Tag Archives: Ultra Orphan

The Orphan Drug Act at 30 Years: What's Next?

30 years ago last week, the US Orphan Drug Act came into being, and with it, a door of possibilities opened up for patients with literally thousands of untreated diseases. The anniversary marks the beginning of a journey where medicines for rare diseases have gained a foothold in the portfolios of drug companies large and […]
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First Orphan Launch a Challenge for AstraZeneca

Four months after AstraZeneca received FDA approval for vandetanib, a treatment for inoperable medullary thyroid cancer, the company announced the drug’s trade name: Caprelsa. FDA said other proposed names – Zactima, for example – too closely resembled other currently marketed proprietary names. Getting FDA approval on a brand name for vandetanib, AstraZeneca’s very first orphan drug, […]
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AstraZeneca Signs Exclusive Distribution Deal for Vandetanib

As the number of drugs targeting small populations increases, managing numerous drug distribution channels and educating the relevant parties – specialty pharmacists, payers, physicians, and patients – becomes a daunting task. Specialty pharmacies that handle seven to 10 major disease states, after all, might only see three or four patients with a given rare disease, […]
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Linguistics: "Salami-slicing" Orphan Drugs

Ultra-orphan? It’s a phrase I never heard before, but increasingly, companies are taking it on and finding it a useful way to describe the very smallest patient groups. “Language is very important, and you have to understand how it affects perception,” says Maria Hardin, vice president of patient services for NORD (National Organization for Rare […]
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