Analysis of the state and problems of pharmaceutical supply for patients with epidermolysis bullosa

Authors

  • N. A. Sliptsova National University of Pharmacy of the Ministry of Health of Ukraine, Kharkiv, Ukraine
  • V. M. Nazarkina National University of Pharmacy of the Ministry of Health of Ukraine, Kharkiv, Ukraine http://orcid.org/0000-0002-0767-6180

DOI:

https://doi.org/10.24959/sphhcj.23.291

Keywords:

epidermolysis bullosa; rare (orphan) diseases; medicines; medical products

Abstract

Aim. To analyze medical technologies (MT) for the treatment of epidermolysis bullosa (EB) and their availability to patients and the healthcare system.

Materials and methods. The study objects were scientific publications, normative legal acts, guidelines (protocols), statistical data, the Orphanet network, official data of the Ministry of Health and the State Enterprise “Medical Procurement of Ukraine”, marketing information. The methods of content analysis, structural and logical, comparison, generalization, marketing analysis were used.

Results. Modern approaches to the classification of types of EB were studied; epidemiological indicators were analyzed; recommendations of clinical protocols were summarized. It has been found that currently there are no available specific MT in the world for the treatment of this severe chronic disease. International clinical guidelines recommend the use of medicines and medical products to relieve symptoms (wound care, reduce pain and itching), prevent secondary pathologies and improve the quality of life of patients with EB. The cost of treating EB depends on the severity of the disease and the patient’s age. The burden of the disease and the impact on the budget are assessed to be high. The state cannot provide 100 % of the need for the necessary medicines and medical products due to budget constraints. The analysis of the national legal framework for providing patients with rare (orphan) diseases, including EB, with the necessary medicines and medical products was carried out. It has been determined that only 9 INNs of the seven groups of medicines recommended by the protocol are included in the National List and can be purchased for budget funds. The annual need, nomenclature and volumes of purchases of drugs for the treatment of EB were studied. According to the State Enterprise “Medical Procurement of Ukraine” in 2023, for patients with EB it is planned to purchase eight groups of medical products for skin and wound care under 20 names in the amount of UAH 119.8 million.

Conclusions. In order to improve approaches to the pharmaceutical provision of patients with EB, it is extremely important to use health technology assessment (HTA) to identify the most effective and economically feasible medicines and medical products.

References

MOZ. (2016). Nakaz vid 25.02.2016 r. “Unifikovanyi klinichnyi protokol pervynnoi, vtorynnoi (spetsializovanoi) ta tretynnoi (vysokospetsializovanoi) medychnoi dopomohy. Buloznyi epidermoliz”. dec.gov.ua. Available at: https://www.dec.gov.ua/mtd/buloznyj-epidermoliz/.

Buloznyi epidermoliz. Adaptovana klinichna nastanova, zasnovana na dokazakh. (2016). DETs, 77. Available at: https://www.dec.gov.ua/wp-content/uploads/2019/11/2016_135_akn_be.pdf.

KMU. (2021). Rozporiadzhennia vid 28.04.2021 r. No.377-r. “Pro skhvalennia Kontseptsii rozvytku systemy nadannia medychnoi dopomohy patsiientam, yaki strazhdaiut na ridkisni (orfanni) zakhvoriuvannia, na 2021-2026 roky”. zakon.rada.gov.ua. Available at: https://zakon.rada.gov.ua/laws/show/377-2021-%D1%80#Text.

Denyer, J., Pillay, E. (2012). Best practice guidelines for skin and wound care in epidermolysis bullosa. International Consensus. DEBRA. Available at: https://wounds-uk.com/consensus-documents/best-practice-guidelines-skin-and-wound-care-epidermolysis-bullosa/.

Orphanet Report Series ‒ Prevalence of rare diseases: Bibliographic data – January 2022. Available at: https://www.orpha.net/orphacom/cahiers/docs/GB/Prevalence_of_rare_diseases_by_alphabetical_list.pdf.

Orphanet Nomenclature Pack For Coding. Available at: https://www.orphadata.com/pack-nomenclature/.

So, J. Y., Teng, J. (1998). Epidermolysis Bullosa Simplex. GeneReviews. Available at: https://www.ncbi.nlm.nih.gov/books/NBK1369/.

Rashidghamat, E., Mellerio, J. E. (2017). Management of chronic wounds in patients with dystrophic epidermolysis bullosa: challenges and solutions. Chronic Wound Care Management and Research, 4, 45–54.

Denier, Zh., Pillai, E., Klapem, Dzh. (2017). Nastanovy z naikrashchoi praktyky z dohliadu za shkiroiu ta ranamy pry buloznomu epidermolizi izhnarodnyi konsensus. Wounds International.

Bader, K. (2023). B-VEC Approved for the Treatment of Dystrophic Epidermolysis Bullosa. Dermatologytimes. Available at: https://www.dermatologytimes.com/view/b-vec-approved-for-the-treatment-of-dystrophic-epidermolysis-bullosa.

Schräder, N. H. B., Korte, E. W. H., Duipmans, J. C., Stewart, R. E., Bolling, M. C., Wolff, A. P. (2021). Identifying Epidermolysis Bullosa Patient Needs and Perceived Treatment Benefits: An Explorative Study Using the Patient Benefit Index. Journal of Clinical Medicine, 10 (24), 5836. doi: 10.3390/jcm10245836.

Brun, J., Devos, C., Leclerc-Mercier, S., Mazereeuw, J., Bourrat, E., Maruani, A. et al. (2017). Pain and quality of life evaluation in patients with localized epidermolysis bullosa simplex. Orphanet Journal of Rare Diseases, 12, 119.

Chan, J. M., King, A., Maksomski, S. et al. (2019). Occupational therapy for epidermolysis bullosa: clinical practice guidelines. Orphanet Journal of Rare Diseases, 14, 129. doi: https://doi.org/10.1186/s13023-019-1059-8.

Angelis, A., Kanavos, P., López-Bastida, J., Linertova, R., Oliva-Moreno, J., Serrano-Aguilar, P. et al. (2016). Social-economic costs and health-related quality of life in patients with epidermolysis bullosa in Europe. The European Journal of Health Economics, 7, 31–42.

Flannery, D., Doyle, C., Hickey, S., Aherne, F., Kennan, A. (2020). Direct Costs of Epidermolysis Bullosa by Disease Severity. Acta Dermato-Venereologica, 100 (8), 5715. doi: 10.2340/10.2340/00015555-3456.

Pillay, E., Clapham, J. (2018). Development of best clinical practice guidelines for epidermolysis bullosa. Wounds International, 9 (4). Available at: https://www.woundsme.com/uploads/resources/83e41a6d33fa875ba1efbf1ade2f1230.pdf.

Feinstein, J. A., Bruckner, A. L., Chastek, B. et al. (2022). Clinical characteristics, healthcare use, and annual costs among patients with dystrophic epidermolysis bullosa. Orphanet Journal of Rare Diseases, 17, 367. doi: https://doi.org/10.1186/s13023-022-02509-0.

Downloads

Published

2023-09-06

Issue

Vol. 9 No. 2 (2023)

Section

Social marketing and pharmacoeconomic research

License

Copyright (c) 2023 National University of Pharmacy

Creative Commons License

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

Authors who publish with this journal agree to the following terms:

  1. 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.
  2. 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.
  3. 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).