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.” | |
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 | |
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.) |