How Can Pharmacists Support Patients Being Treated with Orphan Drugs?

Gordon Vanscoy, PharmD, MBA, explains the challenges patients face when being treated with orphan drugs, and how pharmacists can help overcome these challenges.


Orphan drugs present a unique set of challenges to society, because there’s such small populations. An orphan drug is less than a population of 200,000, an ultra-orphan drug is less than 2,000 people. These small populations, in order to get them onto the market takes a lot of investment, and obviously they become very expensive products. So, once they are released it’s our challenge to not only get the drug and provide care to these people, but find them finical resources so they can actually afford it. It can be a tremendous burden for these patients and that’s a big responsibility for us to point them in the right direction.

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