ArticleNature reviews. Methods primers2022
Designing clinical trials for rare diseases: unique challenges and opportunities.
Article in Nature reviews. Methods primers, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 36 papers, 3 of them syntheses 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
36 citing papers in PubMed, 3 syntheses or guidelines pooled it.
- Characteristics of pediatric drug-related investigator-initiated trials in Chinese children's hospitals: a comparative analysis based on ChiCTR and ClinicalTrials.gov registration data.Frontiers in pharmacology · 2026Pooled it
- Acceptance of external control arms by HTA agencies: a review of oncology submissions in France, England, Germany and Norway from 2021 to 2023.British journal of cancer · 2025Pooled it
- Keratin-associated epidermolysis bullosa simplex: phenotypes and challenges in clinical trials - a narrative review and systematic update.Orphanet journal of rare diseases · 2025Pooled it
- Garadacimab in Hereditary Angioedema: Modeling Suggests Sustained Improvements in Attack-Free Probability and Quality of Life Over 5 Years.Clinical and translational science · 2026Trial
- Associations Between Short-Term Changes in Disease Activity and Long-Term Clinical Outcomes in Activated Phosphoinositide 3-Kinase Delta Syndrome.Journal of immunology research · 2026Trial
- Emerging therapeutic strategies for mitochondrial DNA-related diseases.Cell reports. Medicine · 2026Review
- The design of a small Bayesian hybrid-controlled trial utilizing time-to-event data from six published sources that used best alternative care to treat liver-predominant metastasis from primary ocular melanoma.Contemporary clinical trials communications · 2026Article
- Non-invasive biomarkers for diagnosis and monitoring of primary mitochondrial diseases.Journal of neurology · 2026Review
- Realising precision oncology through shared real-world data infrastructure.Acta oncologica (Stockholm, Sweden) · 2026Article
- AMCP Market Insights: The role of managed care in improving patient care in mucopolysaccharidoses.Journal of managed care & specialty pharmacy · 2026Article
- Screening failure in systemic sclerosis randomized trials: reporting, rates, causes and trends over time.Rheumatology advances in practice · 2026Article
- New therapeutic strategies for Lafora disease: Evaluation of the safety, efficacy, pharmacokinetics and metabolomic profile of intravenous VAL-1221 treatment.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2026Article
- Personalized Medicine for Systemic Sclerosis-Associated Interstitial Lung Disease.Current treatment options in rheumatology · 2025Article
- Update on Disease-Modifying Pharmacological Treatments for Frontotemporal Dementia (FTD): A Scoping Review of Registered Trials.NeuroSci · 2025Review
- Innovative Approaches to Improve the Success of Pituitary Surgery in Cushing's Disease: A Cost-Effectiveness Perspective.Clinical endocrinology · 2025Article
- Multisystem Symptoms in Myotonic Dystrophy Type 1: A Management and Therapeutic Perspective.International journal of molecular sciences · 2025Review
- Clinical Trial End Points for Childhood CKD.Journal of the American Society of Nephrology : JASN · 2025Article
- Precision therapies for genetic epilepsies in 2025: Promises and pitfalls.Epilepsia open · 2025Article
- Predictive machine learning and multimodal data to develop highly sensitive, composite biomarkers of disease progression in Friedreich ataxia.Scientific reports · 2025Article
- Using Simulation to Optimize the Design of Adaptive Clinical Trials.Journal of the American Society of Nephrology : JASN · 2025Article
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
Abstract
Orphan drug development is a rapidly expanding field. Nevertheless, clinical trials for rare diseases can present inherent challenges. Optimal study design and partnerships between academia and industry are therefore required for the successful development, delivery and clinical approval of effective therapies in this group of disorders.
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.