Facts and figures of the compassionate use of onco-hematological drugs from 2016 to 2021: Results from the multicentric observational study Compass-O

Background: Compassionate drug use (CDU) is a topic of debate in the scientific community characterized by a lack of information, especially in the onco-hematology area. Aim: The Compass-O study aimed to provide updated data on CDU of onco-hematological drugs for the period 2016–2021. Method: Compass-O is a retrospective observational study with data obtained from 7 Italian Units for cytotoxic drug preparations (Unità Farmaci Antiblastici, UFA). Drugs and Therapeutic Indications (TI) were described separately and as drug-TI combinations, providing the mean Patients Per Year (mPPY) and the Years of Use (YoU). Results: A total of 783 requests of CDU were retrieved, referring to 156.3 mPPY (max: 272 in 2021; min: 84 in 2018). The 52.2% of subjects was female, and the 63.2% aged >60 years. A total of 709 (90.5%) CDUs referred to solid tumors and 74 (9.5%) to liquid tumors. CDU referred to 93 drug-TI combinations. The most recurrent drug-TIs were nivolumab-lung carcinoma (mPPY: 36.5; YoU: 2) for solid tumors, and azacytidine-acute myeloid leukemia (6.0; 1) for liquid tumors. The most frequent TIs were lung carcinoma (45.7; 6) and breast cancer (37.0; 4) for solid tumors, and chronic lymphocytic leukemia (5.0; 2) and multiple myeloma (4.7; 6) for liquid tumors. Conclusion: Compass-O provided an updated framework of the CDU in onco-hematology, showing its large-scale phenomenon and the relevance of UFA as a data source, underlining the need for a national observatory to monitor CDU.