Analysis of the availability of health technologies for the treatment of phenylketonuria in Ukraine and the world
DOI:
https://doi.org/10.24959/sphhcj.23.278Abstract
Phenylketonuria (PKU) is a rare (orphan) disease characterized by a severe, chronic, progressive course. Patients' quality of life depends on timely detection (screening) and proper treatment - the use of special medical food products, and therefore, their availability and affordability.
The goal (objective) of the study is to analyze the available health technologies for the treatment of PKU in Ukraine and the world.
Materials and methods. Research objects: scientific publications, guidelines, statistical data, marketing information. Methods: analysis, comparison, generalization, system analysis.
Results of the research. Guidelines and protocols for the treatment of patients with PKU/HFA define a diet as a key component, taking into account the age and features of the course of the disease. The analysis of providing patients with PKU with special medical food products in different countries shows significant differences in the approaches to their assignment and reimbursement. The quantity, availability, range and composition of these products vary significantly. In the UK, they are included in the national formulary, are available on prescription and are subject to reimbursement. In Ukraine, local authorities provide PKU patients with special medical food products by procurement through the ProZorro electronic system in accordance with the calculated need. In the domestic market, there is a wide range of specialized products for patients with PKU/HFA, mainly of imported production, which have a high cost and, accordingly, low accessibility for the population.
Conclusions. The introduction of reference centers and extended neonatal screening, the creation of a register of such patients, the development of domestic production of products for PKU/HPA, and the creation of effective procurement and reimbursement mechanisms will contribute to increasing the effectiveness of treatment, the quality and life expectancy of patients.
References
DETs MOZ Ukrainy (2015). Adaptovana klinichna nastanova, zasnovana na dokazakh. Fenilketonuriia ta inshi hiperfenilalaninemii [Adapted evidence-based clinical practice. Phenylketonuria and other hyperphenylalaninemias]. Retrieved from: http://www.dec.gov.ua/
Tutuk V. V., Nazarkina V. M. (2023). Suchasni medychni tekhnolohii diahnostyky y likuvannia fenilketonurii: dostupnist dlia patsiientiv [Modern medical technologies for diagnosis and treatment of phenylketonuria: accessibility for patients]. Formuvannia Natsionalnoi likárskoi polityky za umov vprovadzhennia medychnoho strakhuvannia: pytannia osvity, teorii ta praktyky (S. 183-194). NUPh.
Wood G., Pinto A., Evans S. et al. (2021). Special Low Protein Foods Prescribed in England for PKU Patients: An Analysis of Prescribing Patterns and Cost. Nutrients, 13 (11), 3977. Retrieved from: https://doi.org/10.3390/nu13113977
rozporiadzhennia KMU «Pro skhvalennia Kontseptsii rozvytku systemy nadannia medychnoi dopomohy patsiientam, yaki strazhdaiut na ridkisni (orfanni) zakhvoriuvannia, na 2021-2026 roky» №377 (28.04.2021). [On the approval of the Concept of the development of the system of providing medical care to patients suffering from rare (orphan) diseases for 2021-2026]. Retrieved from: https://zakon.rada.gov.ua/laws/show/377-2021-%D1%80#Text
Statystychni dani systemy MOZ. Tsentr hromadskoho zdorovia MOZ Ukrainy [Statistical data of the system of the Ministry of Health. Public Health Center]. Retrieved from: http://medstat.gov.ua/ukr/statdanMMXIX.html
nakaz MOZ Ukrainy «Unifikovanyi klinichnyi protokol pervynnoi, vtorynnoi (spetsializovanoi) ta tretynnoi (vysokospetsializovanoi) medychnoi dopomohy. Fenilketonuriia ta inshi hiperfenilalaninemii» №760 (19.11.2015). [Unified clinical protocol of primary, secondary (specialized) and tertiary (highly specialized) medical care. Phenylketonuria and other hyperphenylalaninemias]. Retrieved from: https://www.dec.gov.ua/wp-content/uploads/2019/11/2015_760_ykpmd_fenilk.pdf
Vockley J., Andersson H. C., Antshel K. M. et al. (2014). Phenylalanine hydroxylase deficiency: diagnosis and management guideline. Genetics in Medicine, 16(2), 188-200. doi: 10.1038/gim.2013.157.
Wegberg A. M. J., MacDonald A., Ahring K. et al. (2017). The complete European guidelines on phenylketonuria: diagnosis and treatment. Orphanet Journal of Rare Diseases, 12(1), 162. doi: 10.1186/s13023-017-0685-2.
Ney D. M., Blank R. D., Hansen K. E. (2014). Advances in the nutritional and pharmacological management of phenylketonuria. Current Opinion in Clinical Nutrition & Metabolic Care, 17(1), 61-8.
Robertson L., Adam S., Ellerton C. et al. (2022). Dietetic Management of Adults with Phenylketonuria (PKU) in the UK: A Care Consensus Document. Nutrients, 14(3), 576. https://doi.org/10.3390/nu14030576
Wiedemann A. et al. (2020). La phénylcétonurie – De la diététique à la thérapie génique. [Phenylketonuria, from diet to gene therapy] [in French]. Med. Sci. (Paris), 36(8-9), 725-734. doi: 10.1051/medsci/2020127.
