EM2 Economic IMPACT of Compassionate Use of Medicines


      The economic impact of clinical trials in the perspective of trial sites has been already investigated. Instead, there is no evidence on the economic net benefit of compassionate use programs for medicines (CUP). This research aims to fill the information gap, investigating the economic consequences of ten CUPs in Italy carried out from May 2015 to April 2020 in the hospitals’ perspective. These programs concern five cancer medicines used in different disease settings and two drugs for neurological disorders


      Economic net benefit includes avoided costs for standard of care (SoC) the patient would have received if he/she has not joined the CUP and costs not covered by the pharmaceutical industry and sustained by the hospital hosting CUP. The latter include costs of adverse event (only severe sides effects generating hospitalisation and ascribed to medicines used in CUP), combination therapies and diagnostic procedures not covered by the sponsor. SoC costing relied on publicly available estimation. Adverse events and diagnostic procedures were retrieved from the CUP and monetized using the relevant fee for episode


      2246 patients were enrolled in the 10 CUP. The SoC mean cost per patient and the total cost ranged from €10743 - €18201 and €24.1 - €40.9 million respectively. The mean cost per patient covered by hospitals hosting CUP and was equal to €1803 (€4 million). The net economic benefit ranged from €20.1 to €36.9 million


      Despite its limitations this paper illustrates for the first time the net economic impact of CUP in the perspective of payers. Additional evaluations are ongoing to better understand the overall effects of CUP implementation, i.e. the economic value of the comparative benefit profile of medicines used in CUP versus the SoC, including potential effects on indirect costs