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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Orphan Drug Pricing Heading To Negotiating Table in US?
The Institute for Clinical and Economic Review is launching an initiative to adapt its value assessment approach to orphan and ultra-orphan drugs, including a possible consideration of what should be a fair return on investment for manufacturers.
Biogen’s Spinraza Needs Long-Term Confirmatory Data, Says EMA
The EMA may have quickly assessed the US biotech’s antisense spinal muscular atrophy therapy for Europe, but the product’s rapid clinical development means long-term data, and data on its effects in milder disease, or even cures, still need to be collected.
Will Physician Demand For Repatha Put Pressure On Payer Restrictions?
FOURIER outcomes data presented at the American College of Cardiology annual meeting fell short of expectations, but could increase physician demand for the PCSK9 inhibitor, making it harder for payers to say no.