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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Orphan Medicines Regulation

The growing number of rare diseases awaiting treatment are a public health issue. The scarcity of incentives for medicines manufacturers and the lack of documentation supporting the applications limit the number of new orphan medicines products.

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Orphan medicines designation

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

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Improve the safety of your medicines (pharmacovigilance)

This section explains what you can do if you suspect a side effect possibly due to your treatment

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Early access to medicines

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.

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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.

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Patient advocates involvement

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

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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.

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Health technology assessment

The goal of HTA is to support health care decisions and to serve policy making through objective information.

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EURORDIS Round Table of Companies (ERTC)

The EURORDIS Round Table of Companies (ERTC) brings together pharmaceutical companies with a common interest in rare diseases and orphan medicinal product development

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