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Joint DIA/AEMPS Statistics Workshop

Nov 10 2014 9:00AM - Nov 11 2014 4:00PM | Tryp Barcelona Apolo Avinguda del Parallel, 57-59 08004 Barcelona Spain

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Overview 

Lifecycle management is a widely accepted concept for the development and marketing of medicines. At each step of this process, pre- and post-licensing, statisticians work to develop methods that can improve efficiency and can enhance decision-making through optimal study design, analysis and inference. The discussions at this workshop will consider not only the methods themselves, but also how to best implement them in a regulatory context.

We will focus on:

  • Different views on the choice of an appropriate estimand and related design options and analysis models in the context of the definition of a trial objective taking into account possible non-adherence to the assigned treatment
  • Statistical aspects of components required for MA in the development of a biosimilar in view of recent attention to methodological issues in the assessment of biosimilarity and biological products due to differences between biosimilars and small molecule generics
  • Illustrating the use of innovative methods to identify subgroups that benefit in particular from a new treatment and how to integrate this knowledge into clinical development plans through adaptive and enrichment designs to develop personalised / stratified treatment strategies

Featured Topics 

  • Lack of adherence and the choice of the right estimand in clinical trials
  • Statistical approaches in biosimilarity
  • How precise can precision medicine be?
  • Key topics in regulatory statistics

Who Should Attend 

Professionals with an interest in the application of, and research into, statistics in the drug development process from the pharmaceutical industry, academia, regulatory and governmental agencies, as well as contract research organisations.

Hotel & Travel 

DIA has blocked a limited number of rooms at the following hotel:

TRYP Barcelona Apolo Hotel
Avinguda del Paral·lel, 57-59,
08004, Barcelona, Spain
Tel: (34) 93 343 30 00
Fax: (34) 93 443 0059
E-mail: tryp.apolo@melia.com
 
at the rate of:
EUR 85.00 single use
incl. of breakfast, excl. of VAT and city tax

To make your reservation, please click here: http://meetings.melia.com/en/DIAEUROPE.html

Important: The room rate is available until 04 October 2014 or until the group block is sold-out, whichever comes first.
In case of no-shows the hotel is authorised to charge the full amount corresponding to the duration of your stay.

Contact Information 

Registration Questions
Phone.: +41 61 225 51 51
Fax: +41 61 225 51 52
Monday-Friday 8:00-17:00 CET
diaeurope@diaeurope.org

Agenda and Event Logistics
Magdalena Lewandowska, Event Manager
Phone: +41 61 225 51 65
Fax: +41 61 225 51 52
magdalena.lewandowska@diaeurope.org

Program Committee 

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Agenda  

Day 1 Monday, Nov 10, 2014

  • 12:30PM - 2:00PM (Central Europe Standard Time)

    REGISTRATION AND WELCOME COFFEE
  • 2:00PM - 3:30PM (Central Europe Standard Time)

    Session 1: LACK OF ADHERENCE AND THE CHOICE OF THE RIGHT ESTIMAND IN CLINICAL TRIALS


    Session Chair(s):

    • Norbert Benda, PhD
      Head of Biostatistics and Special Pharmacokinetics
      Federal Institute For Drugs and Medical Devices (BfArM), Germany
    • William Malbecq, DrSc
      Executive Director Biostatistics
      MSD Europe, Inc., Belgium

    Recently, an ICH concept paper on choosing appropriate estimands and defining sensitivity analyses in confirmatory clinical trials was drafted. In settings where pronounced lack of adherence to the study protocol is expected, the difference between the ideal treatment effect, if the medication is taken as directed, and the treatment effect if the medication is taken as observed is crucial in the assessment of the drug’s benefit. The handling of missing data and the use data observed after treatment discontinuation or change is strongly related to the targeted estimand. An estimand is what is being estimated and precisely defines a treatment effect regarding population, outcome measure, and the parameter defined by the underlying probabilistic model either under the assumption of perfect treatment adherence or incorporating the actual adherence. The session will discuss the use and definition of different estimands and the consequences with respect to study design and analysis in the context of the drug approval process.

