Pre-Conference Tutorials, Monday, March 8, 2010, 09:00

This is a preliminary programme. New speakers and sessions will be added. Visit www.diahome.org for the most recent additions to the programme.
Pre-Conference Tutorials, Monday, March 8, 2010, 09:00 - 12:30
TUTORIAL 8
ADAPTIVE DESIGNS FOR CONFIRMATORY CLINICAL TRIALS
Norbert Benda, Statistical Methodologist, Novartis Pharma AG, Switzerland
This tutorial will give an introduction to the theory and practice of adaptive
designs for pivotal clinical trials. Adaptive designs allow for mid-course design
modifications such as the adjustment of sample size, the dropping of treatment
arms or the selection of a subpopulation. We will review and discuss statistical
methodology that allows such adaptations without compromising the overall
type I error rate. All methods will be illustrated by examples. Several case
studies will be presented, explaining in detail both methodological and practical
issues which arise in designing and analysing an adaptive clinical trial.
Learning Objectives
At the conclusion of this tutorial, attendees will be able to:
• Understanding the principles, opportunities and challenges for adaptive
designs
• Understand operational considerations
Target Audience
Clinical project team members within drug development
TUTORIAL 9
IMPLEMENTATION OF THE EU VARIATION REGULATION:
WHY – WHEN – WHAT
Merete Schmiegelow, Director, Regulatory Intelligence, Novo Nordisk A/S,
Denmark
Peter Bachmann, Senior Expert, European Drug Regulatory Affairs and German
Member of the CMD(h), BfArM, Germany
The EU variation regulation came into force 1 January, 2009 and is mandatory
to use from 1 January, 2010. What is the first impression on whether the
implementation will provide a simpler, clearer and more flexible regulation for
variations within Quality – Safety and Efficacy?
Learning Objectives
At the conclusion of this tutorial, attendees will be able to obtain an overview
of:
• Key issues from the EU variation regulation and implementation guidelines
• How is the EU variation regulation compared to the US system?
• Whether the implementation will give a simpler, clearer and more flexible
regulation for variations within Quality – Safety and Efficacy?
• Changes to biologicals compared to chemical entities – any differences?
• What is a regulatory pre-approved protocol?
Target Audience
People involved in lifecycle management regulatory issues of late-stage
development drug products and lifecycle management of approved products
within quality/production, safety, clinical and regulation.
TUTORIAL 10
ORPHAN DRUGS IN THE EU: FROM DESIGNATION TO MARKETING
AUTHORISATION
Katrin Rupalla, Associate Director, Regulatory Affairs Europe, Celgene
International SARL, Switzerland
Jordi Llinares, Head of Orphan Medicines, European Medicines Agency, EU
In this tutorial you will hear about the orphan drug legal framework in general
and what are the latest updates. In addition, this tutorial is meant to provide an
overview of the orphan drug application process from designation to approval
of marketing authorisation and its practical implications based on recent
examples. This also includes in-depth review and analyses of topics such as
orphan market exclusivity, similarity and significant benefit. Speakers will
include experts from the European authorities, but you will also hear from
industry representatives about the strategic aspects, experiences and learnings
with regard to regulatory framework for orphan drugs in the EU.
Learning Objectives
At the conclusion of this tutorial, attendees will be able to:
• Apply the information they have obtained on procedures and requirements for
an orphan drug application in the EU and US
• Identify and respond to potential issues that may arise during an orphan
designation procedure
Target Audience
This tutorial will be of interest to all those who would like to hear about the
latest update of the orphan drug legal framework in the EU and most recent
experience with regard to practical implications. They will be, in general,
professionals from drug regulatory affairs, clinical science and public affairs.
TUTORIAL 11
SIGNAL DETECTION METHODS FOR BEGINNERS – OVERVIEW AND CASE
STUDIES
Steve Jolley, Principal, SJ Pharma Consulting, USA
Jacinta Aniagolu, Director, Pharmacovigilance & Risk Management,
Synowledge, LLC, USA
This tutorial will provide a theoretical and methodological review of the
application of signal detection and data mining techniques to safety
surveillance. An overview of strategies and specific situation applications will be
presented.
Highlights:
• Recommended approach to signal detection and use of data mining
techniques
• Use of visualisation tools to enhance signal detection
• Comparison of approaches for a large and small company
Specific topics covered will include:
• Analysis of demographics, risk assessment, dose effects, product interactions,
and differences from known background rates
• Strategies for incorporating all relevant data sources, including clinical trials,
individual case reports, PSURs, external databases (FDA, WHO), and registries
• Understanding the benefit/risk ratio of a product and how to discover product
adverse event relationships
• Measuring the strength of the relationship of the adverse event and the drug
using the Bayesian Confidence Propagation Neural Network algorithm
(BCPNN)
• Illustration of the power of technology that can not only provide overall
analysis, but also “drill down” to specific patient records
Learning Objectives
At the conclusion of this tutorial, attendees will be able to:
• Recognise the basic concepts of data mining and principles of signal detection
• Outline how to apply these techniques within their company
• Discuss data mining techniques to analyse large volumes of adverse event
report data
Target Audience
Clinical safety professionals involved in pharmacovigilance;
pharmacoepidemiology; regulatory affairs; quality assurance;
medical product safety assessment; labelling
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