ReviewFrontiers in medicine2021
Regulatory Standards in Orphan Medicinal Product Designation in the EU.
Review in Frontiers in medicine, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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
15 citing papers in PubMed.
- Orphan Designation for Drugs Approved in the United States and European Union: A Comparative Analysis.Clinical pharmacology and therapeutics · 2026Article
- Paediatric medicines development for Duchenne muscular dystrophy: An EU regulatory perspective.Journal of neuromuscular diseases · 2026Review
- Strategic Drivers Behind Early Withdrawal of Orphan Designations in the EU: A Retrospective Analysis (2000-2024).Therapeutic innovation & regulatory science · 2026Article
- Reconceiving orphan drug market exclusivity as a conditional public-law entitlement: a sustainable regulatory governance model with comparative lessons for China.Frontiers in medicine · 2026Article
- Time to consider fracture nonunion an orphan disease? An update into pathophysiology, epidemiology and therapeutic solutions.European journal of trauma and emergency surgery : official publication of the European Trauma Society · 2025Review
- Navigating the orphan medicinal product designation: Evidence requirements for gene therapies in Europe.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- Regulatory aspects of optogenetic research and therapy for retinitis pigmentosa under EU law.Frontiers in medical technology · 2025Review
- Key challenges in developing a gene therapy for Usher syndrome: machine-assisted scoping review.Journal of community genetics · 2024Article
- Advancing rare disease treatment: EMA's decade-long insights into engineered adoptive cell therapy for rare cancers and orphan designation.Gene therapy · 2024Review
- Implementation status of pharmacological studies in the development of orphan drugs.Orphanet journal of rare diseases · 2024Article
- Skin-Derived ABCB5International journal of molecular sciences · 2022Review
- Editorial: Prevention, diagnosis and treatment of rare disorders.Frontiers in pharmacology · 2022Article
- Real-world use of orphan medicinal products (OMPs) in rare disease (RD) patients: A population-based registry study.Frontiers in pharmacology · 2022Article
- Defining Satisfactory Methods of Treatment in Rare Diseases When Evaluating Significant Benefit-The EU Regulator's Perspective.Frontiers in medicine · 2021Article
- Patient Centricity: Design and Conduct of Clinical Trials in Orphan Diseases: Third of Three Sets of Expanded Proceedings from the 2020 ISCTM Autumn Conference on Pediatric Drug Development.Innovations in clinical neuroscienceArticle
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Twenty years of orphan regulation in Europe have now elapsed, with almost 2,400 orphan designated medicinal products and more than 190 orphan products authorised in the EU. Alongside the evolution in understanding of rare diseases, considerable regulatory knowledge has also been accumulated regarding the level of evidence that would support inclusion of products into the framework. This article reviews publications and regulatory documents pertaining to orphan medicinal product designation in the EU and discusses the general expectations in submitted applications as reflected in the current regulatory practise. Important elements to recommend granting a European orphan designation are the key considerations of orphan condition, medical plausibility, seriousness, and prevalence, while significant benefit is also assessed when there are authorised medicinal products for the sought indication. This review attempts to clarify the specific concepts currently used in that regard and discusses how the available data can be used to justify the criteria for designation. Moving away from theoretical expectations or assumptions, it stresses that the applications have to be complemented with nosological and epidemiological justifications pertaining to the proposed condition, as well as relevant data in specific non-clinical
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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.