What is an Orphan Drug?

I have to confess I didn’t really know, I had to google it, this is what I found out.

According to www.orphanet.org.uk an orphan drug is a pharmaceutical agent that has been developed specifically to treat a rare medical condition, the condition itself being referred to as an orphan disease.

Orphan drugs are intended to treat diseases which are so rare that sponsors are reluctant to develop them under usual marketing conditions.

The small size of the market they represent would not allow sponsors to recover the capital invested on the research and development of the project.

Patients with rare diseases cannot stay on the sidelines of the progress made by science and therapeutics, they have the same rights to treatment as any other patient.

In order to stimulate research and development in the sector of orphan drugs, public authorities have implemented incentives for health and biotechnology industries.

Rare diseases, are diseases which affect a small number of people compared to the general population, and specific issues are raised in relation to their rarity.

In Europe, a disease is considered to be rare when it affects 1 person per 2000. There are thousands of rare diseases. To date, six to seven thousand rare diseases have been discovered, and new diseases are regularly described in medical literature.

Many thanks

I attend many Orphan Drug Conferences and it is pleasing to note the high interest in Ataxia (it may be as many symptoms of many conditions overlap.

It is also good to be able to interact with many from the Rare disease / Orphan Drug Community, indeed my patient engagement reputation is very welcome.