Аннотация: The article analyzes current trends in the global orphan drug market regarding the registration of new orphan drugs and the expansion of indications for previously approved orphan medications intended for the pathogenetic treatment of rare diseases, as approved by the Food and Drug Administration (FDA) over the past five years. The findings are compared with current trends in the Russian market, including the expansion of screening programs, market competition, and patient access to therapy. The study results in a forecast of the introduction of 19 new orphan drugs that have been approved globally but are not yet available in Russia, including pediatric indications potentially funded through the Circle of Kindness Foundation (“Krug Dobra” Foundation), as well as adult indications eligible for reimbursement through regional budgets of the Russian Federation.