Analysis of the economic burden of epidermolisis bulous and the availability of technologies for its treatment

Epidermolysis bullosa (EB) is a group of rare diseases of the skin and mucous membranes that leads to the formation of blisters and erosions. The incidence of EB in the world is 17 cases per 1 million newborns. According to the DEBRA Ukraine Center, a public organization of patients with EB, there are about 300 such patients in Ukraine, 199 of them are pediatric patients and 120 require intensive treatment. Nowadays, the disease is classified as incurable, and the available technologies for treating EB are focused on symptomatic treatment (wound care, pain relief, etc.). The purpose of the study is to analyze the economic burden of EB and identify ways to increase the availability of technologies for its treatment. The objects of the study were scientific publications, guidelines (protocols), statistics, Orphanet, official data of the Ministry of Health and the State Enterprise «Medical Procurement of Ukraine», and data from the official websites of authorized bodies. We used content analysis, comparison, generalization and marketing analysis. The analysis of regulatory documents and information materials on the organization of medical care and pharmaceutical support for patients with EB in Ukraine and other countries was conducted. The data on the economic burden of epidermolysis bullosa in the world were analyzed and summarized. It was concluded that non-medical and indirect medical costs occupy the predominant share in the structure of the cost of treating EB. These types of costs are difficult to compare, as each country has its own health care system and, in particular, reimbursement plan for orphan patients. At the same time, the rare and clinically heterogeneous nature of the disease itself is a limiting factor in conducting a comparative analysis. It has been proved that the cost of wound care (medicines and special dressings) also greatly impacts the financial situation of patients with EB and their families, as they are usually reimbursed selectively. In Ukraine, the situation with the provision of pharmaceuticals for patients with serious systemic skin diseases, including EB, requires regulatory and legal regulation, in particular, revision of the procurement nomenclature and changes in approaches to pharmaceutical provision (including the provision of free prescriptions), taking into account the specifics of the disease.