ArticleOrphanet journal of rare diseases2015
Principles for consistent value assessment and sustainable funding of orphan drugs in Europe.
Article in Orphanet journal of rare diseases, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers, 1 of them a synthesis 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
17 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
- Debates over orphan drug pricing: a meta-narrative literature review.Orphanet journal of rare diseases · 2025Review
- Unraveling elements of value-based pricing from a pharmaceutical industry's perspective: a scoping review.Frontiers in pharmacology · 2024Article
- Defining 'therapeutic value' of medicines: a scoping review.BMJ open · 2023Article
- Literature Review on Health Emigration in Rare Diseases-A Machine Learning Perspective.International journal of environmental research and public health · 2023Review
- The actual status of drug prices and adjustment factors for drug price calculation: an analysis of ultra-orphan drug development in Japan.Orphanet journal of rare diseases · 2022Article
- Consideration of quality of life in the health technology assessments of rare disease treatments.The European journal of health economics : HEPAC : health economics in prevention and care · 2022Article
- Assessing the value of orphan drugs using conventional cost-effectiveness analysis: Is it fit for purpose?Orphanet journal of rare diseases · 2022Review
- Availability and Accessibility of Orphan Medicinal Products to Patients in Slovakia in the Years 2010-2019.Frontiers in pharmacology · 2022Article
- Estimating the fiscal impact of rare diseases using a public economic framework: a case study applied to hereditary transthyretin-mediated (hATTR) amyloidosis.Orphanet journal of rare diseases · 2019Article
- Revealed preferences towards the appraisal of orphan drugs in Poland - multi criteria decision analysis.Orphanet journal of rare diseases · 2018Article
- Multi-Criteria Decision Analysis (MCDA) Models in Health Technology Assessment of Orphan Drugs-a Systematic Literature Review. Next Steps in Methodology Development?Frontiers in public health · 2018Article
- A Cost-Effectiveness Analysis of Nintedanib in Idiopathic Pulmonary Fibrosis in the UK.PharmacoEconomics · 2017Article
- Recommendations from the European Working Group for Value Assessment and Funding Processes in Rare Diseases (ORPH-VAL).Orphanet journal of rare diseases · 2017Article
- Ethical imperatives of timely access to orphan drugs: is possible to reconcile economic incentives and patients' health needs?Orphanet journal of rare diseases · 2017Article
- Do payers value rarity? An analysis of the relationship between disease rarity and orphan drug prices in Europe.Journal of market access & health policy · 2017Article
- A comparative study of orphan drug prices in Europe.Journal of market access & health policy · 2017Article
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.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The European Orphan Medicinal Products (OMP) Regulation has successfully encouraged research to develop treatments for rare diseases resulting in the authorisation of new OMPs in Europe. While decisions on OMP designation and marketing authorisation are made at the European Union level, reimbursement decisions are made at the national level. OMP value and affordability are high priority issues for policymakers and decisions regarding their pricing and funding are highly complex. There is currently no European consensus on how OMP value should be assessed and inequalities of access to OMPs have previously been observed. Against this background, policy makers in many countries are considering reforms to improve access to OMPs. This paper proposes ten principles to be considered when undertaking such reforms, from the perspective of an OMP manufacturer. We recommend the continued prioritisation of rare diseases by policymakers, an increased alignment between payer and regulatory frameworks, pricing centred on OMP value, and mechanisms to ensure long-term financial sustainability allowing a continuous and virtuous development of OMPs. Our recommendations support the development of more consistent frameworks and encourage collaboration between all stakeholders, including research-based industry, payers, clinicians, and patients.
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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.