Press review

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The odyssey of finding treatment for rare diseases

MADRID- Nov 3 (APM) - On Monday, daily El País carried a lengthy story about the difficulties in accessing treatments for rare diseases depending on the region where patients are treated and their high prices.
According to the newspaper, only 46 out of the 95 EU-approved orphan drugs are available in Spain. The article included the stories of a number of patients, some of whome said they had had to move house or change their tax address because the drugs they needed were not available in their region or their reference hospital.
It is up to regional pharmaceutical commissions or even individual hospitals in Spain to decide whether they provide specific orphan drugs or not. “Sometimes, a final decision is made based on clinical criteria; sometimes it depends on the drug’s price and its impact on the budget. In any case, there are not homogeneous criteria,” El País reported.
Antonio González-Meneses, a paediatrician at Virgen del Rocío Hospital in Seville, told El País that patients' medical records containing the same objective information can receive different funding reports depending on which region the patient lives in. He admitted this can also happen to patients with very similar conditions treated in the same region.
El País said another reason why it can be difficult to get treatment for a rare disease is lack of efficacy of available treatments.
Genzyme’s Pompe disease drug Myozyme (alglucosidase alpha), for example, has shown efficacy in children. However, when administered in cases where the disease is diagnosed later in life, its benefits are considered “limited”, the newspaper reported.
Orphan drugs are therefore “special” both in terms of price and efficacy. Following approval by the EU, pricing talks in each country will result in different access conditions, El País said.

Thalidomide and other drugs which 'came back from the dead'

On Wednesday, daily El Mundo carried a story about a number of unsuccessful or even prohibited drugs which are now being use in unexpected ways, including thalidomide.
The newspaper's theory is that this will happen with an even bigger number of old drugs due to new research strategies.
The best known of this group of drugs is Grünenthal’s thalidomide, which induced a change in the way drug safety controls are established following the “catastrophe” it caused in the late 1950s .
Thalidomide is still used today because shortly after it was withdrawn, a physician from Israel discovered it could alleviate inflammatory lesions in patients with leprosy. Later on, benefits in multiple myeloma were observed, El Mundo reported.
A research team from Pompeu Fabra University in Barcelona is currently studying the benefits of rimonabant, a failed anti-obesity drug withdrawn due to psychiatric side effects, in patients with fragile X syndrome.
Cisapride and tolcapone are other examples of these drugs the newspaper referred to as “zombie” medicines.
El Mundo said Barcelona-based drug repositioning firm Som Biotech has been successful in using Parkinson’s disease drug tolcapone to treat transthyretin amyloidosis, a rare condition which affects cardiac and nervous tissues.

Stada moves tax address out of Barcelona

Generics firm Stada has moved its tax address from Barcelona to Madrid due to political agitation, it was widely reported on Tuesday.
Daily ABC quoted a company statement as saying the firm decided to relocate the tax address of its Spanish subsidiary “to protect its clients, employees and stockholders’ interests". According to the statement, this change will not affect “in any way” drug supplies to community pharmacies, wholesalers or hospitals, the newspaper said.
Financial Expansión reported that a total of over 1,800 companies have moved their headquarters out of Catalonia since the crisis started, to ensure legal guarantees are protected. (APMHE 55010)
El Economista noted that Stada sells more than 600 generic medicines and over-the-counter drugs in Spain.
Dailies 20 Minutos, El Periódico, La Vanguardia and medical journal Redacción Médica also carried the story.

Data on Merck KGaA’s Mavenclad presented at ECTRIMS

Data on Merck KGaA’s Mavenclad (cladribine) were presented at the latest ECTRIMS/ACTRIMS meeting in Paris, medical journals El Médico Interactivo, Gaceta Médica and Diario Médico reported on Saturday and Sunday.
Gaceta Médica carried the headline: “Cladribine protects patients with relapsing multiple sclerosis for four years". The drug has already been approved by the European Commission and is available in Germany, whereas in Spain pricing talks are ongoing, the journal noted.

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