JOURNAL OF RARE DISEASES 2025;4(4):399-405

doi:10.12376/j.issn.2097-0501.2025.04.001

Current Status, Challenges, and Prospects of Clinical Research on Rare Tumors in China

Sai GE 1 ; Wenfei LI 2 ; Qi WANG 3 ; Jian LI 2 ; Xiaotian ZHANG 1 ; Lin SHEN 2

Affiliations

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Keywords

rare tumors; clinical research; new drug research and development; specialty construction

Country

China

Language

Chinese

Abstract

Although the incidence of each individual type of rare tumor is relatively low, their cumulative impact affects a vast patient population, and their survival prognosis is significantly worse than that of common tumors. In recent years, China has made remarkable progress in clinical research on rare tumors, with a rapid increase in the number of studies and a diversified exploration of treatment modalities. However, clinical research on rare tumors still faces numerous challenges, such as uneven distribution of medical resources, the absence of a foundational registration system, and insufficient motivation for original research and development. Looking ahead, key measures including specialized development, a national collaborative group model, full utilization of real-world data, application of novel clinical research designs, and artificial intelligence technologies will strongly drive comprehensive advancements in China′s rare tumor prevention and treatment system.