Ulteriori riflessioni sulla determinazione del valore dei farmaci oncologici alla luce dell’approvazione dei nuovi farmaci immuno-terapici

Introduction Over the past few years economic stagnation has made the challenge for sustainability an even more complicated issue for public healthcare systems, also affecting pharmaceutical pricing. The objective of this study is to delve into the impact on decision-making of the adoption of different effectiveness measures, with a specific interest for the introduction of new drug treatments into the market – including a significant number of immunotherapies. Methods The study has examined molecules for first-line treatment of metastatic cancer, approved in Italy for public reimbursement over the last ten years. Through statistical regression, the analysis has researched the relation between treatment cost, clinical benefits, potential market size (expressed in terms of patients eligible for treatment) and the year in which reimbursement was approved. Clinical benefits have been expressed in terms of absolute and relative increase of average and median OS and PFS. Results Analysis has brought to light significant pricing differences, both related to the endpoint taken into consideration and to the measurement methods. The “confounding” effect of the market size seems limited. The variable inherent to the year of approval seems, on the contrary, significant. Discussion This analysis suggests that it is worthwhile carry out an in-depth reflection on the choice of the benefit measures considered in the negotiation process: especially considering the different capabilities of the measurements themselves in grasping different mechanisms of action of the drugs employed, that might induce different types of patterns in patient responsiveness. The impact of the chosen endpoint is very relevant for the “new generation” of immunotherapy treatments.