Orphan Drugs Clinical Trials
22 – 23 November 2021, online training
This training course presents a unique blend of Orphan Drugs regulatory guidance and clinical trials strategies. Delegates will explore the legal basis for approvals, learn how to design a study and which methodology to use. Evidence-Based Medicine data analysis, conclusions, and reporting will be thoroughly discussed.
Who should attend?
Rare Disease Specialists, Clinical Research Associates, Clinical Operations Analysts, Clinical Diagnostic Specialists, Pharmacovigilance Scientists, Regulatory Managers and Specialists, Process Science Research & Development, Drug Product Development Scientists, Orphan Drug Products Formulators, Quality & Compliance Associates
- EMA and FDA expectations for Orphan Drugs clinical studies
- The Importance of avoiding Bias and Regression to the Mean
- Working with Evidence Based Medicine and Clinical trials
- How to design a study and which methodology to use?
- Practical implementation of regulatory pathways
- Where do we need randomised trials?
- Orphan Drugs EU and US regulatory guidance
- Legal basis for approvals (Regulatory pathways) & Working Groups
- Trial concept, design and conclusions
- The Importance of Precision and Confidence Intervals
- Study designs and methodology considerations
- What constitutes convincing evidence?
- Training materials will be provided in digital form
- All delegates will receive digital and LinkedIn certificates