Patient access to orphan drugs

Abstract

ABSTRACTBackground: The Orphan Drug Act of 1983 has been a success in terms of spurring development of orphan disease treatments. However, rising orphan drug spending coupled with increasing numbers of orphan approvals have led to greater payer sensitivity with respect to reimbursement. Given the relatively high per-patient costs of many orphan drugs, reimbursement is widely regarded as the most important determinant of patient access.Methods: In this study, we examined patient access by analyzing payer reimbursement of orphan drugs approved in the U.S. from 2000 through 2016. Subsequently, we analyzed reimbursement by 20 leading payers of this subset of orphan approvals.Results: The median payer in our sample of 20 leading payers covered 93% of orphan approvals, which represented a decrease compared to previous studies.Conclusions: As per unit costs of orphan drugs and numbers of approvals increase, there is a trend towards imposition of more barriers to patient access. However, growth in U.S. orphan drug spending is expected to plateau. Accordingly, besides cost-effectiveness, payer formulary management considerations should encompass a broader concept of the value of orphan drugs, which would include the nature of the disease being targeted and whether the drug addresses an unmet need.