Preview

Kachestvennaya Klinicheskaya Praktika = Good Clinical Practice

Advanced search

Pharmacoeconomic analysis of therapy for dystrophic and junctional types of inherited epidermolysis bullosa

https://doi.org/10.37489/2588-0519-GCP-0036

EDN: JFZIWQ

Abstract

Relevance. Inherited epidermolysis bullosa (EB) is a severe, chronic, systemic rare disease requiring lifelong therapy. In the Russian Federation, the unregistered drug beremagene geperpavec is used for the dystrophic type of EB. In 2025, a new drug Filsuvez® — a triterpene complex in gel formulation for topical application — was registered in the Russian Federation for both dystrophic and junctional types of EB.

Objective. To compare budget impact of using the triterpene complex versus beremagene geperpavec in patients with EB in the Russian Federation.

Methods. A budget impact analysis was performed from the healthcare perspective. Costs were based on the planned price of the triterpene complex and state procurement prices for beremagene geperpavec. Dressing costs were included. The patient population (patients under 18 years of age) was modelled using data from the Russian EB registry. Time horizon 3 years.

Results. The budget impact analysis demonstrates a reduction in treatment costs of up to 14.5-fold when using the triterpene complex in comparison with beremagene geperpavec. In scenario expecting 30 % of patients with dystrophic EB receive triterpene complex therapy, the total savings over 3 years amount is to 17.0 billion RUB, at the same time both patients with dystrophic EB and additionally, due to cost saving, patients with junctional EB receive treatment.

Conclusion. The use of the triterpene complex for dystrophic and junctional types of EB is pharmacoeconomically justified, significantly reduces the budget burden, and expands access to pathogenetic therapy also for patients with junctional EB.

About the Authors

S. K. Zyryanov
Patrice Lumumba Peoples' Friendship University of Russia; City Clinical Hospital No. 24 of the Moscow Department of Health
Russian Federation

Sergey K. Zyryanov — Dr. Sci. (Med.), Professor, Head of the Department of General and Clinical Pharmacology, RUDN University; Deputy Chief physician GKB No. 24 DZM

Moscow


Competing Interests:

This publication was prepared with support from Chiesi Pharmaceuticals LLC. All stages of study design, data analysis and interpretation, manuscript writing, and the decision to publish were the sole responsibility of the authors. The authors bear full responsibility for the content of the article. There are no other conflicts of interest.



I. N. Dyakov
Scientific and Practical Center for the Study of Rational Pharmacotherapy and Pharmacoeconomics; Mechnikov Research Institute of Vaccines and Sera; Gamaleya National Research Center of Epidemiology and Microbiology
Russian Federation

Ilya N. Dyakov — Cand. Sci. (Biol.); leading research fellow, head Laboratory of Immunoglobulin Biosynthesis of the Department of Immunology and Allergology; researcher, Laboratory of Bacterial Genetics, Department of Medical Microbiology

Moscow


Competing Interests:

This publication was prepared with support from Chiesi Pharmaceuticals LLC. All stages of study design, data analysis and interpretation, manuscript writing, and the decision to publish were the sole responsibility of the authors. The authors bear full responsibility for the content of the article. There are no other conflicts of interest.



References

1. Врожденный буллезный эпидермолиз. Клинические рекомендации. Общероссийская общественная организация «Российское общество дерматовенерологов и косметологов», Общероссийская общественная организация «Союз педиатров России». 2020 [Congenital Epidermolysis Bullosa. Clinical Guidelines. All-Russian Public Organization "Russian Society of Dermatovenerologists and Cosmetologists," All-Russian Public Organization "Union of Pediatricians of Russia." 2020 (In Russ.)].

2. Has C, Nyström A, Saeidian AH, et al. Epidermolysis bullosa: Molecular pathology of connective tissue components in the cutaneous basement membrane zone. Matrix Biology : Journal of the International Society for Matrix Biology. 2018 Oct;71-72:313-329. DOI: 10.1016/j.matbio.2018.04.001.

3. Uitto J, Bruckner-Tuderman L, McGrath JA, et al. EB2017-Progress in Epidermolysis Bullosa Research toward Treatment and Cure. J Invest Dermatol. 2018 May;138(5):1010-1016. doi: 10.1016/j.jid.2017.12.016.

4. Кубанов АА, Альбанова ВИ, Чикин ВВ, Епишев РВ. Современные методы терапии врожденного буллезного эпидермолиза. Вестник дерматологии и венерологии. 2014;90(6):47-56. doi: 10.25208/0042-4609-2014-90-6-47-56 [Kubanov AA, Albanova VI, Chikin VV, Yepishev RV. Modern methods of the treatment of hereditary epidermolysis bullosa. Vestnik dermatologii i venerologii. 2014;90(6):47-56. (In Russ.)].

5. El Hachem M, Zambruno G, Bourdon-Lanoy E, et al. Multicentre consensus recommendations for skin care in inherited epidermolysis bullosa. Orphanet J Rare Dis. 2014 May 20;9:76. doi: 10.1186/1750-1172-9-76.

6. Кубанов АА, Альбанова ВИ, Чикин ВВ, Епишев РВ. Современные методы терапии врожденного буллезного эпидермолиза. Вестник дерматологии и венерологии. 2014;90(6):47-56. doi: 10.25208/0042-4609-2014-90-6-47-56 [Kubanov AA, Albanova VI, Chikin VV, Yepishev RV. Modern methods of the treatment of hereditary epidermolysis bullosa. Vestnik dermatologii i venerologii. 2014;90(6):47-56. (In Russ.)].

7. Pope E, Lara-Corrales I, Mellerio J, et al. A consensus approach to wound care in epidermolysis bullosa. J Am Acad Dermatol. 2012 Nov;67(5):904-17. doi: 10.1016/j.jaad.2012.01.016.

