Research Reports | Market Assessment
October 2015

U.S. Payer Perspectives on Reimbursement for Orphan Drugs | Niche and Rare Pharmacor | G7 | 2015

By definition, rare diseases affect relatively few individual patients, but as a group they encompass up to 8,000 recognized rare diseases affecting approximately 10% of the U.S. population. The general lack of effective therapies and consequent low threshold for new product approval, engaged physicians and patient communities, as well as regulatory incentives, including expanded market exclusivity for companies investing in orphan diseases, all underscore the substantial commercial opportunities in this space. Historically, payers have tolerated high price points for orphan disease therapies owing to low patient numbers and very high unmet need. However, increased scrutiny of high prices for specialty drugs in the U.S. could be driving payers to impose onerous reimbursement restrictions just as a wave of innovative disease-modifying treatment options for debilitating rare diseases are reaching the market.

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