Health technology assessment and reimbursement policy for oncology orphan drugs in Central and Eastern Europe

被引：14

作者：

Malinowski, Krzysztof Piotr ^{[1
]}

Kawalec, Pawel ^{[1
]}

Trabka, Wojciech ^{[2
]}

Sowada, Christoph ^{[1
]}

Petrova, Guenka ^{[3
]}

Manova, Manoela ^{[3
,4
]}

Savova, Alexandra ^{[3
,4
]}

Draganic, Pero ^{[5
,6
]}

Slaby, Juraj ^{[7
]}

Mannik, Agnes ^{[8
]}

Marky, Kristof ^{[9
]}

Rugaja, Zinta ^{[10
]}

Gulbinovic, Jolanta ^{[11
]}

Tesar, Tomas ^{[12
]}

Paveliu, Marian Sorin ^{[13
]}

机构：

[1] Jagiellonian Univ, Fac Hlth Sci, Inst Publ Hlth, Med Coll, Ul Grzegorzecka 20, PL-31531 Krakow, Poland

[2] Andrzej Frycz Modrzewski Krakow Univ, Fac Med & Hlth Sci, Bioinformat & Publ Hlth Dept, Krakow, Poland

[3] Med Univ Sofia, Fac Pharm, Sofia, Bulgaria

[4] Natl Council Prices & Reimbursement Med Prod, Sofia, Bulgaria

[5] Agcy Med Prod & Med Devices Croatia, Zagreb, Croatia

[6] Univ Rijeka, Dept Biotechnol, Rijeka, Croatia

[7] State Inst Drug Control, Sect Pricing & Reimbursement Regulat, Prague, Czech Republic

[8] Univ Tartu, Inst Family Med & Publ Hlth, Tartu, Estonia

[9] Natl Inst Hlth Insurance Fund Management, Budapest, Hungary

[10] Natl Hlth Serv, Riga, Latvia

[11] Vilnius Univ, Fac Med, Inst Biomed Sci, Dept Pathol Forens Med & Pharmacol, Vilnius, Lithuania

[12] Comenius Univ, Fac Pharm, Dept Org & Management Pharm, Bratislava, Slovakia

[13] Titu Maiorescu Univ, Bucharest, Romania

来源：

ORPHANET JOURNAL OF RARE DISEASES | 2020年 / 15卷 / 01期

关键词：

Orphan drugs; Oncology; Reimbursement; Health technology assessment; Policy; Central and Eastern Europe;

D O I：

10.1186/s13023-020-01556-9

中图分类号：

Q3 [遗传学];

学科分类号：

071007 ; 090102 ;

摘要：

BackgroundThe reimbursement of orphan drugs (OD) is an increasingly important for country policymakers, and still insufficiently understood, especially in Central and Eastern Europe. The aim of this research was to provide a comprehensive description of country-specific health technology assessment (HTA) policies as well as evaluate the percentage of HTA recommendations and reimbursement decisions for oncology OD. In addition, the study was designed to elucidate the impact of reimbursement of these drugs on the public budget and the agreement between HTA recommendations and reimbursement decisions in the analysed countries. A questionnaire survey was used to collect data on the reimbursement status, HTA recommendation, marketing authorisation, and public expenses on reimbursement in 2014, 2015, and 2016 for all oncology drugs with an orphan designation by the European Medicine Agency in 2017 in Bulgaria, Croatia, Czechia, Estonia, Hungary, Latvia, Lithuania, Poland, Romania, and Slovakia. The agreement between the HTA recommendation and reimbursement status was assessed using the kappa coefficient. The Pearson's correlation was used to analyse the relationship between gross domestic product (GDP) and GDP per capita and reimbursement expenses.ResultsA total of 36 drugs were analysed (25% conditionally approved; 5.56% approved under exceptional circumstances). The share of reimbursed drugs ranged from 11.11% in Latvia to 41.67% in Poland. The highest share of positive recommendations was observed for Bulgaria and Estonia (36.11%), and the lowest, for Latvia (11.11%). The agreement varied from 0.4 for Poland to 1 for Latvia, Hungary, and Slovakia. Expenses were correlated with GDP (0.95 [0.81-0.99]), and not with GDP per capita (0.54 [-0.136 to 0.873]). Expenses per capita were not correlated with GDP per capita (0.52 [-0.15 to 0.87]).ConclusionsIn Hungary, Latvia, and Slovakia, a positive recommendation was associated with a reimbursement, and a negative one, with the lack of reimbursement. The reimbursement of oncology OD is associated with a growing burden for public budget, and the expenses are correlated with the total GDP. The highest share of drugs with any recommendation was observed in Poland, and the lowest, in Latvia and Romania. The share of reimbursed drugs was the lowest in Latvia and the highest in Poland.

引用

页数：13