Tag Archives: Orphan Drugs

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

By Clark Herman | Published: January 8, 2013
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

By Ben Comer | Published: July 20, 2012
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

By Ben Comer | Published: August 2, 2011
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, isn’t [...]
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Creating Hope For Orphan Diseases

By Jennifer Ringler | Published: June 8, 2011
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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AstraZeneca Signs Exclusive Distribution Deal for Vandetanib

By Ben Comer | Published: April 27, 2011
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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