High cost orphan drugs are regulated with different levels of rigorousness in different CEE countries. Some drugs are covered within drug reimbursement list, and some of them are financed from the other areas of special funds e.g. therapeutic programs, but the more drug is costly the stricter regulations should be enforced. The objective of this Forum is to present and discuss current regulations benefits in the region, as well as to highlight the bottlenecks that should be improved in order to enlarge the coverage and enable patient access to the therapies of proven efficacy. The conclusions of the forum presentations could be also used as a guiding principle to build the evidence based management of rare diseases in developing countries with limited resources and limited capacities to perform complex economic analysis. This interactive forum anticipates to entice high participation from the audience.