Orphan drugs

Is there an upper limit on what should be spent on a single patient? – The case of orphan drugs. In the European Union, around 30 million people suffer from rare diseases. The EU defines a rare disease as a disease which affects less than one person in every 2,000 people. Because expected sales for drugs to treat rare diseases are small, there is little incentive for drug companies to develop new therapies to diagnose and treat such disorders.


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In 1999 the EU unanimously passed the Orphan Medicinal Products Regulation, to encourage industry to develop therapies for rare conditions by giving financial incentives. Orphan drugs are drugs developed for diagnosing and treating rare diseases. In the current system, drug candidates are designated as “orphan” and market authorised through a centralised European procedure. Availability and reimbursement remain a national responsibility. Although orphan drugs can improve health for millions of people, their high development costs, a small market and marketing exclusivity mean that tend to be very expensive for the patients and health care systems.

Page created: 21/02/2011
Page last updated: 07/11/2014
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