What is an orphan drug?
About 26 million people living in the European Union (EU) suffer from a rare disease. The European Medicines Agency (EMA) plays a central role in facilitating the development and authorisation of medicines for rare diseases, which are termed ‘orphan medicines’ in the medical world.
This is the status assigned to a medicine intended for use against a rare condition. The medicine must fulfil certain criteria for designation as an orphan medicine so that it can benefit from incentives such as protection from competition once on the market.
Can I be treated with a medicine for my rare disease as soon as it is given orphan designation?
No. Orphan designation does not itself permit the use of a medicine. Orphan designation simply signals that the medicine looks promising. There might be little or no proof that it works in patients. Only when the medicine is given its marketing authorisation can you be sure that the medicine has EMA’s confidence that it works and is acceptably safe.
How long does it take to get marketing authorisation?
Generally, the application for orphan designation comes quite early in the medicine’s development – the medicine is considered for marketing authorisation only when enough evidence of its effects has built up.
Can I get the drug via a ‘compassionate use’ programme?
Exceptionally, a doctor might consider using the medicine before it has its marketing authorisation, for example through a compassionate use program. When contemplating use of a medicine that does not have marketing authorisation, the patient and the doctor need to be fully convinced that there is no other suitable treatment option (including leaving the condition untreated). In some cases, it might be possible to enrol on a clinical trial set up to test the medicine’s effects on patients.
This information is from the European Medicines Agency (EMA). To read more visit their website