Legal basis of pricing for orphan medicines in the context of government regulation

in this paper, the authors investigate the legal basis of pricing orphan medicines in the context of government regulation and limited healthcare resources. Special attention is paid to economic mechanisms for stimulating the development of rare medicines, which include the provision of market exclusivity for a limited period, tax incentives for development companies, simplified and accelerated registration procedures, as well as direct government funding for preclinical and clinical research. The system of legal regulation of the cost of orphan drugs is considered, including the establishment of marginal selling prices, rules for state registration of prices, and procedures for their revision, taking into account data on clinical efficacy and actual practice of use. Cost recovery approaches based on multi–level financing (federal and regional), as well as the use of pharmacoeconomic assessment tools such as cost-effectiveness and budget impact analysis are analyzed. The current regulatory legal acts of the Russian Federation regulating pricing, the inclusion of drugs in the lists of vital and essential medicines, the public procurement procedure and contractual risk?sharing models have been studied. The results of the study confirm the need to improve pricing mechanisms, increase transparency of procedures and develop a comprehensive incentive system that ensures a balance between the availability of therapy for patients with rare diseases and maintaining economic motivation for pharmaceutical companies to innovate. The authors have developed innovative approaches to the regulation of orphan drugs, providing for the introduction of adaptive pricing based on data from real clinical practice. This approach will allow dynamically changing the level of compensation, taking into account the actual achieved therapeutic effectiveness and the impact of therapy on the quality of life of patients.

orphan medicines, rare diseases, government regulation of prices, economic incentives, cost recovery system, pharmacoeconomic assessment, exclusive rights to the market