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Training Overview

This 2 half-days course presents a blend of Orphan Drugs regulatory guidance and approaches to clinical trials and assessment of evidence. The course covers the legal basis for approvals, how to design studies and a look at some recent methodological ideas that have been proposed. Evidence-Based Medicine and clear reporting of studies will be thoroughly discussed.

Key Takeaways:

• 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 (and don’t we) need randomised trials?

Who should attend?

  •  Drug Product Development scientists
  • Regulatory Managers and specialists
  • Clinical Development staff
  • Statisticians
  • Pharmacovigilance scientists
  • Clinical research associates
  • Clinical trial operations staff


Dr. Simon Day
Dr. Simon DayDirector at Clinical Trials Consulting & Training

Download the Agenda

Download detailed Agenda - Clinical Trials in Rare Diseases MasterClass