Orphan Drugs

(asked on 13th December 2018) - View Source

Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, whether he is taking steps to introduce a revised definition for ultra-orphan medicine.


Answered by
Jackie Doyle-Price Portrait
Jackie Doyle-Price
This question was answered on 20th December 2018

Currently, rare diseases are defined as conditions affecting no more than five in 10,000 people in the European Union. The new European Clinical Trial Regulation (Regulation (EU) No 536/2014) will introduce an ‘ultra-rare’ condition and states that clinical trials for the development of orphan medicinal products and those of medicines affecting no more than one person in 50,000 in the EU (ultra-rare diseases) should be fostered, and in such areas, a rapid yet in-depth assessment is of particular importance. The new clinical trials regulation is expected to be implemented in the EU in late 2020. The Government has confirmed that United Kingdom law will remain aligned with the parts of the new EU clinical trials Regulation that are within the UK’s control.

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