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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0 hours — 100 hours

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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

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

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

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

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

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

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