ArticleFrontiers in pharmacology2019
Reimbursement Legislations and Decision Making for Orphan Drugs in Central and Eastern European Countries.
Article in Frontiers in pharmacology, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers, 1 of them a synthesis that pooled it.
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Who cites it
18 citing papers in PubMed, 1 synthesis or guideline pooled it.
- A Review of Rare Disease Policies and Orphan Drug Reimbursement Systems in 12 Eurasian Countries.Frontiers in public health · 2019Pooled it
- Evolution and synergy of healthcare security policies for rare diseases in China: a quantitative content analysis.Frontiers in public health · 2026Article
- Disparities in access to systemic therapies for patients with hepatocellular carcinoma: an analysis from the International Liver Cancer Association.The Lancet regional health. Europe · 2025Review
- Mapping challenges in the accessibility of treatment products for urea cycle disorders: A survey of European healthcare professionals.Journal of inherited metabolic disease · 2025Article
- Clinical aspects of reimbursement policies for orphan drugs in Central and Eastern European countries.Frontiers in pharmacology · 2024Article
- Clinical and non-clinical aspects of reimbursement policy for orphan drugs in selected European countries.Frontiers in pharmacology · 2024Article
- COVID-19 in patients with chronic lymphocytic leukemia: a multicenter analysis by the Czech CLL study group.Annals of hematology · 2023Article
- Novel approach to decision making for orphan drugs.International journal of technology assessment in health care · 2023Article
- Evaluating the national system for rare diseases in China from the point of drug access: progress and challenges.Orphanet journal of rare diseases · 2022Article
- Real-world use of orphan medicinal products (OMPs) in rare disease (RD) patients: A population-based registry study.Frontiers in pharmacology · 2022Article
- Availability and Accessibility of Orphan Medicinal Products to Patients in Slovakia in the Years 2010-2019.Frontiers in pharmacology · 2022Article
- Gene Therapy for Neuromuscular Diseases: Health Economic Challenges and Future Perspectives.Journal of neuromuscular diseases · 2022Review
- Pricing and Reimbursement Pathways of New Orphan Drugs in South Korea: A Longitudinal Comparison.Healthcare (Basel, Switzerland) · 2021Article
- A Comparative Analysis of Pricing and Reimbursement of Cystic Fibrosis Transmembrane Conductance Regulator Modulators in Europe.Frontiers in pharmacology · 2021Review
- Assessing the Accuracy of Sales Forecasts Submitted by Pharmaceutical Companies Applying for Reimbursement in Austria.Frontiers in pharmacology · 2021Article
- How to Value Orphan Drugs? A Review of European Value Assessment Frameworks.Frontiers in pharmacology · 2021Review
- Health technology assessment and reimbursement policy for oncology orphan drugs in Central and Eastern Europe.Orphanet journal of rare diseases · 2020Article
- Public spending on orphan medicines: a review of the literature.Journal of pharmaceutical policy and practice · 2020Article
Corrections and comments
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Authors and funding
16 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundReimbursement policies influence access of patients to orphan drugs in the European countries.
objectivesTo provide a comprehensive description of orphan drug reimbursement policies and to assess reimbursement decision-making process in the EU-CEE countries as well as the impact of the type of approval and disease on reimbursement decisions.
methodsFor each drug, the information regarding conditional approval or approval under exceptional circumstances was obtained from the EMA website. The reimbursement status for analyzed drugs was collected in a questionnaire survey performed in a group of experts in reimbursement policy. The agreement between countries was assessed using the κ coefficient, nominal variables tests were compared using the χ2 test or the Fisher exact test. The impact of the EMA's conditional approval and approval under exceptional circumstances was assessed using logistic regression and presented as an odds ratio (OR).
resultsThe analysis revealed that most orphan drugs were authorized for the treatment of oncological or metabolic diseases [36 drugs (38%) and 22 drugs (23%), respectively]. The shares of reimbursed orphan drugs varied significantly (
conclusionIn considered countries specific regulations on reimbursement of orphan drugs are valid but in Lithuania and Romania no formal HTA process was employed; in case of some countries higher ICER values for orphans are used. The share of reimbursed orphan drugs varied significantly across the countries, but it was not associated with GDP per capita.
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