Tag Archives: Orphan Drugs

Missed Opportunities in Rare Disease

At CBI’s 7th Annual Rare Disease and Orphan Drug Leadership Congress, speakers and attendees identified areas where pharma can improve the way it approaches rare disease and treatment.
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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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Creating Hope For Orphan Diseases

The Creating Hope Act of 2011, along with advancements in science and a shift in focus for Big Pharma, means that rare and pediatric orphan diseases may finally get the attention they deserve. “As a society, as we’re having this big debate around healthcare, everyone agrees that there’s a certain baseline of people that we […]
Posted in FDA, Gene therapy, healthcare, Regulatory | Also tagged , , | 1 Comment

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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Amid Big Pharma Woes, Shire in “Best Shape Ever”

Angus Russell, Shire’s longtime CFO—now CEO—likens the failing US automobile industry to pharma. “All the problems with the automobile industry were a long time coming,” he says. “It’s just that the SUVs and the relatively cheap price of gas created a bubble, just like a booming economy and blockbusters.” But now that the bubble has […]
Posted in Deals, Emerging Markets, R&D, Strategy | Also tagged , | 1 Comment
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