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.
1. О здоровье народа и системе здравоохранения. Кодекс Республики Казахстан от 7 июля 2020 года № 360-VI ЗРК. URL: https://adilet.zan.kz/rus/docs/K2000000360 (дата обращения: 18.01.2024).
2. Об утверждении правил проведения экспертизы лекарственных средств и медицинских изделий. Приказ Министра здравоохранения Республики Казахстан от 27 января 2021 года № ҚР ДСМ-10. URL: https://adilet.zan.kz/rus/docs/V2100022144 (дата обращения 10.10.2024).
3. Об утверждении правил формирования перечня орфанных заболеваний и лекарственных средств для их лечения. Приказ Министра здравоохранения Республики Казахстан от 16 октября 2020 года № ҚР ДСМ-135/2020. URL: https://adilet.zan.kz/rus/docs/V2000021454 (дата обращения: 18.01.2024).
4. Перечень орфанных заболеваний и лекарственных средств для их лечения (орфанных). Приложение 1 Приказа и.о. Министра здравоохранения РК от 31.12.2021 № ҚР ДСМ-142. URL: https://adilet.zan.kz/rus/docs/V2000021479 (дата обращения: 18.01.2024).
5. Правила регистрации и экспертизы лекарственных средств для медицинского применения. Решение Совета Евразийской экономической комиссии от 3 ноября 2016 г. № 78. URL: https://pravo.by/document/?guid=3871&p0=F91600333. (дата обращения: 18.01.2024).
6. Государственный реестр ЛС и МИ. URL: http://register.ndda.kz/category/search_prep (дата обращения: 18.01.2024)
7. Совещание по проблемным вопросам лекарственного обеспечения детей с орфанными заболеваниями и эпилепсией в MЗ PК от 08 июня 2021 года. URL: https://bala-ombudsman.kz/i-snova-ob-orfannyh-zabolevaniyah/ (дата обращения 24.01.2024).
8. Walkowiak D., Bokayeva K., Miraleyeva A., Domaradzki J. The Awareness of Rare Diseases Among Medical Students and Practicing Physicians in the Republic of Kazakhstan. An Exploratory Study // Front Public Health. 2022 Apr 8. 10:872648. doi: 10.3389/fpubh.2022.872648. PMID: 35462837. PMCID: PMC9031913.
9. Adachi T., El-Hattab A.W., Jain R., Nogales Crespo K.A., Quirland Lazo C.I., Scarpa M., Summar M., Wattanasirichaigoon D. Enhancing Equitable Access to Rare Disease Diagnosis and Treatment around the World: A Review of Evidence, Policies, and Challenges // Int J Environ Res Public Health. 2023 Mar 8. 20(6):4732. doi: 10.3390/ijerph20064732. PMID: 36981643. PMCID: PMC10049067].
10. American Association for the Advancement of Science 7000 Challenges: The Basis and Burden of Rare Diseases 2021. Available at: https://www.science.org/content/webinar/7000-challenges-basis-and-burden-rare-diseases (accessed 20.12.2023).
11. Balkhi B., Almuaither A., Alqahtani S. Cross-national comparative study of orphan drug policies in Saudi Arabia, the United States, and the European Union // Saudi Pharm J. 2023 Sep. 31(9):101738. doi: 10.1016/j.jsps.2023.101738. Epub 2023 Aug 9. PMID: 37638213. PMCID: PMC10458326.
12. Chan A.Y.L., Chan V.K., Olsson S., Fan M., Jit M., Gong M., Zhang S. et al. Access and Unmet Needs of Orphan Drugs in 194 Countries and 6 Areas: A Global Policy Review with Content Analysis. Value Health. 2020 Dec. 23(12):1580-1591. doi: 10.1016/j.jval.2020.06.020. Epub 2020 Oct 31. PMID: 33248513.
13. Daniel M.G., Pawlik T.M., Fader A.N., et al. The Orphan Drug Act: restoring the mission to rare diseases // Am. J. Clin. Oncol. 2016. 39:210–213.
14. European Commission EU, 2024. Public Health - Union Register of medicinal products. Community Register of orphan medicinal products. Available at: https://ec.europa.eu/health/documents/community-register/html/reg_od_act.htm?sort=a. (accessed 23.01.2024).
15. Giannuzzi V., Conte R., Landi A., et al. Orphan medicinal products in Europe and United States to cover needs of patients with rare diseases: an increased common effort is to be foreseen. Orphanet J. Rare Dis. 2017; 12:1–11
16. Herder M. What is the purpose of the orphan drug act? PLoS Med. 2017. 14: e1002191.
17. Ministerio de Salud Minsa financió a más de 42 mil Personas Con Enfermedades Raras, Huérfanas y de Alto Costo; 2019 Available at: https://www.gob.pe/institucion/minsa/noticias/49122-minsa-financio-a-mas-de-42-mil-personas-con-enfermedades-raras-huerfanas-y-de-alto-costo (accessed 25.12.2023).
18. Orphan Drug Act of 1983. Pub L. No. 97–414, 96 Stat. 2049. (accessed 20.12.2023). Available at: https://www.fda.gov/media/99546/download
19. Orphan drugs in the United States of America. (accessed 15.01.2024). Available at: https://rarediseases.org/wp-content/uploads/2021/03/orphan-drugs-in-the-united-states-NRD-2020.pdf
20. Padula W.V., Parasrampuria S., Socal M.P. et al. Market exclusivity for drugs with multiple orphan approvals (1983–2017) and associated budget impact in the US // Pharmaco Economics. 2020. 38, 1115–1121
21. Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products. Available at: https://eur-lex.europa.eu/legal-content/EN/TXT/?uri=celex%3A32000R0141 (accessed 20.01.2024).
