Monday, March 30, 2015

An Overview Of Orphan Drugs

Intelligent Solutions in Pharmacy Benefits
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PBIRx's overview of orphan drugs
Generic drugs, brand name drugs, biosimilars, orphan drugs - with so many drug categories out there, it can be difficult to keep up with the definitions of all the categories of drugs and the recent trends involved. If you frequent our blog often, you may already be familiar with terms such as generic drugs and biosimilars because we have talked about them before. But on another note, orphan drugs may be something you are still unsure about and if you are a payer, remember that orphan drugs are often subject to Limited Distribution Pharmacy discounts, which are significantly lower than your other specialty drug discounts. Be sure to get a copy of your most current Specialty Drug list and associated discounts to INCLUDE Limited Distribution drugs and see for yourself. 

Today, we put together an overview of this drug category specifically to educate you! See below for a few facts about the industry, the drugs, and recent trends; but first, the definition:

Orphan drugs are pharmaceuticals that have been designed to treat a rare disease or condition. Through the Orphan Drug Act (ODA), drugs or biological products can be given this status so long as they meet criteria as outlined by the ODA and FDA.

Facts and Trends:
  • In 2014, orphan drugs cost patients approximately $137,782 per year. At the same time, non orphan drugs came in at a price of $20,875, on average.
  • By 2020, it is expected that orphan drugs will make up about 19% of prescription drug sales (not including generic drugs).
  • Phase III orphan drug development costs are half of non orphan development drugs. However, the amount of time it takes to develop such orphan drugs is not shorter than non orphan drugs.
  • As a result of the cancer drug Opdivo, Bristol-Myers Squibb is predicted to surpass Novartis as the leader in orphan drug sales in the year 2020.
For more information about orphan drug trends, please click here.

As one of Connecticut's leading pharmacy benefit auditing and consulting firms, we have made it our business to learn everything there is to know about orphan drugs and how your pharmacy benefit spend may be impacted by them. To learn how we can help you get more control over your healthcare costs, especially at a time where they are so high as demonstrated in the above list, please visit us at or contact us at (888) 797-2479 today.

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