The analysis of the legislative framework regarding pharmaceutical provision of orphan patients in Ukraine

Abstract

A rare (orphan) disease is a disease that threatens a person’s life or chronically progresses; it leads to a reduction in life expectancy or disability, which prevalence among the population is not more than 1 : 2000 in Ukraine and not more than 1 : 5000-8000 in other countries. Aim. To analyze the current legal framework on pharmaceutical provision of orphan patients in Ukraine. Materials and methods. The current regulatory framework on pharmaceutical provision of orphan patients in Ukraine was analyzed by constructing and studying the hierarchy of legislative acts regulating the issues of management of orphan (rare) diseases in the world and in Ukraine. Results and discussion. The article describes the main stages of the legislation formation, starting from the USA in 1983 to the EU and Ukraine in 2021. The practical value of the article consists in the current list of normative acts regulating the provision of patients with orphan diseases. The scientific significance is in the use of the results of previous studies of others domestic researchers (2016). The analysis of the legislation and the main issues of providing orphan patients allowed us to formulate the basic problems of the national healthcare system listed in the article. Conclusions. The current state of the legal regulation of orphan patients in Ukraine has been studied. It has been found that the legislative regulation of the issue under study has been actively carried out over the past 7 years by implementing the adopted Law of Ukraine, resolutions of the Cabinet of Ministers and orders of the Ministry ofHealth. The results of the study of international experience in introducing the basic concepts of orphan diseases and orphan patients into the healthcare system in the United States (since 1983), EU countries (since 1999), Japan (since 1993), etc., have been summarized. It has been found that 275 nosologies of orphan diseases have been approved in Ukraine; they are grouped by 11 nosological categories, there are 104 nosologies (+40 %) more than in 2016. A positive trend towards intensification of the process of improving the provision of orphan patients in Ukraine has been revealed.