Availability and Accessibility of Orphan Medicinal Products to Patients in Slovakia in the Years 2010-2019

Tatiana Foltanova; Alan Majernik; Eva Malikova; Stanislava Kosirova

doi:10.3389/fphar.2022.768325

Availability and Accessibility of Orphan Medicinal Products to Patients in Slovakia in the Years 2010-2019

Front Pharmacol. 2022 Jan 26:13:768325. doi: 10.3389/fphar.2022.768325. eCollection 2022.

Authors

Tatiana Foltanova^{1

2}, Alan Majernik², Eva Malikova^{1

3}, Stanislava Kosirova¹

Affiliations

¹ Department of Pharmacology and Toxicology, Comenius University in Bratislava, Bratislava, Slovakia.
² Slovak Alliance for Rare Diseases, Pezinok, Slovakia.
³ State Institute for Drug Control, Bratislava, Slovakia.

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.

Keywords: data-based decision-making; legislation—EEC; orphan medicinal product; patient access to medicines; rare diseases.