FDA Blog: The Orphan Drug Act: 30 Years and Still Going Strong!

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Commentary You have free access to this content Do we need authorized orphan drugs when compounded medications are available?

Summary

What is known and Objective: Orphan drugs are used to diagnose, prevent or treat a rare disease. This Commentary aims to present a number of case studies questioning the need for designating compounded medications with a long history of effective use, which is well-supported by published clinical evidence.

Comment: Prior to the market introduction of orphan drugs, medication compounding was done in our hospital pharmacy for several rare diseases. Examples include amifampridine for the treatment of Lambert–Eaton myasthenic syndrome (Firdapse^®), ibuprofen for the treatment of neonatal patent ductus arteriosus (Pedea^®) and zinc acetate for the treatment of Wilson’s disease (Wilzin^®). Several ‘non-orphan’ pharmaceutical products, used off-label for the treatment of rare diseases, that became orphan medicinal products include Hydrea^® for the treatment of sickle-cell syndrome (Siklos^®) and Viagra^® for the treatment of pulmonary arterial hypertension (Revatio^®).

What is new and Conclusion: In our opinion, as indicated by our examples, a better balance should be struck between the development of orphan drugs along the recently established regulatory pathways and the pragmatic use of pharmacy-compounded products and evidence-based off-label use of already available commercial products. Societal needs would be best met by focusing orphan drug development on rare diseases for which there is a high unmet medical need.

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K-V Pharmaceutical And Orphan Drug Makena: Legal Update (12/22/12) ITC Won't Investigate Complaint

Orphan Druganaut Blog has a summary of the events in the trade complaint that K-V Pharmaceutical filed in October 2012 with the US International Trade Commission (ITC). The complaint was an attempt to stop compounding pharmacies from importing 17a hydroxyprogesterone caproate (“HPC”) to unlawfully manufacture copies of orphan drug Makena. The US ITC that it will not investigate the complaint.ad
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Another FDA lawsuit over Exclusivity

FDA Law blog is reporting  that there is another  Orphan Drug Battle in that Depomed has Sued the FDA Over GRALISE Orphan Drug Exclusivity.  The complaint and post can be read here.

The Makena Story

Michelle Oxman has written a blog entry entitled, What Does the Makena Story Tell Us About the Orphan Drug Act?, (September 24, 2012).  The article deals with why prescription drugs are a large portion of health care spending.  The article does a good job of explain the  Orphan Drug Act (ODA) (P.L. 97-414)  and of discussing the K-V v. FDA decision.  To read this article click here.

New Article: Do we need authorized orphan drugs when compounded medications are available?

M. Dooms, Hilde Pince and Steven Simoens  have written a Commentary for the Journal of Clinical Pharmacy and Therapeutics.  The commentary is entitled, Do we need authorized orphan drugs when compounded medications are available?  It appears in the September 14, 2012 edition and can be viewed here