Objective To analyze the off-label use of novel anti-tumor drugs, and conduct evidence-based pharmaceutical evidence grading and rationality analysis.
Methods The off-label use of new anti-tumor drugs in the medical records of discharged patients in the hematology department from October 2022 to December 2024 was collected, analyzed and evaluated using the DrugDex classification system of the Thomson Micromedex database.
Results A total of 15 new anti-tumor drugs in the hematology department were used off-label, all of which were off-indication drugs, involving 222 cases. Among them, 4.05% (9/222) of the off-label use cases were graded as level I, 44.14% (98/222) as level IIa, 50.90% (113/222) as level IIb and 0.90% (2/222) as uncertain. The rationality rate of the results of evidence-based medical evaluation and analysis was 99.09% (220/222).
Conclusions The vast majority of off-label use of novel anti-tumor drugs in the hematology department is supported by evidence-based pharmaceutical evidence, but the quality of individual evidence is low, and further management is needed to reduce potential risk.