Skip to content

Orphan Drugs: Cancer By The Numbers

April 11, 2013

Note: The Orphan Druganaut Blog is currently attending the World Orphan Drug Congress USA in Washington, D.C. We have decided to republish a popular 2013 Blog Post.

According to a 2011 Report “Orphan Drugs in Development For Rare Diseases”, from the Pharmaceutical Research and Manufacturers of America (, more than 33% of orphan drugs in development are rare cancer disease drugs in late stages of the pipeline. Some of the rare cancer disease drugs are for solid tumors of the pancreas and thyroid, blood cancer, melanoma, and others. This statistic is reinforced by the Thomas Reuters study that came out over the summer in 2012, that says that about 40% of orphan drugs are used to treat cancer, the largest rare disease category.

Another amazing set of statistics for orphan drugs and cancer (EALRP = Estimated Average Lifetime Revenue Potential) :

  1. 40% of the top orphan drugs are oncology drugs with an EALRP = $70 billion/per drug
  2. 60% of the top orphan drugs are non-oncology drugs with an EALRP = $41 billion/per drug.

Of the top 10 revenue generating orphan drugs, the following 4 drugs are for oncology :

  1. Rituximab
  2. Lenalidomide
  3. Imatinib Mesylate
  4. Bortezomib.

Imatinib Mesylate, a blockbuster oral-dose drug, is approved to treat 9 different cancers with more than $4 billion in worldwide sales during FY ending March 2012. Imatinib receives orphan drug exclusivity 4 times since 2001. Cancer orphan drugs usually end of being designated for 1 or more additional indications, thus increasing its potential market revenue.

Americans spent more than $23 billion in 2011 for cancer drugs, more than what was paid for prescriptions to treat anything else. Almost all of the 10 bestselling cancer drugs are targeted therapies – personalized medicine.

From IMS Health numbers, 7 of the 10 top-selling cancer drugs in U.S. sales in 2011 are orphan drugs :

  1. Rituxan – $3 billion
  2. Avastin – $2.66 billion
  3. Herceptin – $1.66 billion
  4. Gleevec – $1.51 billion
  5. Alimta – $1.04 billion
  6. Erbitux – $703.3 million
  7. Velcade – $692.7 million.

Please Note:Pascal Triangle Animated” by Hersfold on the English Wikipedia (Template:Bill) [Public domain] | Wikimedia Commons.

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

Leave a Comment

Leave a Reply

Fill in your details below or click an icon to log in: Logo

You are commenting using your account. Log Out /  Change )

Google+ photo

You are commenting using your Google+ account. Log Out /  Change )

Twitter picture

You are commenting using your Twitter account. Log Out /  Change )

Facebook photo

You are commenting using your Facebook account. Log Out /  Change )


Connecting to %s

%d bloggers like this: