SynthesisOrphanet journal of rare diseases2016
Systematic review on the evaluation criteria of orphan medicines in Central and Eastern European countries.
Synthesis in Orphanet journal of rare diseases, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 34 papers, 6 of them syntheses that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
34 citing papers in PubMed, 6 syntheses or guidelines pooled it, 66 citations in OpenAlex.
- Issues, Challenges and Opportunities for Economic Evaluations of Orphan Drugs in Rare Diseases: An Umbrella Review.PharmacoEconomics · 2024Pooled it
- Criteria and Scoring Functions Used in Multi-criteria Decision Analysis and Value Frameworks for the Assessment of Rare Disease Therapies: A Systematic Literature Review.PharmacoEconomics - open · 2021Pooled it
- A systematic review of moral reasons on orphan drug reimbursement.Orphanet journal of rare diseases · 2021Pooled it
- Potential Criteria for Frameworks to Support the Evaluation of Innovative Medicines in Upper Middle-Income Countries-A Systematic Literature Review on Value Frameworks and Multi-Criteria Decision Analyses.Frontiers in pharmacology · 2020Pooled it
- Multi-criteria decision analysis for health technology assessment: addressing methodological challenges to improve the state of the art.The European journal of health economics : HEPAC : health economics in prevention and care · 2019Pooled it
- A Review of Rare Disease Policies and Orphan Drug Reimbursement Systems in 12 Eurasian Countries.Frontiers in public health · 2019Pooled it
- Rare disease patients protection: theoretical discussion and institutional construction of orphan drug designation in China.Orphanet journal of rare diseases · 2026Article
- The Significance of Hypophosphatemia in Deciding on an Optimal Clinical Choice of Parenteral Iron Therapy in Patients with Chronic Inflammatory Bowel Disease in Slovenia: An Umbrella Review and Economic Evaluation.Healthcare (Basel, Switzerland) · 2026Article
- Evolution and synergy of healthcare security policies for rare diseases in China: a quantitative content analysis.Frontiers in public health · 2026Article
- Optimizing Patient Access to Orphan Medicinal Products: Lessons from Central and Eastern Europe.Journal of market access & health policy · 2025Article
- Rare disease challenges and potential actions in the Middle East.International journal for equity in health · 2025Article
- Special FDA designations for drug development: orphan, fast track, accelerated approval, priority review, and breakthrough therapy.The European journal of health economics : HEPAC : health economics in prevention and care · 2024Review
- Clinical and non-clinical aspects of reimbursement policy for orphan drugs in selected European countries.Frontiers in pharmacology · 2024Article
- Clinical aspects of reimbursement policies for orphan drugs in Central and Eastern European countries.Frontiers in pharmacology · 2024Article
- Literature Review on Health Emigration in Rare Diseases-A Machine Learning Perspective.International journal of environmental research and public health · 2023Review
- The reimbursement for expensive medicines: stakeholder perspectives on the SMA medicine nusinersen and the Dutch Coverage Lock policy.BMC health services research · 2022Article
- Should Hungary Pay More for a QALY Gain than Higher-Income Western European Countries?Applied health economics and health policy · 2022Article
- The Awareness of Rare Diseases Among Medical Students and Practicing Physicians in the Republic of Kazakhstan. An Exploratory Study.Frontiers in public health · 2022Article
- Real-world use of orphan medicinal products (OMPs) in rare disease (RD) patients: A population-based registry study.Frontiers in pharmacology · 2022Article
- How Can We Optimize the Value Assessment and Appraisal of Orphan Drugs for Reimbursement Purposes? A Qualitative Interview Study Across European Countries.Frontiers in pharmacology · 2022Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors at 3 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundIn case of orphan drugs applicability of the standard health technology assessment (HTA) process is limited due to scarcity of good clinical and health economic evidence. Financing these premium priced drugs is more controversial in the Central and Eastern European (CEE) region where the public funding resources are more restricted, and health economic justification should be an even more important aspect of policy decisions than in higher income European countries.
objectivesTo explore and summarize the recent scientific evidence on value drivers related to the health technology assessment of ODs with a special focus on the perspective of third party payers in CEE countries. The review aims to list all potentially relevant value drivers in the reimbursement process of orphan drugs.
methodsA systematic literature review was performed; PubMed and Scopus databases were systematically searched for relevant publications until April 2015. Extracted data were summarized along key HTA elements.
resultsFrom the 2664 identified publications, 87 contained relevant information on the evaluation criteria of orphan drugs, but only 5 had direct information from the CEE region. The presentation of good clinical evidence seems to play a key role especially since this should be the basis of cost-effectiveness analyses, which have more importance in resource-constrained economies. Due to external price referencing of pharmaceuticals, the relative budget impact of orphan drugs is expected to be higher in CEE than in Western European (WE) countries unless accessibility of patients remains more limited in poorer European regions. Equity principles based on disease prevalence and non-availability of alternative treatment options may increase the price premium, however, societies must have some control on prices and a rationale based on multiple criteria in reimbursement decisions.
conclusionsThe evaluation of orphan medicines should include multiple criteria to appropriately measure the clinical added value of orphan drugs. The search found only a small number of studies coming from CEE, therefore European policies on orphan drugs may be based largely on experiences in WE countries. More research should be done in the future in CEE because financing high-priced orphan drugs involves a greater burden for these countries.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.