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

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.