Introduction: Rare diseases are becoming more and more important since awareness is increasing. There are a lot of initiatives to ensure access to orphan drugs intended to treat these diseases but due to high price patients have low access and policy makers and payers are struggling with costs and assuring patient access rights to the available medication.
Aim: Objective of this study is to assess current situation regarding rare diseases in Bosnia and Herzegovina (BiH) and availability of orphan drugs. Four domains are examined: legislation and policy, diagnostics and research, patient organizations and reimbursement and availability of medicines for rare diseases.
Results: There are official programs and legislation ground for rare diseases in both entities of BiH: Republic of Srpska (RS) and Federation of BiH (FBiH). Diagnostic and genetic counseling are available but only for few rare diseases. Patient organizations are formed and active but still without engagement in some of international organizations. There are no special reimbursement criteria for orphan medicines. Number of reimbursed medicines is significantly lower than those on ORPHANET list, 67 and 26 in RS and FBiH respectively. Huge difference in number of reimbursed medicines within the country entities may be consequence of counting method.
Conclusion: Even thou rare diseases in BiH are recognized in countries official programs and documents, with patients having their organizations, much more have to be done in availability of medicines and diagnostics in the rare disease field. Insufficient funds are huge obstacle toward this goal. New methods of medicine evaluation are need and recommended in order of better availability.
Keywords: Rare diseases; health policy; orphan drug; pharmacoeconomic.