The Orphans Amongst Drugs

01.03.2007

Five to ten million people in the European Union suffer from rare diseases. Nobody knows the exact number, as nobody has counted the persons concerned as yet. Often it takes a long time until a diagnosis is established. Drugs are only seldom available. A regulation from the EU creates incentives to make research more attractive.

The search for the right diagnosis lasted for two years. Claus SchröterŽs daughter wasnŽt even a year old when her blood test results started to raise questions. The family was send from one physician to the other, until the illness finally received a name: Marfan syndrome, a rare genetic disease, that can lead to death if it isnŽt detected, as the widened common carotid artery can tear abruptly. It was this experience that led Schröter to launching the Nationale Netzwerk Seltener Krankheiten (national network for rare diseases) with which he wants to spare thousands of affected people this odyssey. "Each day we receive three inquiries from people who are searching for the right physician”, says Schröter. "In the meantime we know where the specialists for about 130 diseases are to be found.”

About 5000 severe illnesses all over the world fall into the category of rare diseases. In this case less than five in 10.000 people are affected. Since there are so many different illnesses and they appear so infrequently it is not only difficult to find the right diagnosis. More often than not there is no drug for the ailment. The pharmaceutical industry is not interested in developing medicine for treating Marfan syndrome, Mucoviscidosis or Fabry disease. They prefer investing their money into research which leads to pills for treating widespread diseases like diabetes. In Germany alone several million people suffer from this illness and not only a couple of thousand like from Marfan syndrome.

The pharmaceuticals against rare diseases are called orphan drugs because they are neglected from pharmaceutical companies. The European Union wanted to change this in the year 2000 by offering financial incentives to pharmaceutical manufacturers. If a company obtains an orphan drug status it for example doesnŽt have to pay fees or it receives a scientific counselling from the European Medicines Agency (EMEA), which normally costs 60.000 Euro, free of charge.

The Committee for Orphan Medicinal Products (COMP) decides who gains this status. Professor Rembert Elbers is the German representative in this EU committee, which consists of 33 members. "The chief advantage for enterprises is the exclusiveness on the market which is guaranteed for ten years after market admission as an orphan drug,” says the Professor who is also head of the oncological department at the Federal Institute for Drugs and Medical Devices. For ten years no other company is allowed to put a similar product on the market. No such regulation exists for other drugs. Thus for example shortly after Pfizer launched its drug against impotence Viagra other companies developed similar drugs.

If the financial incentives are really working can be doubted according to Claus Schröter. He thinks the decree is a good idea, but "it doesnŽt really work”, he says, "most of the orphan drugs still originate in the USA.” The Orphan Drug Act in the USA was already launched in 1983. Elbers also isnŽt confident if the stimuli works: "The applications for orphan drugs are indeed rising continuously”, he says. Thus in the EU more than 450 applications for an orphan drug status were made until 2005. "However, this does not come as a surprise. For up to the year 2000 there was no possibility in the EU to designate a medicament as an orphan drug. It is difficult to say how much research was really initiated by the incentives.”

The pharmaceutical industry might have to rethink their concept in the future anyway. For due to modern genetic examinations, illnesses can be diagnosed more and more exactly. Thus widespread diseases like hypertension start to split into evermore subspecies. "Where in the past one has seen only one disease, one starts to realize today that actually there are many different subspecies which might all be in need of a different therapy”, explains Elbers. Thus, the good old days in which a sole drug for one disease could be developed which would then be bought by millions of people, might soon belong to the past.

REHACARE.de

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