ReviewOrphanet journal of rare diseases2023
Decentralized clinical trials and rare diseases: a Drug Information Association Innovative Design Scientific Working Group (DIA-IDSWG) perspective.
Review in Orphanet journal of rare diseases, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 26 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
26 citing papers in PubMed, 1 synthesis or guideline pooled it, 47 citations in OpenAlex.
- Recommendations to promote equity, diversity and inclusion in decentralized clinical trials.Nature medicine · 2024Guideline
- Advancing clinical trials for rare renal cell carcinoma subtypes: Consensus statements from the International Kidney Cancer Symposium North America 2025 think tank.Urologic oncology · 2026Article
- TrialR: critical enablers and the need for reusable Rare Disease Clinical Trial infrastructure in Western Australia.Orphanet journal of rare diseases · 2026Article
- Development of at-home video recordings for functional skill assessment in Angelman Syndrome: a pilot study.Journal of neurodevelopmental disorders · 2026Article
- Areas of research priorities in epilepsy: A position paper of the European Reference Network for Rare and Complex Epilepsies, EpiCARE.Epilepsia open · 2026Article
- Article
- Decentralized clinical trials: A comprehensive analysis of trends, technologies, and global challenges.PLOS digital health · 2026Article
- Lumbar Acceleration Gait Estimation: "Step-by-Step" Algorithm Updates and Improvements.Journal of medical Internet research · 2025Article
- Trial characteristics, methods and reported challenges of decentralised clinical trials: a scoping review.BMJ open · 2025Article
- Opportunities in Development of Patient-Centric and Decentralized Clinical Trials: Insights from Patients and Healthcare Professionals in Respiratory and Rare Diseases.Therapeutic innovation & regulatory science · 2025Article
- Recommendation Paper on Advancing the Use of Decentralised Elements in Clinical Trials.Therapeutic innovation & regulatory science · 2025Article
- Insights on Clinical Development of Cell and Gene Therapy for Rare Diseases-by DahShu Innovative Design Scientific Working Group (IDSWG).Therapeutic innovation & regulatory science · 2025Review
- Decentralized Clinical Trials in Pediatrics: Is Italy Ready?Journal of clinical medicine · 2025Article
- Digital Cardiovascular Twins, AI Agents, and Sensor Data: A Narrative Review from System Architecture to Proactive Heart Health.Sensors (Basel, Switzerland) · 2025Review
- Baicalein-mediated regulation of Nrf2/ARE, NFĸB, and MAPK signaling in Huntington's disease: a promising strategy against neuroinflammation and neurodegeneration.Inflammopharmacology · 2025Review
- The Current Landscape of Clinical Trials.Journal of clinical medicine · 2025Article
- Building a Digital Health Research Platform to Enable Recruitment, Enrollment, Data Collection, and Follow-Up for a Highly Diverse Longitudinal US Cohort of 1 Million People in the All of Us Research Program: Design and Implementation Study.Journal of medical Internet research · 2025Article
- Key challenges in developing a gene therapy for Usher syndrome: machine-assisted scoping review.Journal of community genetics · 2024Article
- A study of implementation factors for a novel approach to clinical trials: constructs for consideration in the coordination of direct-to-patient online-based medical research.BMC medical research methodology · 2024Article
- Monitoring Activity and Gait in Children (MAGIC) using digital health technologies.Pediatric research · 2024Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
10 authors at 8 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundTraditional clinical trials require tests and procedures that are administered in centralized clinical research sites, which are beyond the standard of care that patients receive for their rare and chronic diseases. The limited number of rare disease patients scattered around the world makes it particularly challenging to recruit participants and conduct these traditional clinical trials. MAIN BODY: Participating in clinical research can be burdensome, especially for children, the elderly, physically and cognitively impaired individuals who require transportation and caregiver assistance, or patients who live in remote locations or cannot afford transportation. In recent years, there is an increasing need to consider Decentralized Clinical Trials (DCT) as a participant-centric approach that uses new technologies and innovative procedures for interaction with participants in the comfort of their home.
conclusionThis paper discusses the planning and conduct of DCTs, which can increase the quality of trials with a specific focus on rare diseases.
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.