Clinical aspects of reimbursement policies for orphan drugs in Central and Eastern European countries.

Autor:	Jakubowski S; Department of Health Promotion and e-Health, Faculty of Health of Science, Jagiellonian University Medical College, Kraków, Poland., Kawalec P; Department of Nutrition and Drug Research, Faculty of Health of Science, Jagiellonian University Medical College, Kraków, Poland., Holko P; Department of Nutrition and Drug Research, Faculty of Health of Science, Jagiellonian University Medical College, Kraków, Poland., Kowalska-Bobko I; Health Policy and Management Department, Faculty of Health of Science, Jagiellonian University Medical College, Kraków, Poland., Kamusheva M; Department of Organization and Economics of Pharmacy, Faculty of Pharmacy, Medical University of Sofia, Sofia, Bulgaria., Petrova G; Department of Organization and Economics of Pharmacy, Faculty of Pharmacy, Medical University of Sofia, Sofia, Bulgaria., Draganić P; Agency for Medicinal Products and Medical Devices of Croatia, Zagreb, Croatia.; Department of Biotechnology, University of Rijeka, Rijeka, Croatia., Fuksa L; Department of Social and Clinical Pharmacy, Faculty of Pharmacy in Hradec Kralove, Charles University in Prague, Prague, Czechia., Männik A; Institute of Family Medicine and Public Health, University of Tartu, Tartu, Estonia., Ispán F; Department of Reimbursement, National Institute of Health Insurance Fund Management, Budapest, Hungary., Briedis V; Department of Clinical Pharmacy of Lithuanian, University of Health Sciences, Kaunas, Lithuania., Bianchi I; The Romanian Association of International Medicine Manufacturers. ARPIM, Bucharest, Romania., Paveliu MS; Faculty of Medicine, Titu Maiorescu University, Bucharest, Romania., Tesar T; Department of Organisation and Management in Pharmacy, Faculty of Pharmacy, Comenius University in Bratislava, Bratislava, Slovakia.
Jazyk:	angličtina
Zdroj:	Frontiers in pharmacology [Front Pharmacol] 2024 Mar 08; Vol. 15, pp. 1369178. Date of Electronic Publication: 2024 Mar 08 (Print Publication: 2024).
DOI:	10.3389/fphar.2024.1369178
Abstrakt:	Objectives: The aim of this study was to characterize the reimbursement policy for orphan drugs (ODs) in Central and Eastern European (CEE) countries in relation to the availability and impact of clinical evidence, health technology assessment (HTA) procedure, selected economic indicators, and the drug type according to indications. Materials and methods: A list of authorized medicines with orphan designation and information about active substance, Anatomical Therapeutic Chemical (ATC) classification, and therapeutic area was extracted from the web-based register of the European Medicines Agency (EMA). A country-based questionnaire survey was performed between September 2021 and January 2022 in a group of selected experts from nine CEE countries (an invitation was sent to 11 countries). A descriptive and statistical analysis was conducted to determine statistical significance, correlations, between the drug or country characteristic and the positive recommendation or reimbursement of ODs . Results: The proportion of reimbursed orphan drugs differed between countries, ranging from 17.7% in Estonia to 49.6% in Hungary ( p < 0.001). The odds that ODs were reimbursed were reduced in countries with a "strong" level of impact of drug safety and efficacy on reimbursement decisions ( p =0.018), the presence of other additional specific clinical aspects (e.g., genomic data) considered in the reimbursement decision ( p < 0.001) and mandatory (without exception) safety assessments ( p =0.004). The probability that ODs were reimbursed was increased in countries with a "moderate" level of impact of drug safety and efficacy on reimbursement decisions ( p =0.018), when reimbursement decisions are dependent on the EMA registration status and orphan drug designation ( p < 0.001), the presence of the "positive HTA recommendation guarantees reimbursement" policy ( p < 0.001), higher GDP per inhabitant ( p =0.003), and higher healthcare expenditure ( p < 0.001). Conclusion: We found that there are differences among CEE countries in the reimbursement of orphan drugs, and we identified aspects that may influence these differences. Safety, efficacy, and specific clinical aspect issues significantly influenced reimbursement decisions. Antineoplastic and immunomodulating agents drugs were the largest group of ODs and increased the chance of getting a positive recommendation. The higher GDP per inhabitant and healthcare expenditures per inhabitant were positively linked to the chance that an OD receives reimbursement. Competing Interests: The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest. The author(s) declared that they were an editorial board member of Frontiers, at the time of submission. This had no impact on the peer review process and the final decision. (Copyright © 2024 Jakubowski, Kawalec, Holko, Kowalska-Bobko, Kamusheva, Petrova, Draganić, Fuksa, Männik, Ispán, Briedis, Bianchi, Paveliu and Tesar.)
Databáze:	MEDLINE
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