Tag Archives: Orphan Drugs

Orphan Drug Laws: A Pharmacist's Perspective

Orphan Drug Laws:  A Pharmacists’ Perspective Guest blogger Dr. Albert Wertheimer Professor and Director, Health Services Research Temple University Philadelphia PA Pharm Exec’s sister organization CBI hosted its annual Orphan Drug Innovation Summit in Philadelphia on July 17-18.  This is a topic that I have professional interest in, as well as a general curiosity.  I […]
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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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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 […]
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