ArticleOrphanet journal of rare diseases2017
Recommendations from the European Working Group for Value Assessment and Funding Processes in Rare Diseases (ORPH-VAL).
Article in Orphanet journal of rare diseases, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 57 papers, 5 of them syntheses that pooled it.
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Who cites it
57 citing papers in PubMed, 5 syntheses or guidelines pooled it, 104 citations in OpenAlex.
- Economic evaluations of disease-modifying therapies for spinal muscular atrophy: a systematic literature review.Orphanet journal of rare diseases · 2025Pooled it
- Institutional Priority-Setting for Novel Drugs and Therapeutics: A Qualitative Systematic Review.International journal of health policy and management · 2024Pooled 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
- TRUST4RD: tool for reducing uncertainties in the evidence generation for specialised treatments for rare diseases.Orphanet journal of rare diseases · 2020Trial
- Guidelines or Other Guidance Documents for Rare Diseases in the Netherlands: When and Where to Invest (Effort, Time and Money)?Clinical and public health guidelines · 2026Article
- Access to orphan drugs in adults with inherited metabolic diseases in Switzerland: a single-center retrospective cohort study.Orphanet journal of rare diseases · 2026Article
- Assessing the Value Contribution of VyvgartAdvances in therapy · 2026Article
- A bibliometric study of rare diseases in English and Chinese databases from 1985 to 2024 based on CiteSpace.Intractable & rare diseases research · 2025Article
- The role of public health in rare diseases: hemophilia as an example.Frontiers in public health · 2025Review
- Uncovering the hidden socioeconomic impact of juvenile idiopathic arthritis and paving the way for other rare childhood diseases: an international, cross-disciplinary, patient-centered approach (PAVE Consortium).Pediatric rheumatology online journal · 2024Article
- Experiences of patient organizations' involvement in medicine appraisal and reimbursement processes in Finland - a qualitative study.International journal of technology assessment in health care · 2024Article
- Comparative policy analysis of national rare disease funding policies in Australia, Singapore, South Korea, the United Kingdom and the United States: a scoping review.Health economics review · 2024Article
- Priority-setting for hospital funding of high-cost innovative drugs and therapeutics: A qualitative institutional case study.PloS one · 2024Article
- Defining 'therapeutic value' of medicines: a scoping review.BMJ open · 2023Article
- Exploring the feasibility of using the ICER Evidence Rating Matrix for Comparative Clinical Effectiveness in assessing treatment benefit and certainty in the clinical evidence on orphan therapies for paediatric indications.Orphanet journal of rare diseases · 2023Article
- Developing a Framework of Cost Elements of Socioeconomic Burden of Rare Disease: A Scoping Review.PharmacoEconomics · 2023Article
- Novel approach to decision making for orphan drugs.International journal of technology assessment in health care · 2023Article
- Literature Review on Health Emigration in Rare Diseases-A Machine Learning Perspective.International journal of environmental research and public health · 2023Review
Corrections and comments
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Authors and funding
15 authors at 12 institutions in 6 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Rare diseases are an important public health issue with high unmet need. The introduction of the EU Regulation on orphan medicinal products (OMP) has been successful in stimulating investment in the research and development of OMPs. Despite this advancement, patients do not have universal access to these new medicines. There are many factors that affect OMP uptake, but one of the most important is the difficulty of making pricing and reimbursement (P&R) decisions in rare diseases. Until now, there has been little consensus on the most appropriate assessment criteria, perspective or appraisal process. This paper proposes nine principles to help improve the consistency of OMP P&R assessment in Europe and ensure that value assessment, pricing and funding processes reflect the specificities of rare diseases and contribute to both the sustainability of healthcare systems and the sustainability of innovation in this field. These recommendations are the output of the European Working Group for Value Assessment and Funding Processes in Rare Diseases (ORPH-VAL), a collaboration between rare disease experts, patient representatives, academics, health technology assessment (HTA) practitioners, politicians and industry representatives. ORPH-VAL reached its recommendations through careful consideration of existing OMP P&R literature and through a wide consultation with expert stakeholders, including payers, regulators and patients. The principles cover four areas: OMP decision criteria, OMP decision process, OMP sustainable funding systems and European co-ordination. This paper also presents a guide to the core elements of value relevant to OMPs that should be consistently considered in all OMP appraisals. The principles outlined in this paper may be helpful in drawing together an emerging consensus on this topic and identifying areas where consistency in payer approach could be achievable and beneficial. All stakeholders have an obligation to work together to ensure that the promise of OMP's is realised.
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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.