Orphan Drugs Clinical Trials
Orphan Drugs Clinical Trials

Course Description

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.
  • 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?
  • 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 Clinical Trials

3 - 4 June 2024, Live Online Training
€1.650,00
Dr. Simon Day

Simon has spent 30 years working in clinical trials, mostly in the pharmaceutical industry but also including five years at the UK and European regulatory agencies.

START TIMES

1:00 PM Vienna time

12:00 PM London time

7:00 AM New York time

4:00 AM Los Angeles time

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Course Description

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.
  • 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?
  • 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?

"Trainer is very competent and has the complete knowledge of the matter."

– Senior Process Expert, B. Braun

"It is not at all frequent to find this grade of preparation and reaction to specific cases such as the ones presented. I'm enthusiastic! Thanks really."

– Tech Transfer Lead, Novartis

"Thank you for sharing all the links and informative material. Very professional organization and execution."

– Manufacturing Manager, Thermo Fisher Scientific

"The trainer was very knowledgeable about the topics and was able to answer all questions."

– Drug Product Subject Matter Expert, Pfizer

"Excellent work of the lecturer, good materials, clear explanations."

– Clinical Pharmacologist, Boehringer Ingelheim

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.

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