A review of rare disease policies and orphan drug reimbursement systems in 12 Eurasian countries

M Czech, A Baran-Kooiker, K Atikeler… - Frontiers in public …, 2020 - frontiersin.org
Background: Despite international initiatives on collaboration within the field of rare
diseases, patient access to orphan medicinal products (OMPs) and healthcare services …

Making orphan drugs and services available and accessible for people who live with rare diseases: what has been done? a systematic scoping review

H Ghiasvand, MS Barnish, T Moradi… - Expert Opinion on …, 2022 - Taylor & Francis
Objectives Rare diseases are recognized as non-prevalent health disorders. Availability,
accessibility, and affordability of Orphan Drugs (ODs), alongside genetic testing, are the …

Reimbursement legislations and decision making for orphan drugs in central and eastern European countries

KP Malinowski, P Kawalec, W Trabka… - Frontiers in …, 2019 - frontiersin.org
Background Reimbursement policies influence access of patients to orphan drugs in the
European countries. Objectives To provide a comprehensive description of orphan drug …

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

KP Malinowski, P Kawalec, W Trąbka… - Orphanet journal of rare …, 2020 - Springer
Background The reimbursement of orphan drugs (OD) is an increasingly important for
country policymakers, and still insufficiently understood, especially in Central and Eastern …

Reimbursement status and recommendations related to orphan drugs in European countries

E Stawowczyk, KP Malinowski, P Kawalec… - Frontiers in …, 2019 - frontiersin.org
Objective: To review the reimbursement recommendations issued by selected European
health technology assessment agencies for orphan drugs and the reimbursement status of …

Identifying patient access barriers for tumor necrosis factor alpha inhibitor treatments in rheumatoid arthritis in five central eastern European countries

A Inotai, D Tomek, M Niewada, L Lorenzovici… - Frontiers in …, 2020 - frontiersin.org
Introduction Although there is a significant utilization gap of biologic medicines in the EU,
many studies estimate equity in patient access to biopharmaceuticals only based on their …

Did the introduction of biosimilars influence their prices and utilization? The case of biologic disease modifying antirheumatic drugs (bDMARD) in Bulgaria

K Tachkov, Z Mitkova, V Boyadzieva, G Petrova - Pharmaceuticals, 2021 - mdpi.com
The aim of this study is to evaluate the effect of the introduction of biosimilars in Bulgaria on
the prices and utilization of biologic disease modifying antirheumatic drugs (bDMARD). It is …

Real-world use of orphan medicinal products (OMPs) in rare disease (RD) patients: A population-based registry study

M Mazzucato, C Minichiello, A Vianello… - Frontiers in …, 2022 - frontiersin.org
Background: Despite calls for the use of real-world data, the rare diseases (RD) treatment
landscape suffers from a scarcity of data referred to orphan medicinal products (OMP) use at …

Systematic Literature Review of Access Pathways to Drugs for Patients with Rare Diseases

C Vargas, R De Abreu Lourenco, M Espinoza… - … Health Economics and …, 2024 - Springer
Objective This article reviews the assessment pathways that have been implemented
worldwide to facilitate access to drugs for patients with rare diseases. Methods The …

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

S Jakubowski, P Kawalec, P Holko… - Frontiers in …, 2024 - frontiersin.org
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 …