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.
• 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
Clinical research associates
Clinical trial operations staff
Dr. Simon DayDirector at Clinical Trials Consulting & Training