Table 3 Potential value drivers of ODs in CEE 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) |