Orphan drug

orphan drugs are drugs that are not widely developed by the pharmaceutical industry for financial reasons, since they are intended for a small group of patients, and yet respond to public health needs.

The United States was the first country to develop a law, the Orphan Drug Act in 1983, making the orphan status allow subsidies for the development of these products, as well as other support measures, until commercial approval. The European Union launched a common policy in this area in 1999.

According to the European Union, an orphan drug is one that meets the requirements:

1. It is intended to establish a diagnosis, prevent or treat a disease that affects less than five people per ten thousand in the European Union.
2. That the treatment of a serious illness or that causes incapacities and whose marketing is unlikely (non-commercial) without stimulus measures.

Criteria for a drug to receive orphan drug status

• United States: useful for treating a disease with less than 200,000 patients/year, corresponding to a prevalence of 7.5/10 000 inhabitants.
• Japan: less than 50 000 patients/year, corresponding to 4/10 000 inhabitants.
• Australia: less than 2000 patients/year, corresponding to 1,1/10 000 inhabitants.
• European Union: affects less than 1 in 2000 (5/10 000) people or a maximum of 250 000 people throughout the European Union.