Rare Disease Clinical Trials Tools
Table of Contents
The Rare Disease Clinical Trial Network is grateful to the following organisations for their kind permission to share their resources with our members.
ECRIN’s Rare Disease Clinical Trials Toolbox has been developed as a practical aid for developers of clinical trials on medicinal products for human use regardless of therapeutic area.
Resources include:
- Guidance
- Evidence pack
- Leaflets
- Posters
- Videos
- FAQs
The RStudio Basics
The RDCTN has compiled a resource on how to do basic analysis and result visualisation using RStudio. This resource provides a step-by-step introduction to using RStudio and suggestions for how to write your project script.
Brief guide to downloading RStudio from posit
This explains what the function of each section in RStudio is, so that you can orientate yourself
This section includes:
- Suggestions for structuring your script
- Create a directory and workspace
- Save your script and work
- Installing and loading libraries
This will explain how to:
- Upload your data in RStudio
- Calculate statistics for numeric variables (such as mean, standard deviation and your sample size)
- Calculate statistics for categorial variables (such as proportions and sample size)
- Create user-friendly and diverse visualisations of your data
Find step-by-step guides, practice data and template script here:
So we can keep improving this resource, please provide feedback here:
European Joint Programme Rare Diseases
The EJP VP Portal provides a single access point for researchers to explore and query a variety of interconnected Rare Disease resources.
Resources include:
- Biobanks
- Patient registries
- Genomics & multi-omics repositories
- Knowledge bases
- Deposition & analysis platforms
- Clinical research supporting material/services
Also visit the EJP-RD Innovation Management Toolbox – a reference library of RD translational medicine resources!
RED is a central resource for information about clinical study regulatory and ethical requirements in Europe. This tool helps locate relevant information needed to support your clinical research.
EuroGEMS.org is an ESHG-approved online resource which provides genetic and genomic information for both educators and individuals.
This resource can assist:
- Genetic professionals
- Non-genetics professionals
- Patients and families
- Universities and students
- Primary and secondary/high schools
Visit the accompanying app which provides explanations for the most frequently used terms in clinical genomics!
The NRDO’s new eLearning Rare Disease Education Programme for healthcare professionals is now available on HSELand!
What will you learn?
- Define what a Rare Disease is
- Explain the impact of a genetic Rare Disease diagnosis
- Identify and understand Rare Disease resources and supports available
The Toghether4RD Toolkit, launched in collaboration with ERDERA, provides resources to foster ERN-industry collaboration.
Sections in this toolkit include:
- Background knowledge – ERNs, industry and the opportunity
- Conceptualising and firming-up a collaborative idea of research
- Practical knowledge transfer – initiating and delivering a well-developed research collaboration
This data inquiry service has been established by the Rare Disease Clinical Trial Network (RDCTN) and Rare Disease Research Catalyst Consortium (RDCat) to support rare disease researchers.
It aims to match enquiries to the most relevant resources available through the RDCTN RDCat toolbox on how to handle research data
Network Partners
