TABLE 1 -.

Adapted multicriteria decision analysis value framework specifically developed for the evaluation of orphan drugs in Spain (10)

Quantitative criteria
Domain – Impact of the disease: • Disease severity • Unmet needs
Domain – Results observed in: • Efficacy/effectiveness • Safety/tolerability • Quality of life (patient-reported outcomes)
Domain – Type of benefit: • Therapeutic impact
Domain – Economic consequences: • Cost of intervention • Other medical costs • Non-medical (indirect) costs
Domain – Understanding the intervention: • Quality of evidence
Contextual/Qualitative criteria
Domain – Regulatory context: • Mandate and scope of healthcare system and population priorities and access • Common goal and specific interests
Domain – Feasibility: • System capacity and appropriate use of intervention