ReviewFrontiers in pharmacology2022
Contribution of patient registries to regulatory decision making on rare diseases medicinal products in Europe.
Review in Frontiers in pharmacology, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 papers, 1 of them a synthesis that pooled it.
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
20 citing papers in PubMed, 1 synthesis or guideline pooled it, 27 citations in OpenAlex.
- Methodologies for generating and evaluating clinical and performance evidence for high-risk and innovative medical devices andFrontiers in medical technology · 2026Pooled it
- Review
- Pathways to improving the awareness, diagnosis and management of lipodystrophy in Brazil: an expert panel discussion.Diabetology & metabolic syndrome · 2025Review
- Real-World Data Included in Post-authorisation Measures: A Case Study of Approved Advanced Therapy Medicinal Products in the European Union between 2013 and 2024.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2025Article
- Navigating the Real World: A Scoping Review of Structured Frameworks to Effectively Identify, Evaluate, and Select Real-World Data Sources for Fit-for-Purpose Studies.Clinical pharmacology and therapeutics · 2025Article
- Optimizing Patient Registries for Regulatory Decision Making - Key Learnings From an HMA/EMA Multistakeholder Workshop.Clinical pharmacology and therapeutics · 2025Review
- Advancing Real-World Evidence Through a Federated Health Data Network (EHDEN): Descriptive Study.Journal of medical Internet research · 2025Article
- Behavioral Phenotypes and Comorbidity in 3q29 Deletion Syndrome: Results from the 3q29 Registry.Journal of autism and developmental disorders · 2025Article
- Incorporating patient-reported outcome measures into routine paediatric pharmacovigilance: opportunities for safety monitoring with practical use scenarios from paediatric oncology.Frontiers in pharmacology · 2025Review
- Framework for Multistakeholder Patient Registries in the Field of Rare Diseases: Focus on Neurogenetic Diseases.Neurology · 2024Article
- A rare partnership: patient community and industry collaboration to shape the impact of real-world evidence on the rare disease ecosystem.Orphanet journal of rare diseases · 2024Review
- Catching up but still miles behind-a patient registry for otoferlin.Experimental & molecular medicine · 2024Article
- Key Drivers of Coagulation Factor Use in Von Willebrand Disease During Hospitalization: An Overview of the French BERHLINGO Cohort.Clinical drug investigation · 2024Article
- Innovative research methodologies in the EU regulatory framework: an analysis of EMA qualification procedures from a pediatric perspective.Frontiers in medicine · 2024Article
- Leigh syndrome global patient registry: uniting patients and researchers worldwide.Orphanet journal of rare diseases · 2023Article
- Real-world outcomes of pazopanib in metastatic soft tissue sarcoma: a retrospective Turkish oncology group (TOG) study.Journal of cancer research and clinical oncology · 2023Article
- The Metreleptin Effectiveness and Safety Registry (MEASuRE): concept, design and challenges.Orphanet journal of rare diseases · 2023Article
- Navigating a transforming landscape: the evolving role of pharmacovigilance physicians in drug development and implications for future challenges and training requirements.Frontiers in drug safety and regulation · 2023Article
- Editorial: Safety and side effects of psychotropic medications.Frontiers in psychiatry · 2023Article
- Contribution of Real-World Evidence in European Medicines Agency's Regulatory Decision Making.Clinical pharmacology and therapeutics · 2023Article
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 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Between 2000 and 2021, the European Medicines Agency (EMA) assigned the orphan designation to over 1,900 medicines. Due to their small target populations, leading to challenges regarding clinical trial recruitment, study design and little knowledge on the natural history of the disease, the overall clinical evidence submitted at the time of marketing authorisation application for these medicines is often limited. Patient registries have been recognised as important sources of data on healthcare practices, drug utilisation and clinical outcomes. They may help address these challenges by providing information on epidemiology, standards of care and treatment patterns of rare diseases. In this review, we illustrate the utility of patient registries across the different stages of development of medicinal products, including orphans, to provide evidence in the context of clinical studies and to generate post-authorisation long term data on their effectiveness and safety profiles. We present important initiatives leveraging the role of registries for orphan medicinal products' development and monitoring to ultimately improve patients' lives.
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.