Regulatory Framework for Gene Therapy Medicinal Products

INTRODUCTION. Advances in gene therapy technologies and their expanding clinical use are progressing faster than the evolution of regulatory frameworks. In the Russian Federation, oversight of cell- and gene-based therapies remains complex and fragmented due to the interplay between national legislation and supranational regulations within the Eurasian Economic Union.
AIM. This study aims to examine the current regulatory framework governing the lifecycle of gene therapy products in the Russian Federation, including their authorization, manufacturing, and funding; to identify regulatory gaps and inconsistencies; and to propose directions for policy improvement.
MATERIALS AND METHODS. A combined systems-based and problem-oriented approach was applied to the analysis of regulatory legal acts. The study was conducted in four stages: first, a content analysis of the regulatory framework was performed to identify key concepts; second, requirements across different legal acts were compared to detect areas of ambiguity and regulatory inconsistency; third, the identified issues were systematized using structural-functional and logical analysis; and finally, recommendations were formulated based on the integrated findings.
RESULTS. The analysis covered Federal Laws No. 61 and No. 180, decisions of the Eurasian Economic Commission Council, and subordinate regulations governing Good Manufacturing Practice (GMP) and the State Guarantees Program (SGP). Depending on their characteristics, gene therapy products may fall under several regulatory categories, including advanced therapy medicinal products (ATMPs), biotechnological medicinal products, or biomedical cell products, each associated with distinct requirements for market authorization, manufacturing, and patient access. The study highlights fundamental differences in the regulatory treatment of personalized therapies compared with standardized products. Notably, there is currently no established legal framework permitting centralized, industrial-scale manufacturing of personalized therapies with subsequent transport of biological materials, which poses a significant barrier to scalability. In addition, further development of reimbursement mechanisms is needed for gene therapy interventions that do not require market authorization, in order to broaden patient access beyond those treatments already covered by the SGP, specifically within List 4 of High‑Tech Medical Care.
CONCLUSIONS. Improving access to innovative therapies will require alignment of Federal Laws No. 61 and No. 180 with Decision No. 78 of the Eurasian Economic Commission Council, harmonization of classification criteria for gene therapy products, establishment of a regulatory pathway for centralized manufacturing of personalized therapies, and the introduction of more flexible reimbursement models to support patient access.

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