Monograph: Rare diseases (3)

[Relationship between rare diseases and special drugs].

We leave the data aside and then focus on a topic that goes hand in hand with diseases: medicines.

Today, the main part of research in Europe is in the hands of private money, and we have nothing new to say when we say that the goal of any private company is to make money. But, of course, drugs intended to cure so few patients are usually not very cost-effective. That is why, in 1999, the European Union created special legislation to encourage and guarantee the research of medicines for these rare diseases. These drugs are called "orphan drugs."

CORTE: The status of orphan drugs is regulated by health authorities to promote drugs that provide little therapeutic opportunity and research that is not cost-effective. Therefore, an "orphan medicine" is the name given by the drug regulatory agency, and in order to be able to claim it, it is necessary to meet certain requirements: the disease must have a very low social presence and must also be a serious disease. In return, health authorities offer advantages to pharmaceutical laboratories investigating these drugs to ensure that people with rare diseases have the same rights as other patients.

The advantages or incentives obtained by private laboratories are basically three: partial or total tax exemption, support in the design of certain protocols and, above all, an exclusive period of 6 years for the commercialization of this drug. But the joy that gives a private laboratory the status of an orphan medicine goes beyond the humanitarian interest.

CORTE: Although the regulations are strict in this regard, and the status of orphan medicinal product can be lost at any time - if the onset of the disease increases, etc. - it is true that there is a second part: it is interesting that an orphan medicinal product reaches the market. In fact, many times when it reaches the market, it starts to be used for other different diseases, which will bring benefits to the laboratory.
The laboratories do not perform the tests, the laboratories sell. If you have received a kind of scholarship to investigate a certain disease and we already have the drug of that disease on the market, we can also use it... not like guinea pigs... but the drug is known and will begin to be used for other diseases, it is used for compassion for those diseases in clinical practice, and the laboratory, of course, makes a profit.

But despite the additional benefits they obtain and the incentives they receive from the public authorities, private laboratories are still far from researching medicines for a large family of rare diseases. Only 40 orphan drugs have been marketed in Europe, compared to 230 in the United States.

CORTE: I think it's political measures. In fact, research is in the hands of private capital, as is the case in most Western European countries. Imaginative policy measures, which are often difficult to implement, are therefore needed, but our politicians will have to decide whether to encourage public investment or whether to pass legislation such as orphan medicines; to give more incentives to laboratories investigating these medicines or even to penalise certain types of research.

Encourage public research, create legislation for new measures and increase the benefits of orphan drug research. They're the ones