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Table 3 Potential value drivers of ODs in CEE countries

From: Systematic review on the evaluation criteria of orphan medicines in Central and Eastern European countries

Disease-related factors Treatment-related factors Economic factors Societal factors
• Prevalence (rarity) of disease
• Severity of disease
• Identifiability of the patients of treatment
• Loss of QALYs without treatment
• Unmet medical need (i.e., availability of treatment alternatives)
• Clinical heterogeneity of the disease (i.e., subgroup of patients)
• Evidence of treatment efficacy or effectiveness
• Capacity to benefit from the treatment (i.e., magnitude of benefit)
• Treatment is curative or delays progression or alleviates symptoms (e.g., palliative care)
• Safety profile of treatment
• Innovative profile of treatment
• Manufacturing complexity
• Cost-effectiveness
• Budget impact
• Number of indications
• Potential for off-label use
• Societal impact of treatment (i.e., indirect costs on families and caregivers)
• Equity in access to treatment
• Legal considerations (i.e., patent status)