A great and much needed step forward for Batten disease, orphan drug designation by the European Medicines Authority, provides incentives for companies to conduct research into rare diseases lets hope accessible clinical trials will follow:
To qualify for orphan designation, a medicine must meet the following criteria:
• it must be intended for the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating;
• the prevalence of the condition in the EU must not be more than 5 in 10,000 or it must be unlikely that marketing of the medicine would generate sufficient returns to justify the investment needed for its development;
• no satisfactory method of diagnosis, prevention or treatment of the condition concerned can be authorised, or, if such a method exists, the medicine must be of significant benefit to those affected by the condition.
