Limits on Use of Health Economic Assessments for Rare Diseases

Summary Funding of expensive treatments for rare (orphan)diseases is contentious. These agents fare poorlyon ‘efficiency’ or health economic measures, suchas the quality-adjusted life years, because of highcost and frequently poor gains in quality of lifeand survival. We show that cost-effectiveness as-sessments are flawed, and have only a limited roleto play in reimbursement decisions for orphan drugsand beyond. Introduction Should European Union (EU) governments accom-modate expensive treatments for rare, ‘orphan’ dis-eases within strained healthcare budgets? Thequestion is a tense one.The opportunity cost is substantial. Some arguethat for example £2.5 million spent on orphandrugs (say 15 patient-treatment-years at £160000annually) would pay for over 520 hip replace-ments. 1 It is argued that financing decisions shouldbe based at least in part on health economic/tech-nology assessments (HTAs) 1,2 on which orphandrugs fare poorly for reasons explained below andwould probably not be financed. For example theNetherlands has questioned whether treatment forFabry and Pompe diseases, costing up toE200000 and E700000 per patient per year, re-spectively, should be fully reimbursed.