Orphan drug lists in Europe and the USA
Orphan Drugs on the market
There is currently no website that provides a list of orphan drugs available for purchase on the market, whether in Europe or globally.
Orphan Drugs authorised to be marketed
In Europe:
The Enterprise DG (Directorate General) of the European Commission offers a list of all designated orphan drugs for the European market.
Only drugs with an entry in the columns (far right) "Trade name EU Centralised Nr." and "Authorisation Date" are authorised for market.
The others are still under development or awaiting authorisation.
Before a drug can be marketed and be available for the patients in a specific country in Europe, however, it may also require additional administrative clearance by the country's authorities (setting of price and public health insurance reimbursement tariffs, for example).
The Enterprise DG list does not provide in-depth details on the drug. However, it provides a trade name for the drug, which you can then use to find in-depth information about the product on the website of the EMEA (European Medicines Agency). The EMEA listing covers all drugs, not just orphan drugs. Information is available in all EU languages.
In America:
The U.S. Food and Drug Administration (FDA) website offers a list of orphan drugs that have received marketing approval in America.
Orphan Drugs waiting to come to market
(These drugs are labelled or "designated" as orphan drugs, but are not yet authorised for sale)
In Europe:
European Union registry of designated orphan drugs
For each drug, there is a "Summary of Opinion" that gives details about the drug.
A Summary describes:
- the name of the drug
- the chemical description of the drug (for some drugs)
- the disease it is intended to treat
- current methods of treatment for the disease
- estimated number of patients affected by the disease in Europe
- the expected action of the drug
- the developmental stage of the drug
- contact information for the pharmaceutical sponsor of the drug
- contact information for a European patient organisation concerned by the disease (if available)
In America:
The U.S. Food and Drug Administration (FDA) website offers a list of orphan drugs that have received orphan designation in America.
Authors: Julia Fitzgerald & Fabrizia Bignami
