ERDERA Learning Portal
The ERDERA Learning Portal brings together courses and resources for researchers, clinicians, patient representatives, policymakers, and industry professionals, suited to different levels of knowledge. Some of the training and resources presented are developed or endorsed by ERDERA, while others are provided by external organisations and are not ERDERA training. By supporting skills development, encouraging collaboration, and facilitating knowledge exchange, ERDERA helps connect people and expertise across the rare disease research community.
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Contact the Training & Education Work Stream. Please note that you will leave the ERDERA environment
Closed training
Materials available
Statistical and operational challenges with master protocols
This webinar introduces master protocols, explains basket, umbrella, and platform trials, and outlines the main statistical challenges and key pros and cons of using platform trials in rare‑disease research.
webinar
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advanced
- Clinical Trials | ERDERA
Closed training
Materials available
The statistical evaluation of surrogate endpoints in clinical trials
This webinar offers an overview of how surrogate endpoint evaluation has evolved over the past 30 years, highlighting its practical applications, promises, and limitations. It also explores how these methods can support clinical research in rare diseases, where traditional endpoints may be difficult to measure.
webinar
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advanced
- Clinical Trials | ERDERA
Closed training
Materials available
Composite endpoints including patient relevant endpoints quality of life
This webinar introduces methods for combining multiple endpoints and explains why generalized pairwise comparisons offer a flexible, small‑sample‑friendly approach. It highlights their key properties and the pros and cons of using them as the primary analysis in rare‑disease trials.
webinar
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advanced
- Clinical Trials | ERDERA | Pairwise Comparison
Closed training
Materials available
Does randomization matter in Clinical Trials?
This webinar explores alternative randomisation methods, explains their key properties, and helps identify which approach best suits different trial settings. It also highlights how thoughtful randomisation can strengthen clinical trial design and analysis in rare diseases.
webinar
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advanced
- Clinical Trials | ERDERA | Pre-Clinical Studies
Closed training
Materials available
Key solutions to model longitudinal natural history data with application in ataxia diseases
Modelling longitudinal natural history data is complex and often requires linear or nonlinear mixed‑effects models, especially when endpoints show ceiling or floor effects. These methods can also help justify sample size in comparative trials. Examples from ataxia illustrate these principles without formal mathematical detail.Proporcione sus comentarios sobre BizChat
webinar
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intermediate
- ataxia | Clinical Trials | ERDERA | longitudinal modelling | model-based approaches | SARA
Closed training
Materials available
Interim Analysis, Adaptations and Master Protocols
This lecture explores how interim analyses can guide decisions in confirmatory clinical trials while maintaining strict control of type I error. It introduces group sequential designs, which enable early stopping for efficacy or futility, and adaptive designs that allow modifications such as sample‑size reassessment or treatment selection. Regulatory guidance and the CONSORT extension on adaptive designs are discussed, alongside an introduction to master protocols and how data‑sharing structures can improve trial efficiency.
webinar
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intermediate
- Clinical Trials | Data Collection and Sharing | Pre-Clinical Studies | Regulatory
Closed training
Materials available
Early phase trial designs in rare diseases
Developing treatments for rare diseases is challenging due to small, diverse patient populations and limited knowledge of disease progression. This tutorial introduces key statistical methods for early‑phase trials, focusing on adaptive phase 1 and phase 1/2 designs that balance learning about dose–toxicity and early efficacy while protecting participants. It highlights how these approaches improve dose‑finding and strengthen early clinical development.
webinar
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intermediate
- Clinical Trials | Early Diagnosis | ERDERA | Pre-Clinical Studies
Closed training
Materials available
Bayesian Methods in Clinical Research
Bayesian methods have grown rapidly in popularity for their ability to handle complex models and offer intuitive results. This course introduces Bayes’ theorem, priors, and posteriors through simple examples, shows how Bayesian approaches are used across research fields, and demonstrates their value in randomized clinical trials. Its aim is to give participants a smooth, practical introduction to Bayesian statistics.
webinar
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intermediate
- Bayesian | Clinical Trials | ERDERA