OBJECTIVES

In 2018, China published its first official list of 121 rare diseases (RD), allowing the further health technology assessment (HTA) for RD in China. However, RD HTA remains challenging around the world and is particularly demanding in China where the HTA system has not been well constructed.

METHODS

We reviewed the latest policies of RD HTA adopted by 5 agencies: US (ICER group), England (NICE), Canada (CADTH), Australia (PBAC), and German (IQWiG). The major challenges in constructing HTA for RD in China were summarized.

RESULTS

In terms of the HTA processes for RD across different agencies, US and England have separate HTA processes for RD, while rest agencies adopt the general HTA processes with special concerns for RD. England and Germany use a special standard of evidence regarding RD. All countries except Canada grant a high cost-effectiveness or cost range for RD technologies. England and Australia have a financial-risk-sharing for technologies of high cost levels.

China faces the following challenges. Firstly, the HTA system for RD in China should be built nationally, either as a separate process or as a general process giving RD special concerns. Secondly, it is essential to recognize the low level of evidence in RD and to understand how to use them in HTA. Finally, it is critical to demonstrate and set up cost-effectiveness or cost in RD, as RD usually have a higher cost.

CONCLUSIONS

Despite the challenges, we believe that the release of the official list of RD is the first and important step regarding HTA for RD in China, and China will find its own adaptive ways of RD HTA.