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.

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Promoting orphan drug development

Orphan drug legislation, aimed at providing incentives for pharmaceutical companies to develop and market drugs to treat

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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

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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.

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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

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Committee for Orphan Medicinal Products

The principal task of this committee is to examine applications for the designation of Orphan Medicinal Product.

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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

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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

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GSK and Italian Telethon enter historic partnership

Turning gene therapy research into treatment

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EURORDIS and Health Technology Assessment

HTA is a highly sophisticated evaluative method being used by health authorities to make decisions on healthcare policy

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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

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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.

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Working together on both sides of the Atlantic

In 2006, Eurordis initiated a comparative analysis of EU and US designated and marketed orphan drugs.

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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.

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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

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EU improves access to orphan drugs

Important steps towards new EU action for improving access to orphan medicines for all patients in the EU.

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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

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CAVOD: Advocating to improve orphan drug access

Rare disease patients do not have equitable and timely access to the approved orphan drugs they need.

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