Abstract
BACKGROUND: A substantial unmet medical need exists for patients with Multiple Myeloma ineligible for autologous stem cell transplantation who relapse after first-line therapy with daratumumab, lenalidomide and dexamethasone (DRd). Three therapeutic options recommended for lenalidomide-refractory patients from EHA-ESMO guidelines are approved in Europe and reimbursed in Italy: carfilzomib and dexamethasone (Kd); pomalidomide, bortezomib and dexamethasone (PVd); selinexor, bortezomib and dexamethasone (SVd). This study aimed to identify key decision criteria and their relevance for assessing these alternatives from a multi-stakeholder perspective. METHODS: Following ISPOR good practices, we developed a multiple-criteria decision analysis framework using the Measuring Attractiveness by a Categorical-Based Evaluation Technique method. Preferences were elicited from multiple stakeholders, including hematologists, methodologists, decision-makers, and patient representatives. Decision criteria were identified through a targeted literature review, discussed in a multi-stakeholder workshop, and shortlisted with a pragmatic literature review to assess data availability for each alternative. RESULTS: The final multiple-criteria decision analysis framework comprised five main criteria: acquisition cost, efficacy, organizational impact, route of administration, and safety. Within the safety criterion, we considered six sub-criteria related to six adverse events: peripheral neuropathy, diarrhoea, nausea, fatigue, anaemia, and thrombocytopenia. Efficacy emerged as the most relevant criterion by most respondents, with a median weight of 38.1%, followed by the safety criterion (26.8% median weight), with peripheral neuropathy being the most relevant safety sub-criterion (34.9%). Based on elicited preferences, SVd was ranked as the most valuable therapy with a global score of 72, followed by PVd (44) and Kd (26), on account of its clinical efficacy. No significant differences in preferences were observed across different stakeholder groups. CONCLUSIONS: This study provides valuable insights into the post-DRd treatment landscape for Multiple Myeloma, supporting decision-making from an Italian multi-stakeholder perspective.