ArticleOrphanet journal of rare diseases2014
A comparison of interventional clinical trials in rare versus non-rare diseases: an analysis of ClinicalTrials.gov.
Article in Orphanet journal of rare diseases, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 70 papers, 5 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
70 citing papers in PubMed, 5 syntheses or guidelines pooled it.
- Systematic Review of Genetic Substrate Reduction Therapy in Lysosomal Storage Diseases: Opportunities, Challenges and Delivery Systems.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2024Pooled it
- The use of digital outcome measures in clinical trials in rare neurological diseases: a systematic literature review.Orphanet journal of rare diseases · 2023Pooled it
- Health-related quality of life scores of typically developing children and adolescents around the world: a meta-analysis with meta-regression.Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation · 2020Pooled it
- Characteristics of clinical trials in rare vs. common diseases: A register-based Latvian study.PloS one · 2018Pooled it
- Pooled it
- Trial
- Randomized controlled trial data for successful new drug application for rare diseases in the United States.Orphanet journal of rare diseases · 2023Trial
- A survey of patients with laryngotracheal stenosis on future clinical trial design.Clinical trials (London, England) · 2022Trial
- Supply and Demand in the Mathematics of Rare Disease Drug Development: Why Choosing the Right Model Is Crucial.Clinical and translational science · 2026Review
- Utility of 3D facial analysis as a biomarker in rare diseases exploration with hereditary angioedema.Frontiers in medical technology · 2026Article
- Assessing allocation bias in stratified clinical trials with multi-component endpoints evaluated using the stratified Wei-Lachin test.PloS one · 2026Article
- Economic burden of Sanfilippo syndrome in the United States.Archives of public health = Archives belges de sante publique · 2025Article
- Development of a radiographic vertebral severity score for evaluation of disease progression in alkaptonuria.Molecular genetics and metabolism · 2025Article
- EMA approved orphan medicines since the implementation of the orphan legislation.Orphanet journal of rare diseases · 2025Article
- Free fat flap transfer in recurrent neurogenic thoracic outlet syndrome pain treatment: FIRST observational pilot study.International journal of surgery protocols · 2025Article
- Article
- The clinical assessment of studies on orphan drugs in relation to the EMA's authorization marketing decisions in Europe.Frontiers in pharmacology · 2025Article
- Pediatric-Specific Drug Loss Issue in Japan: Comparison of Pediatric Development Status Between Japan and the United States.Therapeutic innovation & regulatory science · 2025Article
- Application of Quantitative Systems Pharmacology Approaches to Support Pediatric Labeling in Rare Diseases.Handbook of experimental pharmacology · 2025Review
- Systemic treatment of recessive dystrophic epidermolysis bullosa with mesenchymal stromal cells: a scoping review of the literature and conclusions for future clinical research.The Journal of dermatological treatment · 2024Article
10 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors.
Funding
Abstract
objectivesTo provide a comprehensive characterisation of rare disease clinical trials registered in ClinicalTrials.gov, and compare against characteristics of trials in non-rare diseases.
designRegistry based study of ClinicalTrials.gov registration entries.
methodsThe ClinicalTrials.gov registry comprised 133,128 studies registered to September 27, 2012. By annotating medical subject heading descriptors to condition terms we could identify rare and non-rare disease trials. A total of 24,088 Interventional trials registered after January 1, 2006, conducted in the United States, Canada and/or the European Union were categorised as rare or non-rare. Characteristics of the respective trials were extracted and summarised with comparative statistics calculated where appropriate.
main outcome measuresCharacteristics of interventional trials reported in the database categorised by rare and non-rare conditions to allow comparison.
resultsOf the 24,088 trials categorised 2,759 (11.5%) were classified as rare disease trials and 21,329 (88.5%) related to non-rare conditions. Despite the limitations of the database we found that rare disease trials differed to non-rare disease trials across all characteristics that we examined. Rare disease trials enrolled fewer participants (median 29 vs. 62), were more likely to be single arm (63.0% vs. 29.6%), non-randomised (64.5% vs. 36.1%) and open label (78.7% vs. 52.2%). A higher proportion of rare disease trials were terminated early (13.7% vs. 6.3%) and proportionally fewer rare disease studies were actively pursuing, or waiting to commence, enrolment (15.9% vs. 38.5%).
conclusionRare disease interventional trials differ from those in non-rare conditions with notable differences in enrolment, design, blinding and randomisation. However, clinical trials should aim to implement the highest trial design standards possible, regardless of whether diseases are rare or not.
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.