Abstract
Objective: Information about the access of Slovak patients to orphan medicinal products (OMPs) in the literature is rather scarce. The main aim of the study was to analyze the accessibility and availability of OMPs to Slovak patients in the years 2010–2019. Methods: The analyzed OMPs were strictly defined according to the European definition. The date of marketing authorization together with its first appearance in the positive drug list was used to count the time to reach the national market. The data from the National Health Information Centre, the Ministry of Health, and health insurance companies were used as data sources of drug usage, expenditure, consumption, reimbursement of OMPs, as well as the total number of treated patients. Results: Out of the 167 OMPs on the European market, we identified 52% (87) OMPs which had any kind of costs recorded in Slovakia. Out of them, 62% (54) OMPs were directly present on the positive drug list. The remaining 33 OMPs were available on exception. The trend in accessibility and availability of OMPs in Slovakia between the years 2010 and 2019 was decreasing (57% OMPs in 2010 vs. 47% OMPs in 2019). The average time for an orphan medicinal product to reach the Slovak market was almost 4 years, 43.5 months [6—202 months]. Together, 10.4% (8 815 patients) out of the theoretical patients’ estimation according to the prevalence in the orphan designation were treated with OMPs available in Slovakia. Conclusion: Presented data clearly show insufficient accessibility and availability of OMPs in Slovakia. Importance of clearly defined criteria for OMPs supporting patients and healthcare professionals’ involvement in the final decision together with other measures such as social impact, improvement of patients’ quality of life, society wide meaning, or no alternative treatment in the final decision is crucial for transparent and sustainable access to OMPs and innovative treatments in Slovakia.
Highlights
The Slovak Republic (SR) is a small Central European country, with a total number of inhabitants of about 5.5 million
21 orphan medicinal products (OMPs) were on the positive drug list before the changes of the reimbursement act in 2011; 34 other OMPs were added to the positive drug list in the period 2011—2019
During the years 2010–2019, every year on average, 11.3 [4–22] OMPs were authorized in Europe annually; the average of new OMPs accepted in the positive drug list in Slovakia every year was less than half of the European average 4.3 [0 -11]
Summary
The Slovak Republic (SR) is a small Central European country, with a total number of inhabitants of about 5.5 million. Slovak patients cannot afford to pay the difference in costs between the services in Slovakia and most European countries. They must use an alternative way to get access to health care abroad, which is an administrative burden and time-consuming for all stakeholders, it is covered by the health fund. This negatively impacts Slovak patients, especially rare disease patients who lack many services for their rare disease at the national level (Wilson et al, 2020)
Sign-in/Register to view highlights & summary Sign-in/Register to access full text options 
Free) 
Talk to us
Join us for a 30 min session where you can share your feedback and ask us any queries you have
Schedule a call