22. Richter T., Nestler-Parr S., Babela R., Khan Z.M., Tesoro T., Molsen E., Hughes D.A. Rare Disease Terminology and Definitions - A Systematic Global Review: Report of the ISPOR Rare Disease Special Interest Group // Value Health. 2015. 18:906–914. doi: 10.1016/j.jval.2015.05.008.
23. Sequeira A.R., Mentzakis E., Archangelidi O., Paolucci F. The economic and health impact of rare diseases: A meta-analysis. Health Policy Technol. 2021. 10:32–44. doi: 10.1016/j.hlpt.2021.02.002.
24. U.S. Food and Drug Administration USFDA, 2024. Search orphan drug designations and approvals. accessed 23.01.2024. https://www.accessdata.fda.gov/scripts/opdlist.cfm
25. Vokinger K.N., Naci H. Negotiating Drug Prices in the US - Lessons from Europe // JAMA Health Forum. 2022. 3(12):e224801. doi:10.1001/jamahealthforum.2022.4801
26. World Economic Forum Global Data Access for Solving Rare Disease - A Health Economics Value Framework. 2020. Available at: https://www3.weforum.org/docs/WEF_Global_Data_Access_for_Solving_Rare_Disease_Report_2020.pdf (accessed 20.12.2023).
References: [1-7]
1. O zdorov'e naroda i sisteme zdravoohraneniуa [On the health of the people and the health care system]. Kodeks Respubliki Kazakhstan ot 7.07. 2020 g. № 360-VI ZRK [Code of the Republic of Kazakhstan dated July 7, 2020. №360-VI ZRK]. Available at: https://adilet.zan.kz/rus/docs/K2000000360 (accessed: 18.01.2024).
2. Ob utverzhdenii pravil provedeniya ekspertizy lekarstvennykh sredstv i meditsinskikh izdelii [On Approval of the Rules for Expert Review of Medicines and Medical Devices]. Prikaz Ministra zdravookhraneniya Respubliki Kazakhstan ot 27 yanvarya 2021 goda № ҚR DSM-10 [Order of the Minister of Health of the Republic of Kazakhstan dated January 27, 2021 № KR DSM-10] Available at: https://adilet.zan.kz/rus/docs/V2100022144 (accessed 10.10.2024).
3. Ob utverzhdenii pravil formirovaniya perechnya orfannykh zabolevanii i lekarstvennykh sredstv dlya ikh lecheniya [On Approval of the Rules for Forming a List of Orphan Diseases and Medicines for Their Treatment]. Prikaz Ministra zdravookhraneniya Respubliki Kazakhstan ot 16 oktyabrya 2020 goda № ҚR DSM-135/2020 [Order of the Minister of Health of the Republic of Kazakhstan dated October 16, 2020, № ҚР ДСМ-135/2020]. Available at https://adilet.zan.kz/rus/docs/V2000021454 (accessed: 18.01.2024).
4. Perechen' orfannykh zabolevanii i lekarstvennykh sredstv dlya ikh lecheniya (orfannyh) [List of orphan diseases and meditsines for their treatment (orphan diseases)]. Prilozhenie 1 Prikaza i.o. Ministra zdravookhraneniya RK ot 31.12.2021 № ҚR DSM-142 [Annex 1 of the Order of the Acting Minister of Health of the Republic of Kazakhstan from 31.12.2021 № KP DSM-142.]. Available at: https://adilet.zan.kz/rus/docs/V2000021479 (accessed: 18.01.2024).
5. Pravila registratsii i ekspertizy lekarstvennykh sredstv dlya meditsinskogo primeneniya [Rules for registration and expert examination of medicinal products for medical use]. Reshenie Soveta Evraziiskoi ekonomicheskoi komissii ot 3 noyabrya. 2016 g. № 78 [Decision of the Council of the Eurasian Economic Commission of November 3, 2016 № 78]. (accessed: 18.01.2024).Available at: https://pravo.by/document/?guid=3871&p0=F91600333.
6. Gosudarstvennyi reestr LS i MI [State Register of Medicinal Products and Medical devices]. Available at: http://register.ndda.kz/category/search_prep (accessed: 18.01.2024)
7. Soveshhanie po problemnym voprosam lekarstvennogo obespecheniya detei s orfannymi zabolevaniyami i epilepsiei v MZ PK ot 08 iyunya 2021 goda [Meeting on problematic issues of drug provision for children with orphan diseases and epilepsy in the MH RK from June 08, 2021]. (accessed 24.01.2024). https://bala-ombudsman.kz/i-snova-ob-orfannyh-zabolevaniyah/
Number of Views: 40
Category of articles:
Original articles
Bibliography link
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:
ANALYSIS OF CLINICAL AND DEMOGRAPHIC INDICATORS OF PATIENTS WITH PARKINSON'S DISEASE IN THE SOUTHERN REGION OF KAZAKHSTAN
A COMPARATIVE STUDY OF DRUG POLICY REGARDING ORPHAN DISEASES IN THE USA, THE EUROPEAN UNION, AND THE REPUBLIC OF KAZAKHSTAN
MUTATIONS IN CARDIAC ION CHANNEL GENES IN KAZAKHSTANI PATIENTS WITH LONG QT SYNDROME
GENETIC FEATURES OF CONGENITAL DYSFUNCTION OF THE ADRENAL CORTEX CAUSED BY 21-HYDROXYLASE DEFICIENCY IN PERSONS OF KAZAKH NATIONALITY
NON-MOTOR SYMPTOMS IN PARKINSON'S DISEASE USING THE EXAMPLE OF AKTOBE PATIENTS