A COMPARATIVE STUDY OF DRUG POLICY REGARDING ORPHAN DISEASES IN THE USA, THE EUROPEAN UNION, AND THE REPUBLIC OF KAZAKHSTAN
Background: Drug policy for orphan diseases is an area of particular focus, given the medical needs associated with rare diseases and the relatively large impact that these diseases and their treatment have the potential to have on health budgets.
Aim of the study: The main objective of this study was to compare drug policies for orphan diseases in the United States of America (USA), the European Union (EU) and the Republic of Kazakhstan and to assess their impact on the approved national lists of medicines for rare diseases.
Materials and methods: Lists of all drugs that have been granted orphan drug status and are authorised in the US, EU and the Republic of Kazakhstan pharmaceutical markets were obtained using national drug registries available in regulatory databases. The study was conducted from December 2023 to January 2024. Search and comparative analyses were performed by ATX code and INN. Statistical analysis included descriptive statistics. Data processing was performed in Microsoft Excel 2022 MSO software (version 2312 Build 16.0.17126.20132).
Results: The study found that the US and EU have introduced drug policies that encourage the development of orphan drugs, while the Republic of Kazakhstan has yet to develop a clear policy, which is a contributing factor to the lower number of approved drugs for rare diseases in the country. The domestic list of orphan drugs includes 14.8% of the total number of (Food and Drug Administration) FDA-approved orphan drugs and 11.6% of the total number of European Medical Agency (EMA) - approved orphan drugs.
Conclusions: The results of this study show a significant gap in the availability of medicines for orphan diseases in the Republic of Kazakhstan compared to the USA and the EU, which indicates the need to improve drug policy for orphan diseases. Adequate measures should be taken to introduce special state incentives for the development of orphan drugs by domestic manufacturers. Based on the results of the study, it would be advisable to ensure that the drugs for the treatment of orphan diseases included in the national list are brought to the pharmaceutical market, and to consider the possibility of expanding the approved list based on drugs included in the drug registers of the USA and Europe.
Dinara Y. Kaliyeva1, https://orcid.org/0000-0001-8231-7965
Asiya K. Turgambayeva1, https://orcid.org/0000-0002-2300-0105
Zakira A. Kerimbayeva1, https://orcid.org/0000-0003-2618-3151
Zhandulla Nakipov1, https://orcid.org/0000-0001-5528-693X
Saniya Saussakova1, https://orcid.org/0000-0003-0394-5117
1 NJSC "Astana Medical University", Department of public health and management,
Astana, Republic of Kazakhstan.
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Kaliyeva D.Y., Turgambayeva A.K., Kerimbayeva Z.A., Nakipov Zh., Saussakova S. A comparative study of drug policy regarding orphan diseases in the USA, the European Union, and the Republic of Kazakhstan // Nauka i Zdravookhranenie [Science & Healthcare]. 2024, (Vol.26) 1, pp. 22-30. doi 10.34689/SH.2024.26.1.003Related publications:
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