EC clarifies orphan drug regulation questions


Following a public conference in November 2015, the European Commission has released new information about the EU’s Orphan Regulation. Orphan medicinal products aim to create treatments for extremely rare diseases which may harm citizens of the EU. Since January 2000 when the Orphan Regulation began, there have been debates surrounding a number of issues:

1. Significant benefit

One of the issues spoken about was what it meant to have a ‘significant benefit’. In order to qualify for the orphan regulation, the disease or illness should affect less than five of 10,000 who reside in the EU. Likewise, this treatment should be the only option available and should not be compensated over other available medicines. It should also not be used to recompense other treatments should they be in short supply or used as a prevention of possible illnesses or diseases. The reasoning behind these claims is to reward the research and development of new, innovative treatments which may become available.

2. Usage for condition outside the EU

The Ebola outbreak of 2014/15 saw the debate come about when thousands died from the disease overseas. It has been argued that the orphan regulation treatment can be used to treat such rare diseases within the EU, due to the very low-risk factor affecting EU residents. Should there be no relevant treatment for sufferers of rare diseases, an awareness of a disaster occurring has been deliberated. Orphan regulation supports the developments of new medicines which can cure otherwise incurable diseases.

“A medicinal product intended to diagnose, prevent or treat a condition which affects a large number of people in certain non-EU countries, but which has a low prevalence or a prevalence of approximately zero in the EU, may be eligible for designation as an orphan medicinal product,” a source stated.

When marketing the medicinal product becomes authorized, all orphan designation criteria must be followed through and the significant benefit must be demonstrated through data analysis, rather than making inaccurate presumptions. Questions have risen about maintaining two products for similar indications, with statements claiming they may not remain in parallel.





Wilson Sonsini Goodrich & Rosati is the premier legal advisor to technology, life sciences, and other growth enterprises worldwide. In today’s fast-growing, highly regulated generic pharmaceutical market, companies require specialized legal guidance beyond the scope of general corporate and securities counsel. WSGR has an experienced team of experts in key practice areas, including intellectual property, litigation, antitrust, FDA/regulatory, technology transactions, exports and FCPA, trade secret, and trademark and copyrights. Learn more at www.wsgr.

About the Author

Related Posts

Leave a Reply