OBJECTIVES: From a multi-stakeholder perspective, propose relevant criteria for the evaluation of rare disease treatments in Brazil.

METHODS: The MCDA exercise was developed based on the ISPOR GRP on MCDA and a literature review process to evaluate the already published MCDA frameworks for rare diseases and orphan drugs.

RESULTS: Sixteen publications were chosen from a pool of 52. For the development of the MCDA proposal, Brazilian laws, health system regulations, and the HTA official process were also considered. A proposal for the country was organized that could be used as a basis for the development of a multi-criteria analysis process for rare diseases in Brazil. This proposal established 5 domains(disease, comparative intervention results, therapeutic value, economic aspects, and health technology evaluation analysis) and correspondent criteria: Affected population; disease severity; Patient's social life impact; Economic load; Caregiver's QoL impact; Life expectancy; Morbidity; Vulnerable groups; Coverage of unmet medical needs; Comparative effectiveness; Comparative safety/tolerability; Comparative results perceived by the patient; Level of evidence; BIA: CEA/CUA; Logistics needs; HTA recommendations in other countries.

CONCLUSIONS: The establishment of a multi-criteria process would bring greater transparency and assertiveness to the evaluation processes of treatments for rare diseases, from the evaluation of its effect on patient and caregiver HRQL, the extent of its therapeutic benefit from efficacy, and the availability of other therapeutic options. Furthermore, the severity of the rare disease, as well as the potential of the treatment, should influence the decision.