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

Abstract

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.

Evaluation of drug cost savings related to clinical trials from the perspective of a university hospital

“…Again, the timeline was not the same and our site was not involved in many studies at the time of this work in onco-gynaecology (three clinical trials). For the other most recent studies,12–14 major cost savings were observed for melanoma, breast cancer and genitourinary cancer. The difference between repartition could be explain by the specific expertise of each centre.…”

“…These legal requirements could lead to a new opportunity for clinical trials, namely the potential for cost savings related to the coverage of ‘best standard of care’ (BSC) expenditure. The literature reports studies primarily focused on cost saving in oncology studies,5–14 with a variable follow-up period (1 to 18 years),5–14 with a variable number of patients (136 to 3195 patients)5–14 and of the trials involved (5 to 88 trials) 5–14. Finally, only a few studies take into account the avoided costs of all clinical trials15–17 and over a wider follow-up period (1 to 2 years).…”

Drug Cost Avoidance Resulting from Participation in Clinical Trials: A 10-Year Retrospective Analysis of Cancer Patients with Solid Tumors

“…Academic research has been considered a source of sustainability of public healthcare systems [25,26], with trial sponsors providing tangible benefits [27,28]. However, quantification of DCA in cancer patients treated in clinical trials has not been consistently documented, particularly due to deficient infrastructure information systems of hospitals, and because trial drugs provided by sponsors are not systematically included in pharmacoeconomic analyses of cancer medications [29].…”