How to deal with rare pediatric surgical diseases in the small developing country with limited resources: Insights from Bosnia and Herzegovina

Abstract

The European Union Regulation on Orphan Medicinal Products defines a disease/condition as rare if it affects < 1 in 2.000 in the European population, making the prevalence data a key tool in creating health care policies for patients with rare diseases (RD). There is approximately 7.000 RD, mainly genetic, affecting approximately 30 million people in Europe and between 25 and 30 million in the United States. Due to increased public awareness, improved understanding and treatment of the relatively more common disorders, elimination of nutritional deficiencies, and the development of laws related to the treatment of RD, pediatric rare diseases (PRD) have recently received increased attention. The numerous challenges clinicians face in treating patients with PRD are due to the lack of standardized diagnostic criteria, referral pathways, and treatment guidelines.