Orphan drugs used for treatment in pediatric patients in the slovak republic

Orphan drugs used for treatment in pediatric patients in the slovak republic Due to the enormous success of scientific research in the field of paediatric medicine many once fatal children's diseases can now be cured. Great progress has also been achieved in the rehabilitation of disabilities....

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Bibliographic Details
Published in:Acta Facultatis Pharmaceuticae Universitatis Comenianae 2012-01, Vol.LIX (Supplement-VI), p.28-34
Main Authors: Foltánová, T., Konečný, M., Hlavatá, A., Štepánková, K., Cisárik, F.
Format: Article
Language:eng ; cze ; ger
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Accessibility
Availability
Orphan drugs
Paediatric regulation
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Summary:Orphan drugs used for treatment in pediatric patients in the slovak republic Due to the enormous success of scientific research in the field of paediatric medicine many once fatal children's diseases can now be cured. Great progress has also been achieved in the rehabilitation of disabilities. However, there is still a big group of diseases defined as rare, treatment of which has been traditionally neglected by the drug companies mainly due to unprofitability. Since 2000 the treatment of rare diseases has been supported at the European level and in 2007 paediatric legislation was introduced. Both decisions together support treatment of rare diseases in children. In this paper, we shortly characterise the possibilities of rare diseases treatment in children in the Slovak republic and bring the list of orphan medicine products (OMPs) with defined dosing in paediatrics, which were launched in the Slovak market. We also bring a list of OMPs with defined dosing in children, which are not available in the national market. This incentive may help in further formation of the national plan for treating rare diseases as well as improvement in treatment options and availability of rare disease treatment in children in Slovakia. Lieky na zriedkavé choroby, používané na liečbu detských pacientov v slovenskej republike Vďaka vedeckému pokroku v liečbe detských ochorení, mnohé doposiaľ neliečiteľné detské choroby sa stávajú liečiteľnými. Veľký pokrok sa zaznamenal tiež v rehabilitácii porúch. Stále však existuje skupina chorôb, definovaných ako zriedkavé, liečba ktorých bola zo strany farmaceutického priemyslu nedostatočne podporovaná a to najmä kvôli finančnej nerentabilite investovaných prostriedkov. Od roku 2000 liečba zriedkavých chorôb je podporovaná európskou legislatívou a v roku 2007 bola predstavená legislatíva na podporu liečby detských chorôb. Obidve rozhodnutia tak spoločne podporujú liečbu zriedkavých chorôb u detí. V tejto práci prinášame stručnú charakteristiku liečebných možností zriedkavých chorôb u detí v Slovenskej republike, zoznam liekov na zriedkavé choroby s definovaným dávkovaním u detí, ktoré sú dostupné na slovenskom trhu. Tiež uvádzame zoznam liekov na zriedkavé choroby s definovaným dávkovaním u detí, ktoré v Slovenskej republike nie sú priamo dostupné. Touto prácou chceme dalej podporiť rozvoj Národného plánu pre zriedkavé choroby.
ISSN:0301-2298
1338-6786
2453-6725
DOI:10.2478/v10219-012-0001-0