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Table 1 MCDA EVIDEM framework version 4.0 adapted to evaluation of medicines indicated for rare diseases

From: Determining the value contribution of selexipag for the treatment of pulmonary arterial hypertension (PAH) in Spain using reflective multi-criteria decision analysis (MCDA)

QUANTITATIVE CRITERIA: MCDA Core Model

DOMAIN: Disease needs

 Disease severity

 Unmet needs

DOMAIN: Comparative outcomes of interventions (selexipag vs iloprost)

 Comparative efficacy/effectiveness

 Comparative safety/tolerability

 Comparative patient-perceived health/patient reported outcomes (PRO)

DOMAIN: Type of benefit provided by selexipag

 Type of preventive benefit

 Type of therapeutic benefit

DOMAIN: Comparative economic consequences of interventions (selexipag vs iloprost)

 Comparative cost of intervention

 Comparative other medical costs

 Comparative other non-medical costs

DOMAIN: Knowledge about selexipag

 Quality of evidence

 Expert consensus/clinical practice guidelines (CPG)

CONTEXTUAL CRITERIA: MCDA Contextual Tool

Population priorities and access

Common goals and specific interests

System capacity and appropriate use of intervention

Opportunity costs and affordability