Challenges in the Economic Evaluation of Orphan Drugs
(2008; 2 pages)


Increasing pressures on health care budgets have led to a growing interest in the use of economic evaluation in reimbursement decisions for drugs and other health technologies. Although economic evaluation methods are becoming more established internationally, doubts have been raised about their use in drugs for rare diseases (often known as "orphan drugs"). This paper discusses the potential deviation between social value and cost effectiveness, the impact of rarity on the estimation of the cost-effectiveness ratio and the key questions surrounding the economics of orphan drugs.

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