Supporting treatment decision-making for patients with multiple myeloma post-DRd in Italy: a multi-criteria decision...

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.

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.

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.

This study provides valuable insights into the post-DRd treatment landscape for Multiple Myeloma, supporting decision-making from an Italian multi-stakeholder perspective.