Orphan drug status gets a lot of buzz in the pharmaceutical and biotechnology industries, but some of you may be wondering “what is it?” and “why should I care about it?”  So, here is some background information and a few key concepts.  The information here applies to the US Orphan Drug Program, but a similar program is available in the EU.

What is it?

The Orphan Drug Act (ODA) was signed into law on January 4, 1983.  It provides Sponsors with incentives to develop drugs for rare diseases.

What is considered a rare disease?

To be eligible for orphan drug status, a product must meet one of two criteria:

1. A product must treat a rare disease affecting fewer than 200,000 Americans.
   
2. A product may treat more than 200,000 Americans, but there is no reasonable expectation that the cost of developing and making the product available in the US will be recovered from sales.

A disease may be highly prevalent outside the US, so long as the US prevalence remains below the 200,000 threshold.  For example, using the above definition, Tuberculosis would be considered a rare disease because, even though it is common in developing countries, there are very few cases in the United States.

What incentives does orphan drug status provide?

Sponsors obtaining orphan drug status receive:

1. Tax incentives
2. Eligibility for clinical research grants
3. Waived or reduced user application fees
4. Additional two years of market exclusivity upon approval
5. Potentially faster approval
6. Protocol assistance from FDA

How common is orphan drug status?

• More than 350 products have received orphan drug designation
  
• There are more than 6,000 documented rare diseases (http://rarediseases.info.nih.gov)

What are some examples?

• Voraxaze (Glucarpidase) to treat toxic methotrexate plasma concentrations due to impaired renal function
• Jakafi (Ruxolitinib) to treat intermediate or high-risk myelofibrosis
• Erwinaze (Asparaginase Erwinia chrysanthemi) to treat lymphoblastic leukemia in patients with hypersensitivity to E.coli-derived asparaginase
• Ferripox (Deferiprone) to treat transfusional iron overload due to thalassemia syndromes when current chelation therapy is inadequate

If you are interested in learning more about orphan drug designation, check out our webinar “Targeted Product Development:  Personalized Medicine versus Orphan Product Development” on August 21^st (the webinar will be available on demand following the 21st).