Regulatory Focus™ > News Articles > Orphan Medicines in the EU: A 15-Year Review

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Posted 29 January 2016 | By Zachary Brennan 

Orphan Medicines in the EU: A 15-Year Review


With the 15th anniversary of the adoption of the Orphan Regulation in the EU, the European Commission notes in a new report that this landmark regulation has incentivized the development, approval and marketing of more than 100 rare disease treatments, or orphan drugs.

According to the European Commission’s fourth inventory of orphan medicines, released Friday, somewhere between 5,000 and 8,000 different rare diseases exist in the EU, impacting between 27 million and 36 million people.

From 2000 to September 2015, the Europeans Medicines Agency (EMA) received 2,302 applications for orphan designation, of which the commission approved 1,544. Currently 1,227 orphan designations are active, as some decisions have expired and some products have been withdrawn by the sponsor.

And though that sounds like a lot of designations in a short period, the commission notes that just 1% of all rare diseases in the region are currently covered by authorized drugs.

About a third of all orphan applications deal with some form of cancer, and the most frequently designated rare conditions include acute myeloid leukemia, cystic fibrosis, glioma, pancreatic carcinoma ovarian cancer, multiple myeloma, chronic lymphoblastic leukemia and hepatocellular carcinoma.

Beginning in 2005, sponsors were required to use the centralized marketing authorization procedure for all orphan products (essentially meaning that a single marketing authorization can be valid in all EU countries), and the commission has to date authorized 117 orphan medicines, 82% of which are new active substances. Before that change, 24 designated orphan products received marketing authorization (22 by centralized procedure and two by national procedures).


The uptick in approvals and interest in orphan drugs is largely thanks to incentives provided by the regulation to industry, particularly the extended marketing exclusivity.

“The most important incentive in the Regulation is 10-year market exclusivity for designated OMPs [orphan medicinal products],” the commission said. “The protection thus granted prevents the Union or a Member State from subsequently issuing or varying a marketing authorisation for a similar product (e.g. the same active substance) and for the same indication.” 

And since nearly 25 orphan marketing authorizations were granted to small- and medium-sized companies (SMEs), the commission also acknowledges that fee waivers for regulatory procedures have played a role in whetting industry’s appetite for

rare disease R&D.

EMA receives a special annual contribution from the EU budget to waive fees in part or in full for some smaller companies and total reductions granted to date amount to €78.4 million ($85 million).

In addition, 951 protocol assistance procedures were completed, of which 264 involved SMEs, over the past 15 years.

Individual member states also have adopted ways to encourage the development of orphan treatments. The commission noted that in Greece, orphan drugs are covered by a compassionate-use program where individual patients are reimbursed in full.

In the Netherlands, registration fees can be waived if the orphan drug is already registered in another Member State and the prevalence of the condition is less than 1:150,000.

And in France, orphan developers are exempt from certain taxes paid by pharmaceutical companies. France’s pricing committee also has made a deal where it will allow “a high price for a medicinal product, but the company must agree to restrict its annual sales to a certain limit and supply all patients who are eligible for the treatment,” the commission noted.

Belgium and the Netherlands have launched a pilot project on the joint procurement of orphan products, and in Spain and Sweden there are specific scientific advice procedures to provide additional support for potential orphan products.

And since many companies look to introduce orphan products in multiple markets at the same time, the EU’s Committee for Orphan Medicinal Products has developed an international liaison on such products with medicines agencies in North America and Japan. The committee also holds a monthly teleconference with the US Food and Drug Administration.

Inventory of Union and Member State incentives to support research into, and the development and availability of, orphan mecidinal products — state of play 2015


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