ArticleOrphanet journal of rare diseases2022
What role can decentralized trial designs play to improve rare disease studies?
Article in Orphanet journal of rare diseases, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 25 papers, 1 of them a synthesis that pooled it.
What it found
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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
25 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Recommendations to promote equity, diversity and inclusion in decentralized clinical trials.Nature medicine · 2024Guideline
- Feasibility study for a fully decentralized clinical trial in participants with functional constipation symptoms.Clinical and translational science · 2023Trial
- 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
- Association of Protocol Design-Related Factors With Enrollment Duration in Clinical Trials.Clinical and translational science · 2026Article
- An updated review of the SMA clinical trial landscape in the United States: Findings from analysis of recruitment targets on ClinicalTrials.gov and a survey of SMA clinical trial sites on factors affecting site capacity and readiness.Contemporary clinical trials communications · 2026Article
- Clinical trial awareness and participation attitudes among patients with epidermolysis bullosa and their caregivers: a nationwide cross-sectional study from Saudi Arabia.Frontiers in medicine · 2026Article
- Digital health technology use in clinical trials of rare diseases: a systematic review.Communications medicine · 2025Article
- The Effectiveness of a Mobile National Remote Emergency System for Malignant Hyperthermia in China: Retrospective Pre-Post Implementation Study.Journal of medical Internet research · 2025Article
- Adaptation of Clinical Research Staff to Decentralized Clinical Trials and Impacts on the Patient-Centered Experience: Qualitative Interview Study.Journal of medical Internet research · 2025Article
- A systematic literature review of the impact and measurement of mobility impairment in rare bone diseases.Therapeutic advances in musculoskeletal disease · 2025Review
- Decentralized subject recruitment for a prospective community surveillance system: The influence of social determinants of health on inclusion of minorities in research.Journal of clinical and translational science · 2025Article
- Clinical trial design, end-points, and emerging therapies in pulmonary arterial hypertension.The European respiratory journal · 2024Review
- Rare disease clinical trials in the European Union: navigating regulatory and clinical challenges.Orphanet journal of rare diseases · 2024Review
- Progressive Supranuclear palsy (PSP) disease progression, management, and healthcare resource utilization: a retrospective observational study in the US and Canada.Orphanet journal of rare diseases · 2024Observational
- Decentralisation in Clinical Trials and Patient Centricity: Benefits and Challenges.Pharmaceutical medicine · 2024Review
- TRPC6 Inhibitor BI 764198 in Focal Segmental Glomerulosclerosis: Phase 2 Study Design.Kidney international reports · 2023Article
- Aiding the Adoption of Master Protocols by Optimizing Patient Engagement.Therapeutic innovation & regulatory science · 2023Article
- How do patients and other members of the public engage with the orphan drug development? A narrative qualitative synthesis.Orphanet journal of rare diseases · 2023Review
- Empowering drug development: Leveraging insights from imaging technologies to enable the advancement of digital health technologies.Clinical and translational science · 2023Review
- Literature Review on Health Emigration in Rare Diseases-A Machine Learning Perspective.International journal of environmental research and public health · 2023Review
Corrections and comments
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Authors and funding
4 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
People affected by rare diseases want to be involved in research and the search for new treatments. Randomized controlled trials remain the best way of finding new interventions, but many elements of traditional study design are not best suited for rare diseases. Barriers to patients and families include the use of specialist hospital sites for recruitment, requiring frequent site-based study visits for data collection, and a high burden of tests and outcome measures in research. While decentralized clinical trial (DCT) designs have been developed in some rare disease trials, changes necessitated by the COVID-19 pandemic present an opportunity for them to become a standard approach. DCT approaches have been shown to be more resilient to changes in enrolment and attrition during COVID-19 than traditional designs and offer benefits in terms of patient burden, convenience, inclusion, and data quality. Digital tools such as wearable devices and electronic clinical outcome assessments may also provide more convenient and environmentally valid measures of how a condition affects the life of an individual in their regular environment (e.g. mobility around the home versus a hospital corridor). Digital solutions have greater ability to support language localization, accessibility, and may lead to increase access to global rare disease trials. In parallel, challenges exist, such as the technical support, the digital divide, ensuring high quality data, and delivering safe trials.
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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.