8. Официальная страница препарата беремаген геперпавек. Режим доступа: https://www.vyjuvek.com/ [Дата обращения: 13.08.2026] [Official page of the drug Beremagene Geperpavec.].

9. Kern JS, Sprecher E, Fernandez MF, et al; EASE investigators. Efficacy and safety of Oleogel-S10 (birch triterpenes) for epidermolysis bullosa: results from the phase III randomized double-blind phase of the EASE study. Br J Dermatol. 2023 Jan 23;188(1):12-21. doi: 10.1093/bjd/ljac001.

10. Общая характеристика лекарственного препарата Фильзувес® ЛП-№(012364)-(РГ-RU) от 07.11.2025. Режим доступа: https://grls.rosminzdrav.ru/GRls_View_v2.aspx?routingGuid=33e0fb89-6744-4f27-8944-010ae9c4740a. [Дата обращения: 20.03.2026] [Summary of Product Characteristics (SmPC) of the drug Filzuves].

11. База данных MedLine. Режим доступа: https://pubmed.ncbi.nlm.nih.gov. [MedLine Database]

12. VYJUVEK™ (beremagene geperpavec-svdt) biological suspension mixed with excipient gel for topical application Initial U.S. Approval: 2023

13. Guide SV, Gonzalez ME, Bağcı IS, et al. Trial of Beremagene Geperpavec (B-VEC) for Dystrophic Epidermolysis Bullosa. N Engl J Med. 2022 Dec 15;387(24):2211-2219. doi: 10.1056/NEJMoa2206663.

14. Мурашкин Н.Н., Епишев Р.В., Орлова О.С., и др. Эпидемиология врожденного буллезного эпидермолиза среди детского населения Российской Федерации. Вопросы современной педиатрии. 2024;23(5):316-328. doi: 10.15690/vsp.v23i5.2808 [Murashkin N.N., Epishev R.V., Orlova O.S., et al. Congenital Epidermolysis Bullosa Epidemiology among Children of Russian Federation. Current Pediatrics. 2024;23(5):316-328. (In Russ.)].

15. Фонд «Круг добра». Буллезный эпидермолиз [Интернет]. https://фондкругдобра.рф. Дата обращения: 14.08.2026. [Circle of Goodness Foundation. Epidermolysis bullosa. (In Russ.)].

16. Румянцев А.Г., Куцев С.И., Намазова-Баранова Л.С. и др. Новые патогенетические подходы к лечению ран при врожденном буллезном эпидермолизе. Клиническая дерматология и венерология. 2026;24(1):104-111. doi: 10.17116/klinderma202625011104. [Rumyantsev AG, Kutsev SI, Namazova-Baranova LS, et al. New pathogenetic approaches to the treatment of wounds in congenital epidermolysis bullosa. Russian Journal of Clinical Dermatology and Venereology. 2026;24(1):104-111. (In Russ.)].

17. Национальный клинический протокол Диагностика и лечение по нозологии «Врожденный буллезный эпидермолиз». 29 марта 2024 года. Узбекистан. Режим доступа: https://clc.li/iDqZi [National Clinical Protocol for Diagnosis and Treatment of Congenital Epidermolysis Bullosa. March 29, 2024. Uzbekistan.].

18. Азэрбайхан Республикасы Зыйуйээ Назирлиунин Коллегиуасынн 2017- ci il 27 iyul tarixli 34 номрли горари илэ тэсдиг едилмиз “Nadir hallarda rast голинан вэ spesifik мuлайсис тэлэб едэн хэстэлйклэрин мuлайсиси цунн нэээрдэ тultural дэрман вазитэлэринни зиуаһиш”нда дэуйэйклйк едилмэсиi барэдэ. “5” fevral 2025-ci il. Режим доступа: https://e-qanun.az/framework/59018.

19. FİLSUVEZ. PRESCRIBING INFORMATION. Режим доступа: https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/215064s000lbl.pdf.

20. Filsuvez: EPAR - Product Information. Режим доступа: https://www.ema.europa.eu/en/medicines/human/EPAR/filsuvez.

21. NICE. Режим доступа: https://www.nice.org.uk/news/articles/nice-recommends-novel-treatment-for-debilitating-inherited-skin-condition.

22. Врожденный буллезный эпидермолиз. Рубрикатор клинических рекомендаций Минздрава РФ. Режим доступа: https://cr.minzdrav.gov.ru/preview-cr/1045_1 [Congenital epidermolysis bullosa. Clinical guidelines of the Russian Ministry of Health.].

23. Данные Единой информационной системы в сфере закупок. Режим доступа: https://zakupki.gov.ru/epz/order/notice/zk20/view/common-info.html?regNumber=0873200009826004122 [Data from the Unified Information System in the Sphere of Procurement. (In Russ.)].

24. Данные Единой информационной системы в сфере закупок. Режим доступа: https://zakupki.gov.ru/epz/order/notice/zk20/view/common-info.html?regNumber=0119200000126004199 [Data from the Unified Information System in the Sphere of Procurement. (In Russ.)].


Review

For citations:


Zyryanov S.K., Dyakov I.N. Pharmacoeconomic analysis of therapy for dystrophic and junctional types of inherited epidermolysis bullosa. Kachestvennaya Klinicheskaya Praktika = Good Clinical Practice. 2026;(3):4-15. (In Russ.) https://doi.org/10.37489/2588-0519-GCP-0036. EDN: JFZIWQ

Views: 53

JATS XML


Creative Commons License
This work is licensed under a Creative Commons Attribution 4.0 License.


ISSN 2588-0519 (Print)
ISSN 2618-8473 (Online)