Background. Hemophilia is a rare disease covered by Russia’s federal program for 14 High-Cost Nosologies (HCN), which ensures nationwide access to expensive drug therapies funded from the federal budget. The program currently includes 11 hemophilia drug products; however, the absence of transparent, clinically grounded criteria for selecting among these options in specific clinical scenarios complicates therapeutic decision-making.

Objective: To conduct a systematic review of the clinical effectiveness for hemophilia drugs included in the 14 HCN program and to develop an approach for establishing treatment selection criteria for specific patient groups.

Material amd methods. A literature search was performed in PubMed/MEDLINE and the Cochrane Library. Comparative clinical trials, systematic reviews, and meta-analyses were included. In addition, clinical guidelines for hemophilia and prescribing information for the relevant drugs were analyzed.

Results. The available evidence base is heterogeneous and does not permit a comprehensive comparison of all drugs included in the program. Nevertheless, several patterns in clinical effectiveness and important limitations of the evidence relevant to treatment selection were identified. Based on clinical trial data, clinical guidelines, and prescribing information, a methodological approach for developing drug selection criteria was proposed. This approach incorporates patient models, typical clinical scenarios, and an automated treatment selection algorithm.

Conclusion. The proposed approach may support the standardization of prescribing in hemophilia and can be adapted for other diseases included in the 14 HCN program.