Availability of orphan drugs in Europe
The granting of marketing approval does not mean the drug is available throughout all the European countries (list of marketed orphan drugs in Europe). The marketing approval holder must decide beforehand on its commercialisation status within every country and the drug will then go through numerous steps in each country in order to condition its management, and usually its price.
Drugs which are exclusively used in hospitals are, following positive mention by the Commission, registered on the list of admitted products for the community. Their price is free.
Despite joint efforts, the heterogeneous approaches among countries make patients access to orphan drugs more complex.
Early access to orphan drugs in Europe
Early access to a drug for patients may be possible before its marketing authorisation is granted to the pharmaceutical industry that develops it, most often during the third phase of the clinical trial and when its safety and efficacy are strongly assumed. Two cases may arise :
- an application for marketing approval has either been or is about to be submitted by the pharmaceutical company developing the drug in the country concerned. Then the company submits a temporary regulatory approval to the administrative authority for a group of patients (Temporary Use Autorization cohort in France and in Italy, or compassionate use authorization in the other European countries) that is valid for a limited time span in the country considered.
- or the physician asks the administrative authorities for a nominative temporary regulatory approval that is valid for a specific patient and for a limited time span in the considered country.