The role of clinical trials in the sustainability of the Italian national health service cancer drug expenditure

OBJECTIVE: Clinical trials offer new and potentially more effective therapeutic options for cancer patients and a potential cost-saving opportunity, especially considering that trial drugs are provided free-of-charge. The aim of this study was to analyse drug-related cost savings in clinical trials in a cancer institute over a 3 year period. The cost savings relate to the pharmaceutical expenditure of our centre, IRCCS Istituto Romagnolo per lo Studio dei Tumori (IRST) “Dino Amadori”. METHODS: We conducted a retrospective analysis of patients taking part in interventional clinical cancer trials approved by a local independent Ethics Committee between 1 January 2018 and 31 December 2020. The standard of care (SOC) was identified as the standard treatment that would have been offered to a patient if he/she had not been enrolled in the study. The sum of SOC costs of all patients represents the potential cost avoidance during the study period. Results were stratified by year, trial promoter, trial phase and tumour type. The same approach was used to perform a secondary analysis of compassionate use programmes. RESULT: In the 3 year analysis, 1,257 patients were treated with experimental therapies in 244 clinical trials, of which 157 were profit and 87 academic. Results showed an overall cost savings of €13,266,518, more than 50% of which (€7,035,009) was related to phase III studies. Profit clinical trials generated €9,069,764 (68.4%) of the drug cost savings compared with €4,196,754 (31.6%) of academic studies. The stratification for tumour type was €3,552,592 (26.8%) genitourinary cancer, €3,268,074 (24.6%) melanoma, €2,574,127 (19.4%) haematological malignancies, €2,330,791 (17.6%) lung cancer, €728,149 (5.5%) gastrointestinal cancer, €557,608 (4.2%) rare tumours and €255,178 (1.9%) breast cancer. The secondary analysis on compassionate use included 122 patients involved in 28 different access programmes and revealed cost savings of €1,649,550. CONCLUSION: The results of our analysis point to the benefits of participating in and planning clinical trials for the public healthcare sector.