Summary
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
Learning Objectives
- 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?
Key Topics
- 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?
Our online training experience includes
- Our client zone – a single source for all training materials as well as pre and post-training communication
- Live interactive format via the Zoom platform
- Direct interaction with the trainer
- Q&As, case studies, breakout rooms
- Revisit recorded sessions for 7 days
- Digital and LinkedIn certificates
Trainers
Orphan Drugs Clinical Trials
Download the full training agenda to reveal complete session details, training takeaways, case studies, daily schedule, special features and full trainer bio.
