About Orphan Drugs
"Orphan drugs" are medicinal products intended for diagnosis, prevention or treatment of life-threatening or debilitating rare diseases. They are "orphans" because the pharmaceutical industry has little interest under normal market conditions in developing and marketing drugs intended for only a small number of patients suffering from very rare conditions.
What is an "orphan drug"?
"Orphan drugs" are medicinal products intended for diagnosis, prevention or treatment of life-threatening or very serious diseases or disorders that are rare.
Encouraging new drug development: laws, incentives and advocacy
Orphan drug legislation, aimed at providing incentives for pharmaceutical companies to develop and market drugs to treat
Granting the label "orphan"
The first step in the development of any orphan drug is to obtain designation as an Orphan Medicinal Product (OMP). The Committee for Orphan Medicinal Products (COMP) is the ins
Orphan drug lists in Europe and the USA
The Enterprise DG (Directorate General) of the European Commission offers a list of all designated orphan drugs for the European market.
Orphan drugs: The role played by Eurordis
Eurordis plays an important role in the orphan drug development process through its participation in the Committee for Orphan Medicinal Products (COMP) at the EMEA (European Med
The Committee for Orphan Medicinal Products (COMP)
The principal task of this committee is to examine applications for the designation of Orphan Medicinal Product.
Working together on both sides of the Atlantic
In 2006, Eurordis initiated a comparative analysis of EU and US designated and marketed orphan drugs.
Rare Disease Patients represented at the EMEA Paediatric Committee
In the aftermath of the adoption of the EC Regulation for Paediatric Drugs, the European Medicines Agency (EMEA) has created a specific committee for paediatric
Improving patient access to orphan drugs in Europe
A good medication for rare disease patients is a medication that is both available in the country where they live and affordable. If one of these two factors is missing, the
Important steps towards new EU action for improving access to orphan medicines for all patients in the EU
How to promote the sustainable development of valuable orphan
Paediatric drugs and rare diseases
Paediatric Drugs: The facts: Many medicines administered to children have not been developed for them. Serious consequences, inefficacy and side effects often stem from in
Patient involvement in Protocol Assistance
Protocol Assistance to pharmaceutical companies developing new drugs is mentioned in the European Regulation on Orphan Medicinal Products. The regulation states that these
Compassionate access to orphan drugs in the USA
NORD, the National Organization for Rare Disorders in the USA, has many programs of advocacy, education, and research. It also offers
