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Table 1 Proposed solution to handle higher ICERs of ODs

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

Proposed Solution Description References
Weighted QALY “Weighted QALYs (according to disease prevalence, severity) attach a higher value to the health gain of a person with a rare disease. Therefore the ICER will decrease, increasing the likelihood of meeting the (standard) threshold.” [32, 3436, 62, 75, 76, 93, 94]
QALY categorization Prioritization of rare disease groups could be achieved by categorizing QALY’s based on e.g., disease states [75]
Higher CE-threshold for ODs Accepting a higher cost-effectiveness threshold for ODs increases the probability that these drugs will be cost-effective [36, 9294]
Special rules above the CE-threshold • Above the cost-effectiveness threshold special support funds or specific political decisions may be needed.
• Assess the profitability of ODs on different price levels (cost is warranted and based on a careful consideration of the manufacturer’s cost and returns on investment.)
[95] [47]