Reimbursement Legislations and Decision Making for Orphan Drugs in Central and Eastern European Countries

Malinowski, Krzysztof Piotr; Kawalec, Paweł; Trąbka, Wojciech; Czech, Marcin; Petrova, Guenka; Manova, Manoela; Savova, Alexandra; Draganić, Pero; Vostalová, Lenka; Slabý, Juraj; Männik, Andres; Márky, Kristóf; Rugaja, Zinta; Gulbinovič, Jolanta; Tesař, Tomáš; Paveliu, Marian Sorin

doi:10.3389/fphar.2019.00487

Cited by 24 publications

(29 citation statements)

References 14 publications

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“…(41), Turkey 6 (40, 42) 10 . Armenia 4 , Kazakhstan 11 (43), Latvia and Romania 12 (44,45), only screen for phenylketonuria and congenital hypothyroidism. England, Scotland, and Wales screen for nine diseases, whereas Northern Ireland (part of the UK as well) screens only for 5 (29) 13,14 .…”

Section: Newborn Screeningmentioning

confidence: 99%

“…England, Scotland, and Wales screen for nine diseases, whereas Northern Ireland (part of the UK as well) screens only for 5 (29) 13,14 . In several countries the number of screened diseases is being expanded or planned to 10 https://dosyaism.saglik.gov.tr/Eklenti/11173,259822214447pdf.pdf?0 (accessed be expanded, notably in Turkey (going from 6 to 10 screened diseases) 11 and the Netherlands (from 20 to 32) (37), but without specific timelines.…”

Section: Newborn Screeningmentioning

confidence: 99%

“…Poland reimburses 48, the vast majority of which within so-called "Drug Programs" (DPs), introduced by the MoH in 2012 for expensive medical technologies (replacing previous "therapeutical programs") (11,(70)(71)(72). DPs are mainly designed to control consumption of the most expensive drugs 22 .…”

Section: Rare Disease Policies and Access To Orphan Drugsmentioning

confidence: 99%

“…Rare disease policies are a high focus area, given the medical need surrounding rare diseases and the relatively large impact these diseases and their treatment potentially have on healthcare budgets. The reimbursement status of orphan drugs in Eastern Europe has been described by several authors recently (11)(12)(13)(14)(15). There have been multiple publications describing OMP policies in Central and Eastern Europe in single countries (16)(17)(18) or covering a larger number of countries in Europe (5,(19)(20)(21)(22).…”

Section: Introductionmentioning

confidence: 99%

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A Review of Rare Disease Policies and Orphan Drug Reimbursement Systems in 12 Eurasian Countries

Czech¹,

Baran-Kooiker

Atikeler

et al. 2020

Front. Public Health

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Section: Newborn Screeningmentioning

confidence: 99%

Section: Newborn Screeningmentioning

confidence: 99%

Section: Rare Disease Policies and Access To Orphan Drugsmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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A Review of Rare Disease Policies and Orphan Drug Reimbursement Systems in 12 Eurasian Countries

Czech¹,

Baran-Kooiker

Atikeler

et al. 2020

Front. Public Health

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“…Total expenditures on the reimbursement of analysed drugs were highly correlated with the total GDP in all . No correlation between GDP and the share of reimbursed orphan drugs was observed for analysed countries [12].…”

Section: Public Payer Expenses For Reimbursement Of Analysed Orphan Dmentioning

confidence: 81%

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

Malinowski

Kawalec

Trąbka

et al. 2020

Orphanet J Rare Dis

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Background The 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. Results A 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]). Conclusions In 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.

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Mapping challenges in the accessibility of treatment products for urea cycle disorders: A survey of European healthcare professionals

Stolwijk,

Häberle,

Huidekoper

et al. 2024

J of Inher Metab Disea

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Current management guidelines for urea cycle disorders (UCDs) offer clear strategies, incorporating both authorized and non‐authorized medicinal products (including intravenous formulations and products regulated as food). These varying product categories are subject to specific accessibility challenges related to availability, reimbursement, and pricing. The aim of this study is to identify potential obstacles to optimal UCD treatment implementation in European clinical practice. A survey aimed at metabolic healthcare professionals (HCPs) managing patients with UCDs in Europe was disseminated through the European Reference Network for Hereditary Metabolic Disorders and the European registry and network for intoxication type metabolic diseases. Forty‐eight survey responses were collected from 21 European countries. In 16 of these countries, at least one metabolic HCP reported challenges in accessing UCD products. Reimbursement issues were reported for most products (8/10), including both authorized and non‐authorized products. Availability‐related challenges were also reported for 8/10 products, although unavailability was limited to non‐authorized products. Prices impacted accessibility for all authorized products (3/3) and one non‐authorized IV product. The accessibility of UCD treatment products varied across Europe, although no clear regional variations could be discerned. Survey data revealed that metabolic HCPs experience challenges in accessing both authorized and non‐authorized products for UCD management in the majority of European countries. This indicates that registering unauthorized products may not resolve all issues. Improved reimbursement policies and fair pricing models, as well as (adjusted) authorization procedures may help address these concerns, thereby optimizing treatment access for UCD patients.

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Reimbursement Legislations and Decision Making for Orphan Drugs in Central and Eastern European Countries

Cited by 24 publications

References 14 publications

A Review of Rare Disease Policies and Orphan Drug Reimbursement Systems in 12 Eurasian Countries

A Review of Rare Disease Policies and Orphan Drug Reimbursement Systems in 12 Eurasian Countries

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

Mapping challenges in the accessibility of treatment products for urea cycle disorders: A survey of European healthcare professionals

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