PrOACT-URL and MCDA for benefit-risk assessment in multiple myeloma: A case study

Introduction The objective of this study was to present the use of the Problem, Objectives, Alternatives, Consequences, Trade-offs, Uncertainty, Risk, and Linked decisions (PrOACT-URL) and multi-criteria decision analysis (MCDA) frameworks as a methodological innovation with the potential to support decision-making in the process of incorporating and monitoring technologies in the Brazilian Unified Health System (SUS). Methods The present is a case study that used these frameworks as a basis for the benefit-risk assessment (BRA) of chemotherapy treatment options for multiple myeloma (MM) in the context of a Brazilian public hospital. Results The application of the PrOACT-URL was not sufficient to guide stakeholders in making decisions about the best treatment alternative for MM, making it necessary to complement the qualitative analysis with the MCDA. In general, comparing the average scores for the five treatment options, the overall survival result was higher in four of the five treatments available for MM. The results that presented the lowest score were the risk of thrombosis and the risk of neuropathy. The sensitivity analysis showed that, for most stakeholder groups (managers, academia and clinicians), the treatment with the highest total value in the ranking was treatment with cyclophosphamide, thalidomide and dexamethasone (CTD). Conclusions The present case study showed that cyclophosphamide, thalidomide and dexamethasone presented the best benefit-risk balance for the treatment of MM and highlights the importance of the complementarity of these two structured approaches for more transparent decision-making, with an expansion of the deliberative process and the incorporation of preferences from different stakeholders.