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.

Orphan Medicines Regulation

The growing number of rare diseases lacking treatment is an important public health issue. Often the scarcity of incentives for drug manufacturers and the lack of evidence supporting the applications limit the number of new orphan drugs that ultimately access the market.

Orphan medicines designation

The first step in the development of any orphan medicinal product is to obtain designation as an Orphan Medicinal Product.

Role of EURORDIS

EURORDIS plays an important role in the orphan drug development process through participation in the Committee for Orphan Medicinal Products at the European Medicines Agency.

Patient advocates involvement

Patient advocates are actively involved in orphan medicines development and regulatory affairs.

Access to orphan drugs

A good medication for rare disease patients is a medication that is both available in the country where they live and affordable.

Compassionate Use

When a new medicine is being developed, patients want to know when it will be available. Not all patients can wait until the end of clinical trials and the evaluation of the marketing authorisation application. Compassionate Use Programmes are designed to satisfy these needs.