ArticleJournal of general internal medicine2014
The Rare Diseases Clinical Research Network's organization and approach to observational research and health outcomes research.
Article in Journal of general internal medicine, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 29 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
29 citing papers in PubMed.
- Novel clinical trial design and analytic methods to tackle challenges in therapeutic development in rare diseases.Annals of translational medicine · 2022Trial
- Launching a multi-level data strategy for rare eye diseases: a methodological case study from national to European scale.Orphanet journal of rare diseases · 2026Article
- Collaboration as a Catalyst for Advancing Rare Disease Research: The Experience of the Rare Diseases Clinical Research Network.Clinical and translational science · 2026Review
- Evolving roles of Data Coordinating Centers in multisite research: Challenges and adaptations from a rapid scoping review.Journal of clinical and translational science · 2026Review
- Towards cross-application model-agnostic federated cohort discovery.Journal of the American Medical Informatics Association : JAMIA · 2024Article
- Article
- Global Regulatory and Public Health Initiatives to Advance Pediatric Drug Development for Rare Diseases.Therapeutic innovation & regulatory science · 2022Review
- Accrual-Monitoring Practices for Various Disease Trials among AACI Member Cancer Centers.Clinics and practice · 2022Article
- The Rare Disease Research Scholars Program: A training curriculum for clinical researchers with mixed methods evaluation study.Translational science of rare diseases · 2022Article
- Randomized and non-randomized designs for causal inference with longitudinal data in rare disorders.Orphanet journal of rare diseases · 2021Review
- Model organisms contribute to diagnosis and discovery in the undiagnosed diseases network: current state and a future vision.Orphanet journal of rare diseases · 2021Article
- The practice of active patient involvement in rare disease research using ICT: experiences and lessons from the RUDY JAPAN project.Research involvement and engagement · 2021Article
- Developments in evidence creation for treatments of inborn errors of metabolism.Journal of inherited metabolic disease · 2021Review
- Assessing disease experience across the life span for individuals with osteogenesis imperfecta: challenges and opportunities for patient-reported outcomes (PROs) measurement: a pilot study.Orphanet journal of rare diseases · 2019Article
- National information system for rare diseases with an approach to data architecture: A systematic review.Intractable & rare diseases research · 2018Review
- Using a meta-narrative literature review and focus groups with key stakeholders to identify perceived challenges and solutions for generating robust evidence on the effectiveness of treatments for rare diseases.Orphanet journal of rare diseases · 2018Review
- Genomic medicine for kidney disease.Nature reviews. Nephrology · 2018Review
- An overview of the impact of rare disease characteristics on research methodology.Orphanet journal of rare diseases · 2018Review
- Future of Rare Diseases Research 2017-2027: An IRDiRC Perspective.Clinical and translational science · 2018Review
- Progress in Rare Diseases Research 2010-2016: An IRDiRC Perspective.Clinical and translational science · 2018Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors.
Funding
Abstract
Established in 2003 by the Office of Rare Diseases Research (ORDR), in collaboration with several National Institutes of Health (NIH) Institutes/Centers, the Rare Diseases Clinical Research Network (RDCRN) consists of multiple clinical consortia conducting research in more than 200 rare diseases. The RDCRN supports longitudinal or natural history, pilot, Phase I, II, and III, case-control, cross-sectional, chart review, physician survey, bio-repository, and RDCRN Contact Registry (CR) studies. To date, there have been 24,684 participants enrolled on 120 studies from 446 sites worldwide. An additional 11,533 individuals participate in the CR. Through a central data management and coordinating center (DMCC), the RDCRN's platform for the conduct of observational research encompasses electronic case report forms, federated databases, and an online CR for epidemiological and survey research. An ORDR-governed data repository (through dbGaP, a database for genotype and phenotype information from the National Library of Medicine) has been created. DMCC coordinates with ORDR to register and upload study data to dbGaP for data sharing with the scientific community. The platform provided by the RDCRN DMCC has supported 128 studies, six of which were successfully conducted through the online CR, with 2,352 individuals accrued and a median enrollment time of just 2 months. The RDCRN has built a powerful suite of web-based tools that provide for integration of federated and online database support that can accommodate a large number of rare diseases on a global scale. RDCRN studies have made important advances in the diagnosis and treatment of rare diseases.
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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.