Overview
This talk challenges apparently simple notions of when to do (or not to do) randomised controlled clinical trials. Beginning with a short case study about Sasha, aged 3, who has cerebral palsy, we explore the constraints and over-simplification of the mantra that is "Evidence Based Medicine" (EBM). This is particularly pertinent when we are trying to study treatments for rare diseases (sometimes called "orphan drugs"), where it is often seemingly impossible to do properly powered randomised controlled trials.
Why you should Attend
We review the poor success rates in drug development; the critical importance of randomisation; the problems looming as a result of well-intentioned but short-sighted legislation; why Bayesian statistics (although not necessarily Bayesian statisticians) might lead us down the wrong road; and at relationships between Evidence Based Medicine and Sir Austin Bradford-Hill’s so-called "criteria" for determining causality.
The webinar concludes with suggestions on when to randomise, and when not.
Areas Covered in the Session
- Clinical Trials
- Randomization
- Evidence-Based-Medicine
- Bradford-Hill criteria
- Strength of Evidence
- Natural Course of Disease
Learning Objectives
- Understating causality and strength of evidence
- Understand the benefits and problems with randomizing very sick patients
Who Will Benefit
- Those working in area of rare Diseases
- Pharma/Biotech Industry
- Clinical Trialists
- Patients and Patient Organisations
Speaker Profile
Dr. Simon Day 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 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 in Medicine, on the editorial board of Translational Sciences of Rare Diseases, and previously joint editor of the Journal of the Royal Statistical Society; he has also served on editorial boards on many other journals, including Pharmaceutical Statistics, Controlled Clinical Trials and British Journal of Clinical Pharmacology.
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. 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 for Clinical Trials" and is joint editor of the "Textbook of Clinical Trials", both published by Wiley.
Instructor
Dr. Simon Day