Full
Outline
Off-label drug use serves as the primary therapeutic approach for rare diseases，yet systematic evaluation of the risks of its associated adverse drug reactions (ADRs) is lacking. Conventional pharmacovigilance systems are often inadequate due to data sparsity，underreporting，and challenges in signal detection. Artificial intelligence (AI) enables the development of proactive，precise，and real-time ADR surveillance and early-warning frameworks. This review summarizes recent advances in applying AI for active surveillance of off-label ADRs in rare diseases. Key applications include identification of concealed patients，intelligent risk early warning based on multi-source data，and clinical decision support systems powered by AI，such as DrugGPT，“Xiehe-TaiChu” and “Nezha-LingTong”. Despite their promising prospects，significant challenges persist，including data scarcity and heterogeneity，limited model interpretability，privacy and ethical concerns，and the absence of a mature regulatory framework. Future efforts require multidisciplinary collaboration among clinicians，pharmacists，data scientists，and policymakers to establish a patient-centered，data-driven，trustworthy and controllable intelligent monitoring ecosystem for the medication safety of rare diseases.
62
10
| 科 Family | 属数 Number of genus | 种数 Number of species | 占总种数比例 Percentage of total species (%) | 属 Genus | 种数 Number of species | 占总种数比例 Percentage of total species (%) |
|---|---|---|---|---|---|---|
| 鹅膏菌科Amanitaceae | 2 | 11 | 5.26 | 鹅膏菌属 Amanita | 10 | 4.78 |
| 小菇科 Mycenaceae | 2 | 12 | 5.74 | 丝盖伞属 Inocybe | 5 | 2.39 |
| 多孔菌科 Polyporaceae | 8 | 14 | 6.70 | 蜡蘑属 Laccaria | 5 | 2.39 |
| 红菇科 Russulaceae | 3 | 23 | 11.00 | 小皮伞属 Marasmius | 6 | 2.87 |
| 小菇属 Mycena | 11 | 5.26 | ||||
| 光柄菇属 Pluteus | 5 | 2.39 | ||||
| 红菇属 Russula | 17 | 8.13 | ||||
| 栓菌属 Trametes | 5 | 2.39 |
关闭全屏
No. 86 Xueyuan South Road, Haidian District, Beijing
010-62662699(Operation and Technical Support)
qkjq@cast.org.cn
Copyright © 2025 China Association for Science and Technology. All rights reserved. For all open access content, the relevant licensing terms apply.
Sponsored by the Office of the Leading Group for Cybersecurity and Informatization of CAST, and supported by Science and Technology Review Publishing House
