Table 1.

Proposed solution to handle higher ICERs of ODs

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, 34–36, 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, 92–94]
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]