EMA Rare Disease Designations

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The European Medicines Agency (EMA) Rare Disease Designations dataset includes information on rare disease (orphan) designations based on applications that have been assessed by the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP).

Complexity

About 30 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 authorization of medicines for rare diseases, which are termed ‘orphan medicines’ in the medical world.
Developing medicines intended for small numbers of patients have a little commercial incentive under normal market conditions. Therefore, the EU offers a range of incentives to encourage the development of designated orphan medicines.
The Committee for Orphan Medicinal Products (COMP) is responsible for reviewing applications for orphan designation for medicines that are intended for the diagnosis, prevention or treatment of rare diseases.
An orphan designation allows a pharmaceutical company to benefit from incentives from the European Union to develop a medicine for a rare disease, such as reduced fees and protection from competition once the medicine is placed on the market.
Applications for orphan designation are examined by the COMP, which adopts an opinion that is forwarded to the European Commission. The European Commission then decides whether to grant an orphan designation for the medicine in question.

Date Created

2011

Last Modified

2018-04-21

Version

2018-04-21

Update Frequency

Irregular

Temporal Coverage

N/A

Spatial Coverage

European Economic Area Countries

Source

John Snow Labs => European Medicines Agency (EMA)

Source License URL

John Snow Labs Standard License

Source License Requirements

N/A

Source Citation

N/A

Keywords

European Medicines Agency, EMA Orphan, Rare Disease Designations

Other Titles

EMA Orphan Designations, European Medicines Agency Orphan Designations

Name Description Type Constraints
Active_SubstanceActive substance of medicinal productstringrequired : 1
Disease_ConditionConditions of the rare diseasestringrequired : 1
Date_Of_DecisionCOMP decision datedaterequired : 1
Decision_TypeCOMP decision typestringenum : Array required : 1
Medicine_NameOrphan drug namestring-
Active_SubstanceDisease_ConditionDate_Of_DecisionDecision_TypeMedicine_Name
SomatropinAIDS wasting2000-08-08Positive
GimatecanTreatment of glioma2003-12-01Positive
AtalurenTreatment of aniridia2015-10-09Positive
ArtesunateTreatment of malaria2007-02-19Positive
ArtesunateTreatment of malaria2007-12-06Positive
ArtesunateTreatment of malaria2012-12-06Positive
ArtesunateTreatment of malaria2015-07-28Positive
ChloroquineTreatment of glioma2014-11-19Positive
FlucytosineTreatment of glioma2018-02-22Positive
TalampanelTreatment of glioma2009-04-29Withdrawn