Comparative Analysis of the Availability of Medicines for Patients with Orphan Diseases in the Republic of Kazakhstan for 2015 and 2020

Abstract

The aim of research: To conduct a comparative analysis of pathologies included in the List of orphan (rare) diseases in Kazakhstan for 2015 and 2020 with a further assessment of the dynamics of changes in the structure of the included diseases. Methods. In this paper, by using logical, systematic, and documentary analysis, a comparative analysis of the current legislation on the regulation of the treatment of orphan diseases in the Republic of Kazakhstan was carried out, as well as a comparative analysis of changes in this legislation. The data was used for 2015 and 2020. Results. Within the framework of the Order "On approval of the List of Orphan (Rare) Diseases" of the year 2015, this List included patients with the following diseases: (C) various types of neoplasms (this group included 16 nosologies, which is 32% of the total number of the included diseases), (E) diseases of the endocrine system, eating disorders and metabolic disorders (9 nosologies were included, the coverage rate was 18%), (D) diseases of the blood, hematopoietic organs and individual disorders involving the immune mechanism (8 nosologies were presented, which accounted for 16% of the total coverage of the included diseases), (G) diseases of the nervous system (total-4 nosologies, 8% of the total coverage), (A-B) some infectious and parasitic diseases (Anthrax, Crimean hemorrhagic fever, Malaria, Leishmaniasis – 4 nosologies, 8% of the total coverage), (Q) Congenital anomalies, deformities and chromosomal abnormalities (there are 3 nosologies, 6% of the total number), (M) Diseases of the musculoskeletal system and connective tissue (there are 2 nosologies, 4% of the total number of nosologies covered) and 1 disease according to the ICD-10 codes: (I) Diseases of the circulatory system, (J) Diseases of the organs, (K) Diseases of the digestive system, (L) Diseases of the skin and subcutaneous tissue. In 2020, the List of orphan diseases and medicines for their (orphan) treatment was updated. According to the new document, 62 groups of nosologies were already covered, that is 12 nosologies more than in 2015 and which accounts for 12%. The ratio of nosology coverage has not changed over time. Conclusions: In conclusion, it should be noted that due to the economic and political changes carried out in the field of health care in Kazakhstan over the past ten years, the coverage of medical care for patients with orphan diseases is constantly increasing. The drug supply of the corresponding categories of patients is also growing. The updated Code and by-laws will contribute to strengthening the health of the nation, including patients with rare (orphan) diseases. It is worth noting that an important factor is the development and approval of rules for the formation of the List of orphan diseases and medicines for their treatment with certain selection criteria. Thus, undoubtedly, there is an improvement in the dynamics of access to medical care for patients with orphan diseases in the Republic of Kazakhstan. Keywords: Orphan diseases, Rare diseases, Orphan medicines, Drug policy, Kazakhstan.