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

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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 12
ANALYSIS OF SAFETY DATA FROM CLINICAL TRIALS
Jürgen Kübler, Global Head, Statistical Safety Sciences, Novartis Pharma AG,
Switzerland
Joachim Vollmar, Executive Consultant, International Clinical Development
Consultants, USA
This tutorial is a combination of theory, guidelines, practical considerations and
real-life solutions for those working in the clinical development environment
(pharmaceutical, biotech industry or CRO). The aim of this course is to provide
a basic understanding of the underlying methodology and the current
guidelines on safety data. Aspects regarding the planning of clinical trials as
well as the problems and pitfalls during the analysis of safety data will be
presented. The presentations will also include case studies.
Learning Objectives
At the conclusion of this tutorial, attendees will be able to:
• Understand relevant guidelines and regulatory requirements
• Contribute to safety analysis plans
• Assess statistical safety analyses
• Identify pitfalls in safety analyses
Target Audience
Clinical researchers; drug safety specialists; medical writers; investigators;
biostatisticians; and project managers
TUTORIAL 13
THE "QUALITY-BY-DESIGN" INITIATIVE – A LOOK INSIDE FROM MULTIPLE
ANGLES
Gert Thurau, Associate Director, Merck & Co., Inc., USA
Quality-by-Design (QbD) is a concept that has the potential for introducing
transformational changes to the way pharmaceutical products are developed,
registered and manufactured. With the regulatory aspect of QbD anchored
around the ICH guidelines ICH Q8, Q9 and Q10, industry has embraced QbD as
beneficial for both the patient and their business processes in R&D and
manufacturing. On the technical level QbD supports concepts like risk
management, continuous improvement and the overall modernisation of
pharmaceutical manufacturing. This new concept is also expected to be
implemented into the EU Variations framework via the revised legislation. A
reality check of the much described vision will be performed, based on recent
implementation of QbD concepts into practice by several pharmaceutical
companies both in newly developed and in line products. This tutorial will give
an introduction to the overall framework of QbD as well as provide an overview
of regulatory and technical approaches currently discussed and utilised to push
the envelope beyond the previously accepted boundaries, including the
utilisation of enablers like risk management and Process Analytical Technology
(PAT). Both of these previously existing approaches have been recognised as
core tools for QbD practitioners, leading to true benefits like continuous
improvement or real-time product release testing. Presentations on both
industry and regulatory interpretations and expectations will be combined with
an overview of currently available guidance and interpretation. Finally, real-life
examples of QbD will help to illustrate what opportunities QbD is opening up
and where its real potential lies for the enhancement of the post-approval
framework to ensure the safety and benefit of patients.
Learning Objectives
At the conclusion of this tutorial, attendees will be able to:
• Identify and analyse Quality by Design (QbD) approaches to product and
process development
• Assess the current regulatory positions on QbD
• Define how the concept could be used in their own work to further patient
safety and benefits
Target Audience
Physicians, pharmacists and pharmaceutical scientists involved in product
(synthesis and formulation) and manufacturing process development;
regulatory personnel involved in drug registration (CMC); manufacturing
personnel with innovation needs; regulators with interest in real-life QbD
examples
TUTORIAL 14
RECENT EUROPEAN REGULATORY KEY ASPECTS OF BIOPHARMACEUTICALS
Gabriele Dallmann, Director Biopharmaceuticals, NDA Regulatory Science Ltd.,
Germany
Paul Chamberlain, Principal, bioLOGICA Consulting, France
The tutorial provides the attendees with an overview of recent key aspects of
activities in the area of biopharmaceuticals. It covers newly suggested, drafted
and finalised regulatory guidelines related to biopharmaceuticals, the main
activities of committees and working parties of the European Medicines Agency
with a focus on the first year of the CAT, and decisions on marketing
authorisation applications of biopharmaceuticals via the centralised procedure.
Based on this, the discussion will include an overview of recent marketing
authorisations and refusals and their reasons, the implementation of the
certification process for ATMPs, the experience with first authorisations of
ATMPs, the preparation of a regulatory guideline on biosimilar monoclonal
antibodies, an update on the plasma master file and pandemic influenza vaccine
activities, feedback on experience with Quality-by-Design or platform
technology dossiers and challenges in the investigation of comparability and
immunogenicity upon introduction of changes into the manufacturing process.
Learning Objectives
• Recognise the current trends in biopharmaceutical-related regulatory
guideline developments and decisions on marketing authorisations in Europe
• Appraise the activities of the CAT and biopharmaceutical-related working
parties of the CHMP
• Interpret recently developed guidelines in their impact on scientific data
requirements
• Estimate how these trends impact current development projects in your
company
Target Audience
Experts of regulatory affairs, R&D and process development departments,
involved in the investigation of new biopharmaceuticals including ATMPs and in
the preparation of CTDs for marketing authorisation applications via the
centralised procedure
TUTORIAL 15
AFTER THE PIP DECISION: MODIFICATION OF THE AGREED PIP, VALIDATION
AND COMPLIANCE CHECK
Paolo Tomasi, Head of Paediatric Medicines, European Medicines Agency, EU
Behrouz Kassai, Associate Professor Department of Clinical Pharmacology, Coordinator
Paediatric Clinical Investigation Centre/INSERM, Coordinator for
EPICIME, University of Lyon, France
This tutorial is aimed at illustrating the steps that follow the European Medicines
Agency decision on an agreed PIP (or on a waiver), including the request for
modification of an agreed PIP, applications for a new PIP (for the same
product), and the procedures at the time of validation/compliance check.
Learning Objectives
At the conclusion of this tutorial, attendees will be able to:
• Decide whether an agreed PIP requires an application for modification
• Judge when a new PIP application should be submitted, versus a request for
modification of the agreed PIP
• Differentiate between validation and compliance check
• Compose an application to the PDCO requesting an interim or final