The place of real-world evidence in conducting clinical and economic analysis

Relevance. Real-world evidence (RWE) can be widely used in cost-effectiveness analysis (CEA) and budget impact analysis (BIA) conducted to justify the inclusion of new medicines in restrictive drug lists. However, researches of RWE use experience in drug assessment in the Russian Federation is currently limited, which makes it necessary to conduct such a research. Our goal is to analyze of the practice of using RWE in preparation of dossiers, conducting CEA and BIA for drugs proposed for inclusion in the restrictive drug lists in the Russian Federation. Materials and methods. The practice of using RWE in a drug assessment was studied through a content analysis of 28 proposals for the inclusion of drugs in restrictive lists (vital and essential drugs list, high-cost nosology list) in 2022, published CEA (n=109) and BIA (n=82) which were used for drug inclusion in 2018–2024. Results. In the dossier, real-world studies were used primarily for descriptive purposes, to characterize the proposed drug, current treatment practice, and the therapeutic area. In published CEAs RWE was most often used to increase the completeness of costs taken into account (34 % of uses), increase the accuracy of cost calculations (24 %), and take effectiveness into account when calculating the cost-effectiveness ratios (19 %). In the BIA RWE was most often used to calculate the target patient population (51 % of evidence uses), increase the accuracy of cost calculations (22 %), and more fully account for costs (18 %). RWE characterized the proposed drug and/or comparator in only 44 % of cases in CEAs and 22 % in BIAs; in the remaining cases, it described patients, characteristics of medical care, or the use of medical interventions after or as a result of the use of the compared medicinal products. The use of RWE from other countries is prevalent in CEAs (62 % of uses) and accounts for a significant share in the BIAs (31 % of uses). Conclusions. In drug assessment RWE is used for a wide range of purposes and characterizes a wide range of parameters for the drugs under scope, target patient groups, treatment patterns, and the long-term outcomes of using the drug. The identified specific features of the RWE use practice differ from expectations, according to which its use should be focused primarily on the compared drugs and describe their effectiveness and safety. It was shown that RWE most often did not describe compared drugs and did not address their efficacy and safety. The significant share of non-local RWE necessitatesthe development of measures aimed at increasing the volume of local RWE.