Pricing has never really been an issue for rare disease drugs, given the low exposure payers have to the therapies and the significant benefits usually offered. But with ultra-orphan price tags ticking higher and higher and drug pricing a simmering political controversy in the US, it is worth checking on the experience of recent launches as BioMarin Pharmaceutical Inc. introduces its Batten disease therapy Brineura (cerliponase alfa) at just over $700,000.
Few Clouds On High-Priced, Ultra-Orphan Drug Horizon
With BioMarin's Brineura hitting the market with a $702,000 annual price tag, recent experience for other high-priced rare disease drugs supports the theory that payers will accept high costs for ultra-rare pediatric disease therapies.
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