PKU Golike: the evolution in PKU nutritional management. Retrieved from: https://metahealthcare.co.uk/product-page/
Al Hafid N., Christodoulou J. (2015). Phenylketonuria: a review of current and future treatments. Transl. Pediatr., 4(4), 304-317. doi: 10.3978/j.issn.2224-4336.2015.10.07.
Pharmacy/medical policy 5.01.585 Pharmacologic Treatment of Phenylketonuria.
Pena et al. (2015). Special low protein foods for phenylketonuria: Availability in Europe and in examination of their nutritional profile. Orphanet Journal of Rare Diseases, 10, 162. DOI: 10.1186/s13023-015-0378-7
Wood G., Pinto A., Evans S. (2021). Special Low Protein Foods Prescribed in England for PKU Patients: An Analysis of Prescribing Patterns and Cost. Nutrients, 13 (11), 3977. https://doi.org/10.3390/nu13113977
Postanova KMU «Pro zatverdzhennia Poriadku zabezpechennia hromadian, yaki strazhdaiut na ridkisni (orfanni) zakhvoriuvannia, likarskymy zasobamy ta vidpovidnymy kharchovymy produktamy dlia spetsialnoho diietychnoho spozhyvannia» №160 (31.03.2015). [On the approval of the Procedure for providing citizens suffering from rare (orphan) diseases with medicines and appropriate food products for special dietary consumption]. Retrieved from: https://zakon.rada.gov.ua/laws/show/160-2015-%D0%BF#Text
Pro vporiadkuvannia bezoplatnoho ta pilhovoho vidpusku likarskykh zasobiv za retseptamy likariv u razi ambulatornoho likuvannia okremykh hrup naselennia ta za pevnymy katehoriiamy zakhvoriuvan: postanova KMU vid 17.08.1998 r. №1303 (zi zminamy). [On regulating the free and subsidized supply of medicines according to doctors' prescriptions in the case of outpatient treatment of certain population groups and for certain categories of diseases] Retrieved from: https://zakon.rada.gov.ua/laws/show/1303-98-%D0%BF#Text
nakaz MOZ Ukrainy «Pro zakhody shchodo udoskonalennia medychnoi dopomohy khvorym na fenilketonuriiu v Ukraini» № 457 (13.11.2001) (zi zminamy). [About measures to improve medical care for patients with phenylketonuria in Ukraine]. Retrieved from: https://zakon.rada.gov.ua/rada/show/v0457282-01#Text
nakaz MOZ ta AMN Ukrainy «Pro udoskonalennia medyko-henetychnoi dopomohy v Ukraini» №641/84 (31.12.2003). [On the improvement of medical and genetic care in Ukraine]. Retrieved from: https://zakon.rada.gov.ua/rada/show/va641282-03#Text
nakaz MOZ Ukrainy «Pro zatverdzhennia metodychnykh rekomendatsii z planuvannia ta rozrakhunku potreby v likarskykh zasobakh, produktakh spetsialnoho kharchuvannia ta vyrobakh medychnoho pryznachennia, shcho zakupovuiutsia za rakhunok koshtiv derzhavnoho ta mistsevoho biudzhetiv» №2498 (17.12.2019). [On the approval of methodological recommendations for planning and calculating the need for medicinal products, special food products and medical products purchased from the funds of the state and local budgets]. Retrieved from: https://zakon.rada.gov.ua/rada/show/v2498282-19#Text
nakaz MOZ Ukrainy «Pro zatverdzhennia Norm fiziolohichnykh potreb naselennia Ukrainy v osnovnykh kharchovykh rechovynakh i enerhii» № 1073 (03.09.2017) [On the approval of the norms of physiological needs of the population of Ukraine in basic food substances and energy]. Retrieved from: https://zakon.rada.gov.ua/laws/show/z1206-17#Text
List of notifications of the intention to place on the market baby food, food products for special medical purposes and food products for weight control. Retrieved from: https://dpssukraine-my.sharepoint.com/:x:/g/personal/n_hyrfanova_dpss_gov_ua/EdCSUfw6wjFDvnXXwP9VkmYB8PFXLBWZ0kzAKFsAgKHlNw?rtime=WQCpjgIr20g
Downloads
Published
Issue
Section
License
Copyright (c) 2023 National University of Pharmacy
This work is licensed under a Creative Commons Attribution 4.0 International License.
Authors who publish with this journal agree to the following terms:- Authors retain copyright and grant the journal right of first publication with the work simultaneously licensed under a Creative Commons Attribution License that allows others to share the work with an acknowledgement of the work's authorship and initial publication in this journal.
- Authors are able to enter into separate, additional contractual arrangements for the non-exclusive distribution of the journal's published version of the work (e.g., post it to an institutional repository or publish it in a book), with an acknowledgement of its initial publication in this journal.
- Authors are permitted and encouraged to post their work online (e.g., in institutional repositories or on their website) prior to and during the submission process, as it can lead to productive exchanges, as well as earlier and greater citation of published work (See The Effect of Open Access).
