Approval and


The approval and funding of orphan drugs is a process involving the European Commission, the EMA and the Ministry of Health. As a result, access to and funding of a treatment differs from one Member State to another. 

Who is in charge of granting the designation orphan designation to a medicine?

The European Medicines Agency (EMA) is responsible for the designation of orphan medicinal products.

Within the EMA, which scientific committee evaluates orphan drugs?

The Committee for Orphan Medicinal Products (COMP).

Who is responsible for the authorisation and supervision of a clinical trial?

The authorisation and supervision of a clinical trial is the responsibility of the country where the trial is conducted. In Spain, this is the Agencia Española de Medicamentos y Productos Sanitarios(AEMPS), a state agency attached to the Ministry of Health.

Where are pricing and reimbursement decisions for orphan drugs made?

Once a medicine obtains marketing authorisation from the European Commission, decisions on price and reimbursement are taken in each country. In Spain, they are decided by the Interministerial Commission on Medicines Prices (CIMP), which is attached to the General Secretariat of Health and Consumer Affairs, as the collegiate body responsible for setting the maximum industrial price.

Who authorises the marketing of an orphan drug?

The European Commission through a centralised procedure, which allows a marketing authorisation to be obtained in all EU countries.