Abstract
e18840 Background: 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 8 CUP in Italy carried out from May 2015 to December 2020 in the hospitals’ perspective. These programs concern five cancer medicines (alectinib, atezolizumab, cobimetinib, polatuzumab vedotin, trastuzumab emtansine TDM-1), for a total of 8 programs. Methods: 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. Results: 1635 cancer patients were treated in eight CUPs. The SoC mean cost per patient ranges from €13355 to €28098 for all cancer drugs. The total cost of the SoC ranges from €21.8 - €45.9 million. The mean cost per patient covered by hospitals hosting CUP was equal to €2732 for cancer drugs, with a total cost of €4.5 million. The net economic benefit ranges from €17.4 million to €41.5 million for cancer treatments. Conclusions: Despite its limitations this paper illustrates for the first time the net economic impact of CUP in oncology patients 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.
Highlights
Compassionate use programs (CUP) for medicines respond to the ethical imperative of providing earlier access to medicines to patients not recruited in trials
The net economic benefit includes the avoided costs from the Standard of Care (SoC) the patients would have received if they had not joined the CUP, and costs not covered by the pharmaceutical industry but instead sustained by payers, such as those associated to adverse events, and costs for combination therapies and diagnostic procedures not used with the SoC
Despite research limitations, this paper illustrates for the first time the net economic impact of CUP in oncology patients from a payer perspective
Summary
Compassionate use programs (CUP) for medicines respond to the ethical imperative of providing earlier access to medicines to patients not recruited in trials. In 2017, a new decree from the Ministry of Health came into effect [5] reflecting the above EU regulations, while authorising, as per previously, the use in CUP of offlabel licensed products and labelled products not yet licensed in Italy By way of this decree, the definition encompasses patients that can access unauthorized medicines, on a named basis as well as in a group program through their physician, who suffer from severe and rare diseases or life-threatening conditions, who have no other valid therapeutic alternatives, and who cannot be recruited in a CT or who had previously participated in a CT and demonstrated positive health outcomes, and can in this way be guaranteed continuity of care outside the CT. Authorization for conducting a CUP is based on evidence from ongoing Phase III CT, Phase II for life threatening or severe diseases, and Phase I for drugs for rare diseases or rare cancers, provided that Phase I results demonstrate efficacy and safety of the medicinal product at a given dosage and schedule of administration
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