for rare diseases， the reimbursement rate of drug application， and the number of funded patients. It is suggested that in the process of establishing and improving the evaluation and reimbursement system for rare diseases drugs in China， the availability of rare diseases drugs should be improved by establishing a separate access assessment path for rare diseases drugs and involving more stakeholders.