Traditional Chinese medicine research on rare diseases: Publication trends and future perspectives

Traditional Chinese medicine (TCM) has increasingly attracted the attention of researchers as a potential complementary or supportive approach for selected rare diseases, but the characteristics and evidentiary strength of this literature remain unclear. This Correspondence summarizes publication patterns identified for the 121 conditions in the First Chinese Rare Disease List using Chinese and English sources searched from database inception through June 30, 2021. Fifty-five rare diseases were identified in 3,030 TCM-related publications, though the ten most frequently studied conditions accounted for more than 80% of the literature. Generalized myasthenia gravis, idiopathic pulmonary fibrosis, and multiple sclerosis together accounted for nearly half of all publications. Clinical studies and case reports or experience summaries predominated, whereas experimental studies represented only 9.03%. Oral herbal formulas were the most frequently reported intervention, and more than 95% of publications originated from mainland China. These patterns indicate growing interest but a fragmented, geographically concentrated, and methodologically limited evidence base; publication volume should not be interpreted as evidence of efficacy or safety. Future research should prioritize mechanistic studies, standardized and innovative clinical designs, rigorous safety and quality-control procedures, real-world evidence, patient-centered outcomes, and international collaboration to clarify the potential roles and limitations of TCM in rare disease management.