Evaluating the performance and development prospects of the High-Cost Nosology program: findings from an expert survey

Abstract

Background. In Russia, one of the key mechanisms for ensuring access to modern drug therapy for patients with severe chronic and rare diseases is the federal drug provision program for 14 High-Cost Nosologies (HCN).

Objective: To evaluate the current performance of the 14 HCN program from the perspective of healthcare professionals directly involved in its implementation and to identify priority areas for program improvement.

Material and methods. A survey was administered to specialists responsible for drug provision within the 14 HCN program. The questionnaire included nine questions using interval and ordinal response scales. Of 87 questionnaires received, 28 valid responses were included in the final analysis. Instrument reliability was assessed using Cronbach’s alpha, and agreement among expert judgments was evaluated using Kendall’s coefficient of concordance and Pearson’s chi-square test.

Results. The questionnaire demonstrated satisfactory internal consistency (α=0.799). According to respondents, the most critical challenges of the program were insufficient funding (mean score 4.29), the need to improve the drug provision system (4.21), and the need to automate the submission and review of applications for medicinal products (4.18). Moderate agreement among experts was observed only in ranking nosologies by priority for implementing improvement measures (W=0.3089). Hemophilia, multiple sclerosis, and malignant neoplasms of lymphoid, hematopoietic, and related tissues were identified as the highest-priority conditions.

Conclusion. The findings confirm need to strengthen the 14 HCN program and may inform the development of organizational and pharmacoeconomic optimization strategies.