Article

Characteristics and data reporting of rare disease clinical trials: Getting better but still room for improvement

2021-12-07

Abstract excerpt

<h4>ABSTRACT</h4> <h4>Background</h4> It is estimated that there are more than 7,000 rare diseases (RDs) worldwide, impacting the lives of approximately 400 million people and only 5% have an approved therapy. Facing special challenges, including patient scarceness, incomplete knowledge of the natural history and only few specialized clinical sites, clinical trials (CT) are limited, making the data from trials c...

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work: 519bed04-4440-5e3f-8431-32390b5ef62c
DOI: 10.1101/2021.12.03.471055

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

Characteristics and data reporting of rare disease clinical trials: Getting better but still room for improvementDOI 10.1101/2021.12.03.471055Read

Rare Diseases Clinical Trials Toolbox - Public resources and main considerations to set up a clinical trial on medicinal products for humans in EuropeSemantic proximity Read

Trend of clinical trials of new drugs for rare diseases in China in recent 10 yearsSemantic proximity Read

Application of observational research methods to real-world studies for rare disease drugs: a scoping review protocolSemantic proximity Read

The Current Landscape of Clinical TrialsSemantic proximity Read

MedDRA Adoption and Adverse Event Reporting Quality in Gastrointestinal and Abdominal Surgery Randomized Controlled Trials: A Cross-Sectional AnalysisSemantic proximity Read

Enhancing global clinical trial transparency for better health outcomes for allSemantic proximity Read

Enhancing global clinical trial transparency for better health outcomes for allSemantic proximity Read

Updated Analysis of Pediatric Clinical Trials Registered in ClinicalTrials.gov, 2008-2019Semantic proximity Read

Clinical trial and real-world data: A comparative study in patients with diabetic kidney diseaseSemantic proximity Read

Prioritization of Research Engaged with Rare Disease Stakeholders: A Systematic Review and Thematic AnalysisSemantic proximity Read

Diagnosis of Rare Diseases: A scoping review of clinical decision support systemsSemantic proximity Read

CureSCi Metadata Catalog—finding and harmonizing studies for secondary analysis of hydroxyurea use for sickle cell diseaseSemantic proximity Read