    Speaker(s):

    • Estimands and Their Role in Clinical Trials: Defining suitable primary scientific questions of interest
      Mouna Akacha
      Statistical Methodologist
      Novartis Pharma AG, Switzerland
    • The Estimand: An ugly necessity, a pie in the sky or a useful tool?
      Michael P O'Kelly, PhD, MA
      Senior Director, Centre for Statistics in Drug Development, Innovation
      Quintiles Ireland Ltd., Ireland
    • Statistical Models for De Facto Estimands
      James Henry Roger, PhD, MA
      Director, Statistics and Programming
      United Kingdom
  • 3:30PM - 4:00PM (Central Europe Standard Time)

    COFFEE BREAK
  • 4:00PM - 4:30PM (Central Europe Standard Time)

    Session 1 continued: LACK OF ADHERENCE AND THE CHOICE OF THE RIGHT ESTIMAND IN CLINICAL TRIALS


    Session Chair(s):

    • Norbert Benda, PhD
      Head of Biostatistics and Special Pharmacokinetics
      Federal Institute For Drugs and Medical Devices (BfArM), Germany
    • William Malbecq, DrSc
      Executive Director Biostatistics
      MSD Europe, Inc., Belgium

    Speaker(s):

    • Applicability and appropriateness of de-facto and de-jure estimands in drug approval
      Ann-Kristin Leuchs
      Statistician
      Bfarm, Germany
    • Estimands and the Handling of Missing Data: Regulatory Evolution
      David Brown
      Statistical Assessor
      MHRA, United Kingdom
  • 4:30PM - 5:30PM (Central Europe Standard Time)

    Panel Discussion with Questions and Answers

    Discussant(s):

    • Concha Prieto
      Spanish Agency for Medicines and Medical Devices (AEMPS), Spain
    • Erik Cobo
      Profesor Titular de Universidad
      Universidad Politecnica Catalunya, Spain
    • Rosa Lamarca
      Head of Biometry
      Laboratorios ALMIRALL, Spain
  • 5:30PM - 6:30PM (Central Europe Standard Time)

    NETWORKING RECEPTION
  • 6:30PM - 6:30PM (Central Europe Standard Time)

    END OF DAY 1

Day 2 Tuesday, Nov 11, 2014

  • 9:00AM - 9:30AM (Central Europe Standard Time)

    Session 2: STATISTICAL APPROACHES IN BIOSIMILARITY


    Session Chair(s):

    • Beatriz Seoane Núñez
      Expert Statistician
      AstraZeneca, Spain
    • Ferran Torres, PhD
      Scientific Director, Biostatistics and Data
      Clinic Hospital of Barcelona, Spain

    A Biosimilar medicine is a growing field. The number of regulatory applications has increased in recent years. This session will highlight the features of the study design options for biosimilar efficacy trials. The choice of margins, their reliability and the different statistical and regulatory considerations associated with them will be some of the topics that will be discussed in this session from both a regulatory and industry point of view.

    Speaker(s):

    • Statistical Challenges in Biosimilarity - a regulator’s view
      Peter Volkers
      Biostatistician
      Paul Ehrlich Institute, Germany
    • Statistical Challenges and Current Experience in Biosimilarity Trial Designs
      Yulan Li
      Biostatistician
      Novartis Pharmaceuticals Corporation, United States
  • 9:30AM - 10:30AM (Central Europe Standard Time)

    Panel Discussion with Questions and Answers

    Discussant(s):

    • Gonzalo Calvo Rojas, PhD
      Consultant in Clinical Pharmacology
      Hospital Clinic Barcelona, Spain
    • Antonio Gomez Outes, MD, PhD, MSc
      Clinical Assesor. Cardiovascular, Respiratory and Gastrointestinal Drugs,
      Spanish Agency for Medicines and Medical Devices (AEMPS), Spain
  • 10:30AM - 11:00AM (Central Europe Standard Time)

    COFFEE BREAK
  • 11:00AM - 12:30PM (Central Europe Standard Time)

    Session 3: HOW PRECISE CAN PRECISION MEDICINE BE?


