Orphan Drugs Clinical Trials

Place
London, UK
Site visit
Dates
TBA & In-house
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

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.

Aims

Learning & Key takeaways

  • MA 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?

Programme

  • Training materials will be provided in printed and digital form
  • All delegates will receive printed and LinkedIn certificates
  • 5 hours of networking with industry peers
  • Networking cocktail reception

Request Full Agenda

Trainers

Dr. Simon Day

CEO

Clinical Trials Consulting & Training

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. He now works as a statistical and regulatory consultant to pharmaceutical and biotechnology companies around the world. He specialises in training and consulting on drug development programmes, scientific advice/end of Phase II meetings and preparations for oral explanations and advisory committees. He is particularly well known for his work in the area of developing treatments for rare diseases.

He is a former president of the International Society for Clinical Biostatistics.  He is joint editor of Statistics inMedicine, on the editorial board of Translational Sciences of Rare Diseases, and has previously served on many other editorial boards. In 2012 he was elected a Fellow of the Society for Clinical Trials. He has published widely in statistical and medical journals, is author of one book “Dictionary forClinical Trials” and is joint editor of the “Textbook of Clinical Trials”, both published by Wiley.

Simon has served on a variety of data monitoring committees both for industry- and government- sponsored trials. He is chairman of the External Advisory Panel for the Department of Statistics at Oxford University and an Associate on the faculty at Johns Hopkins University in Baltimore. He formerly served as vice-Chairman of the West London Research Ethics Committee. He has given numerous lectures and courses on statistics and clinical trials all around the world, including courses at the FDA on development and regulatory assessment of orphan drugs.

Dr. Simon Day

CEO

Clinical Trials Consulting & Training

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. He now works as a statistical and regulatory consultant to pharmaceutical and biotechnology companies around the world. He specialises in training and consulting on drug development programmes, scientific advice/end of Phase II meetings and preparations for oral explanations and advisory committees. He is particularly well known for his work in the area of developing treatments for rare diseases.

He is a former president of the International Society for Clinical Biostatistics.  He is joint editor of Statistics inMedicine, on the editorial board of Translational Sciences of Rare Diseases, and has previously served on many other editorial boards. In 2012 he was elected a Fellow of the Society for Clinical Trials. He has published widely in statistical and medical journals, is author of one book “Dictionary forClinical Trials” and is joint editor of the “Textbook of Clinical Trials”, both published by Wiley.

Simon has served on a variety of data monitoring committees both for industry- and government- sponsored trials. He is chairman of the External Advisory Panel for the Department of Statistics at Oxford University and an Associate on the faculty at Johns Hopkins University in Baltimore. He formerly served as vice-Chairman of the West London Research Ethics Committee. He has given numerous lectures and courses on statistics and clinical trials all around the world, including courses at the FDA on development and regulatory assessment of orphan drugs.

Tour info

Testimonial

"The interaction between training participants is great!" - Elcam Medical

Price per Delegate (EUR)

1898.00
Register now

Request Full Agenda

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Contact us

Lydia Makori

l.makori@symmetric.events
+421 222 200 543