C076 | AVOIDED DRUG COST BY ENROLLING PATIENTS WITH HEMATOLOGICAL DISEASES IN CLINICAL TRIALS: A SINGLE CENTER EXPERIENCE

Pharmaceutical expenditure is constantly growing, in parallel with the increasing incidence of cancers and drug development costs. Enrolling patients in clinical trials provides countless advantages for both patients and the healthcare system. The pharmacoeconomic point of view is usually the least to be considered, but its impact should not be underestimated. The aim of this retrospective study was to estimate the costs “avoided” by enrolling patients with hematological diseases in interventional clinical trials at a single institution. For each patient, the Standard of Care (SOC) therapy, that would have been administered outside the clinical trial, was identified based on international guidelines, local clinical practice and Italian eligibility. The sum of the costs for SOC drugs represents the potential pharmaceutical expenditure avoided. Patients who would have received treatment through compassionate use programs or best supportive care were excluded from the analysis. Between January 2020 and December 2024, 124 patients were enrolled in 37 interventional clinical trials, 23 profit and 14 academic. The majority were phase 3 trials (59%), the remaining phase 2. The pathological area that enrolled the largest number of patients was non Hodgkin lymphoma (NHL), with 74 patients enrolled. The total avoided drug expenditure for SOC therapy over 5 years was 754.556 € (Figure 1), with a mean of 173.663 € saved annually. Thanks to an academic clinical trial 13 patients with Acute Myeloid Leukemia received gemtuzumab ozogamicin, avoiding around € 270.000 of SOC therapy. Enrolling patients with NHL generated a total avoided pharmaceutical expenditure of € 254.546, with a mean of more than 50.000 €/year. This allowed us to avoid the costs of drugs such as polatuzumab vedotin (more than 70.000€ enrolling 2 patients), intravenous and subcutaneous rituximab, liposomal doxorubicin, bendamustine and lenalidomide. Our analysis demonstrates the significant economic benefit of enrolling patients with hematological diseases in clinical trials. Participation in clinical trials not only reduces the direct pharmaceutical load on the Hematology Department budget, but also allows securing grants, reimbursements, and payments, factors not assessed in this analysis.