Posts tagged with pharma

Speaking of the Orphan Drug Market….

Posted Monday September 29, 2014 by Melissa

Sometimes I like to think that we’re making progress in the rare...

Sometimes I like to think that we’re making progress in the rare disease community. The “we” being the collective we  – the interconnected and interdependent rare disease community that involves...

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Melissa at 2011 MPS Family Conference

Media: Mucopolysaccharidosis and Other Rare Disease in the News

Posted Monday May 13, 2013 by Melissa

My friends Jill Wood (an MPS IIIC mom), the Leider family (with...

My friends Jill Wood (an MPS IIIC mom), the Leider family (with two MPS II boys) and I were incredibly blessed to be involved in a project by Lisa Jarvis,...

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Why I Fear the Bubble, Part 1: The Pricing of #RareDisease Drugs

Posted Saturday January 05, 2013 by Melissa

NPS Pharmaceuticals just this week priced their drug for short bowel syndrome,...

NPS Pharmaceuticals just this week priced their drug for short bowel syndrome, Gattex, at an annual cost of $295,000 per patient. This price may not raise too many eyebrows among...

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Orphan Drug Development Requires Unique Strategies

Posted Monday July 02, 2012 by Melissa

I recently read  a paper entitled Strategies for the Sustainable Development and...

I recently read  a paper entitled Strategies for the Sustainable Development and Delivery of Drugs for Rare Diseases by Kristen Cardinal and Chris Meier of the London Business School. The...

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What we want from (big) pharma

Posted Monday April 23, 2012 by Melissa

During my recent time at the World Orphan Drug Congress, I had...

During my recent time at the World Orphan Drug Congress, I had the pleasure of a nice dinner and conversation with some operations folks from a larger pharmaceutical company, otherwise...

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