    Session Chair(s):

    • Hans-Ulrich Burger
      Senior Director of Biostatistics
      F. Hoffmann-La Roche Ltd., Switzerland
    • Tim Friede, PhD
      Chair of Department of Medical Statistics
      Georg-August-University Goettingen, Germany

    Precision medicine becomes more and more important in drug development. Among the many topics possible we picked out two, biomarker identification and seamless enrichment designs. There will be a short introduction into the topic, an overview and different methods, then two examples from industry and a regulatory evaluation. Finally the two sessions will be rounded up by a panel discussion.

    Speaker(s):

    • Adaptive Enrichment Designs for Stratified Medicine
      Tim Friede, PhD
      Chair of Department of Medical Statistics
      Georg-August-University Goettingen, Germany
    • How Good is Your Biomarker? Comparison of Development Plans for Confirmatory Biomarker Enrichment
      Kaspar Rufibach
      Biostatistician, Oncology Biostatistics
      F. Hoffmann-La Roche AG, Switzerland
    • Bayesian Approaches to Enrichment Designs
      Heinz Schmidli, PhD, MSc
      Senior Expert Statistical Methodologist
      Novartis Pharma AG, Switzerland
  • 12:30PM - 2:00PM (Central Europe Standard Time)

    LUNCH
  • 2:00PM - 2:30PM (Central Europe Standard Time)

    Session 3 continued: HOW PRECISE CAN PRECISION MEDICINE BE?

    Speaker(s):

    • Regulatory Views of Precision Medicine
      Norbert Benda, PhD
      Head of Biostatistics and Special Pharmacokinetics
      Federal Institute For Drugs and Medical Devices (BfArM), Germany
    • Key Issues in Enrichment Designs from a Development Point of View: An Introduction to the Panel Discussion
      Hans-Ulrich Burger
      Senior Director of Biostatistics
      F. Hoffmann-La Roche Ltd., Switzerland
  • 2:30PM - 3:30PM (Central Europe Standard Time)

    Panel Discussion with Questions and Answers

    Discussant(s):

    • Fernando de Andres
      Universidad Complutense Schools of Dentistry and of Medicine,, Spain
  • 3:30PM - 4:00PM (Central Europe Standard Time)

    COFFEE BREAK
  • 4:00PM - 5:30PM (Central Europe Standard Time)

    Session 4: KEY TOPICS IN REGULATORY STATISTICS


    Session Chair(s):

    • Norbert Benda, PhD
      Head of Biostatistics and Special Pharmacokinetics
      Federal Institute For Drugs and Medical Devices (BfArM), Germany
    • Hans-Ulrich Burger
      Senior Director of Biostatistics
      F. Hoffmann-La Roche Ltd., Switzerland

    This session will pick up two relevant topics which are important for regulatory bodies as well as for industry. For each of the topics we will have one presentation from a more regulatory view and a panel discussion. The first topic will deal with issues in developing new therapies in small populations, rare diseases and paediatric indications. The second issue will discuss issues around censoring for time to event analyses and what the right primary analysis should be.

    At the end of the session panel discussion with Q&A will take place.

    Speaker(s):

    • Overview on Issues in Developing New Therapies in Small Populations, Rare Diseases and Paediatric Indications
      Kit B. Roes, PhD
      Professor of Biostatistics; Director of Quality and Patient Safety
      University Medical Center Utrecht, Netherlands
    • 20 Years of ICH E4 -Dose-Response / Dose-Finding Quo Vadis?
      Andrew Peter Grieve, DrSc, PhD, MSc
      Senior Vice President Clinical Trial Methodology
      Aptiv Solutions, Germany
    • Statistical Analysis in Alzheimer’s Disease
      David Brown
      Statistical Assessor
      MHRA, United Kingdom
  • 5:30PM - 5:30PM (Central Europe Standard Time)

    END OF CONFERENCE

Registration Fees 

Other Fees

Student
€200.00

Member

Member Academia
€710.00
Member Government
€710.00
Member Standard
€1420.00

Non-Member

NonMember Academia
€840.00
NonMember Government
€840.00
NonMember Standard
€1550.00
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