ArticleOrphanet journal of rare diseases2020
Registries for orphan drugs: generating evidence or marketing tools?
Article in Orphanet journal of rare diseases, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 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
17 citing papers in PubMed, 26 citations in OpenAlex.
- A toolkit for new facioscapulohumeral muscular dystrophy trial sites.Journal of neuromuscular diseases · 2026Review
- Medicine Development and Access for Rare Diseases: Can We Do Better?Journal of inherited metabolic disease · 2026Review
- The Economics of Drug Development: A Comparison between Orphan and Non-orphan Drugs.Current drug discovery technologies · 2026Article
- Orphan drugs: The final frontier in the fight against rare diseases.Indian journal of pharmacology · 2025Article
- Framework for Multistakeholder Patient Registries in the Field of Rare Diseases: Focus on Neurogenetic Diseases.Neurology · 2024Article
- Twice weekly dosing with Sebelipase alfa (Kanuma®) rescues severely ill infants with Wolman disease.Orphanet journal of rare diseases · 2024Article
- Cross-national comparative study of orphan drug policies in Saudi Arabia, the United States, and the European Union.Saudi pharmaceutical journal : SPJ : the official publication of the Saudi Pharmaceutical Society · 2023Article
- Core protocol development for phase 2/3 clinical trials in the leukodystrophy vanishing white matter: a consensus statement by the VWM consortium and patient advocates.BMC neurology · 2023Article
- The Muscular Dystrophy Association's neuroMuscular ObserVational Research Data Hub (MOVR): Design, Methods, and Initial Observations.Journal of neuromuscular diseases · 2023Article
- How to translate and implement the current science of gene therapy into haemophilia care?Therapeutic advances in hematology · 2023Review
- Access to medicines for rare diseases: A European regulatory roadmap for academia.Frontiers in pharmacology · 2023Article
- Rare lysosomal disease registries: lessons learned over three decades of real-world evidence.Orphanet journal of rare diseases · 2022Review
- Modified Delphi procedure-based expert consensus on endpoints for an international disease registry for Metachromatic Leukodystrophy: The European Metachromatic Leukodystrophy initiative (MLDi).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
- Remodeling an existing rare disease registry to be used in regulatory context: Lessons learned and recommendations.Frontiers in pharmacology · 2022Article
- Disease monitoring programs of rare genetic diseases: transparent data sharing between academic and commercial stakeholders.Orphanet journal of rare diseases · 2021Article
- Perspectives, Expectations, and Concerns of European Patient Advocates on Advanced Therapy Medicinal Products.Frontiers in medicine · 2021Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
8 authors at 3 institutions in 3 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Independent disease registries for pre-and post-approval of novel treatments for rare diseases are increasingly important for healthcare professionals, patients, regulators and the pharmaceutical industry. Current registries for rare diseases to evaluate orphan drugs are mainly set up and owned by the pharmaceutical industry which leads to unacceptable conflicts of interest. To ensure independence from commercial interests, disease registries should be set up and maintained by healthcare professionals and patients. Public funding should be directed towards an early establishment of international registries for orphan diseases, ideally well before novel treatments are introduced. Regulatory bodies should insist on the use of data from independent disease registries rather than company driven, drug-oriented registries.
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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.