NP-012 Cost effectiveness analysis and indirect comparison can support the price’ definition of a drug? The case of encorafenib and binimetinib in metastatic melanoma, the pharmacist’s point of view

Background and importance The hospital pharmacist (HP) plays a key role in accessing new drugs at both regional and hospital level. Aim and objectives The aim of this study is to propose a cost-effective approach, from the perspective of the HP, comparing the case of Encorafenib and Binimetinib, (EB) two BRAF metastatic melanoma treatments (soon to be commercialized), with similar competitor drugs. Materials and methods We developed a probabilistic model and a survey to quantify the economic impact of the price of these two new drugs. A cost-effectiveness model (in terms of OS) was developed using Montecarlo’s simulations comparing EB vs Dabrafenib-Trametenib (DT) vs Vemurafenib-Cobimetinib (VC): The comparison arm of the 3 drugs registration studies was the Vemurafenib as the results of the arms were similar (both PFS and OS). In order to estimate the potential cost of EB (currently in negotiation), a survey was conducted within a Regional University Hospital taking into account Clinicians, HPs and Economists point of view. Results The survey showed that EB could cost up to 18% more than DT, due to the 7.5-months of life gained (MoLG) more than the best competitor. The price emerged from the survey was used to conduct the cost-effectiveness analysis in order to estimate an incremental relationship between the alternatives. From the 10,000 simulations carried out considering a threshold value of € 5,000/MoLG emerged that EB was cost effective in 80% vs. DT showing an incremental ratio of € 4,239/MoLG; Furthermore, it was found to be cost effective in 83% vs.VC showing an incremental ratio of € 3,129/MoLG. Conclusion and relevance EB potential cost could be more than the alternatives, but with a negotiation price similar to the available alternatives, the NHS would benefit in terms of health (MoLG) without significant additional expense. Furthermore, when direct comparisons are not available, it is advisable to analyze possible strategies of indirect comparison such as the Network metaanalysis. The methodology used in the survey to investigate the HP’s willingness to pay could be a support tool that could be used from AIFA representatives to assess the perception of added value in the analysis of new therapeutic treatments.