CRE Enfermedades Raras (Creer)
Burgos
Information Sections
Orphan drugs
- What are they
- Why are they given this name
- Who researches them
- Who produces them
- What regulations cover them
- A window onto orphan drugs
What are they
The named "orphan drugs" are those that are aimed at the treatment of diseases so infrequent that industry is reluctant to commercialize them under usual market conditions.
They are drugs hardly developed by chemist industry due to financial reasons, because they are aimed at reduced groups of patients and that, however, they answer public health necessities.
Why are they given this name
Their name comes from being similar to "those children who having no parents (orphan children), depended on different institutions to grow up".
Who researches them
They are researched by the same professionals who do it with the rest of drugs. But the conditions they must work in are much more difficult.
Who produces them
The process that goes from the discovery of a new molecule until it is brought to the market is long (10 years on average), expensive (several tens million euro) and no very certain (only 1 out of 10 tested molecules works therapeutically). Developing a drug aimed at treating a rare disease doesn’t allow, generally, recovering the capital invested in its research.
Industries that produce them are usually the same that produce other drugs, but they need a special financial support that only can be given by governments either direct support or through tax advantages and different kind of exemptions.
What regulations cover them
Every country has, in general, its own regulations related to orphan drugs. In our case, we are also affected by European regulations.
The European Union, in the Regulation (EU) number 141/2000 of the European Parliament and the Council of 16th December 1999 on orphan medicinal products, (date of effect: 22nd January 2000)
(108 Kb. pdf), defines the characteristics that a medicinal product must have to be called "an orphan drug". These are the following ones:
- That one pointed to establish diagnosis, prevention or treatment of a disease that affects less than five people per ten thousand in a community.
- That one intended to a serious disease treatment, or to a seriously debilitating condition, and that is unlikely to bring to the market without incentives, although there can be significant benefit to those affected by the treated disease.
