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.
Promoting orphan drug development
Orphan drug legislation, aimed at providing incentives for pharmaceutical companies to develop and market drugs to treat
Orphan drug designation
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 US
The Enterprise DG (Directorate General) of the European Commission offers a list of all designated orphan drugs for the European market.
Role of 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 EMA (European Medi
Committee for Orphan Medicinal Products
The principal task of this committee is to examine applications for the designation of Orphan Medicinal Product.
Paediatric drugs and rare diseases
The use of unlicensed and off-label medicines, widespread among children affected by rare diseases resulted in inefficacy and serious side effects due to incorrect dosage
Survey: Patients’ Access to Orphan Drugs in Europe
EURORDIS and National Alliances join forces to assess the reality of access to orphan drugs for rare disease patients in Europe
GSK and Italian Telethon enter historic partnership
Turning gene therapy research into treatment
EURORDIS and Health Technology Assessment
HTA is a highly sophisticated evaluative method being used by health authorities to make decisions on healthcare policy
Compassionate access to orphan drugs in US
NORD, the National Organization for Rare Disorders in the US, has many programs of advocacy, education, and research. It also offers m
Celebrating 10 Years of the Orphan Drug Regulation in Europe
An event is being held this month at the European Parliament to celebrate 10 years of the Orphan Drug Regulation in Europe.
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 EMA 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 drugs.
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
EU improves access to orphan drugs
Important steps towards new EU action for improving access to orphan medicines for all patients in the EU.
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
CAVOD: Advocating to improve orphan drug access
Rare disease patients do not have equitable and timely access to the approved orphan drugs they need.
