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

T. Foltánová 1 , M. Konečný 2 , A. Hlavatá 3 , K. Štepánková 4 , and F. Cisárik 5
  • 1 Faculty of Pharmacy, Department of Pharmacology and Toxicology, Comenius University in Bratislava
  • 2 Department of Clinical Genetics, St. Elizabeth Cancer Institute, Bratislava
  • 3 2Department of Pediatrics, UniversityChildren's Hospital, Bratislava
  • 4 Slovak Cystic Fibrosis Association, Košice
  • 5 Department of Medical Genetics, Faculty Hospital, Žilina

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.

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