Abstract
Most health economists agree that benchmarking annual treatment cost and cost-effectiveness of drugs for rare diseases (DRDs) versus non-DRD drugs is inappropriate. However, there is limited agreement regarding an acceptable level of premium for rarity to the annual cost per patient and incremental cost-effectiveness ratios (ICER) for the DRDs. This study aimed to evaluate whether annual drug costs and/or sponsor submitted ICERs for DRDs were correlated with epidemiologic factors, such as patient population size, and availability of treatment comparators using information from the Canadian Agency for Drugs and Technologies for Health (CADTH) reimbursement reviews database.
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