Article

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

2024-05-16

Abstract excerpt

<h4>Objective</h4> The primary objective is to identify which observational research methods have been used in the last 5 years in rare disease drug evaluation and how they are applied to generate adequate evidence regarding the real-world effectiveness or safety of rare disease drugs. <h4>Background</h4> Rare disease is an umbrella term for a condition which affects <200,000 people each year and despite the rarit...

Topics

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

Identifiers and source

Literature Corpus work: 0253f347-a1bf-5b40-9da8-fb9b629f0155
DOI: 10.1101/2024.05.16.24307474

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

Application of observational research methods to real-world studies for rare disease drugs: a scoping review protocolDOI 10.1101/2024.05.16.24307474Read

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

Recommendations from the IRDiRC Working Group on methodologies to assess the impact of diagnoses and therapies on rare disease patientsSemantic proximity Read

A systematic scoping review on quality criteria for clinical practice guidelines for rare diseasesSemantic proximity Read

Aging and rare diseases: from epidemiology to a call to actionSemantic proximity Read

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

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

Efficacy and Safety of AYUSH-64 in COVID-19: A Systematic Review and Meta-AnalysisSemantic proximity Read

Nationwide Comprehensive Epidemiological Study of Rare Diseases in Japan Using a Health Insurance Claims DatabaseSemantic proximity Read

A Methodological Review of Randomised n-of-1 Trials.Semantic proximity Read

The PRISMA 2020 statement: an updated guideline for reporting systematic reviewsSemantic proximity Read

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