Objective: Compassionate Drug Use (CDU) allows patients with a specific disease and no further treatment option to access unauthorized treatments. In this study, we analyzed the requests of CDU approved by the Ethics Committee of Fondazione Policlinico Gemelli in the period January 1, 2018-June 30, 2021. We also estimated the economic impact of CUs.
Materials and methods: CDU requests were analyzed by year, by frequency and by regulatory status of the medicines requested. If an ex-factory price was available at the cutoff date of June 30, 2021, we estimated what would have been the costs for the National Health System (NHS) if the price was already negotiated at the time of CDU request.
Results: In the study period, 463 CDU requests were processed by the Ethics Committee. The number of requests increase linearly from 45 in 2018 to an estimated number of 260 in 2021. The requests included 68 medicines or combinations of medicines; 16 products out of 68 accounted for 75% of all requests. For 7 of these 16 highly requested treatments, accounting for 110 requests out of 463, it was possible to estimate the costs of therapies according to their ex-factory prices. If these products were to be purchased by the NHS, the estimated cost was € 5.472.225.
Conclusions: The access to unauthorized drugs through CDUs is undergoing a huge increase in the last few years. Such increase meets the ethical need to provide patients with the most recent, often innovative, therapeutic options.