Returning to London for its 7th year is Adaptive Designs in Clinical Trials conference

1. Book by 19th December and save £400 • Book by 30th January and save £200 • Book by 27th February and save £100
SMi Presents the 7th Annual Conference on…
Adaptive Designs
in Clinical Trials
BUSINESS BENEFITS FOR 2015:
• Understand the objectives of Adaptive
Licensing and MAPPs as the “breakthrough”
designations to accelerate the approval of new
medicines.
• Learn how Bayesian Belief Networks can
support reliability management and decision
making.
• Discover novel techniques in CARA and
Seamless Adaptive Designs and Bayesian
Bandit models to optimise efficiency in dose-finding
strategies.
• Benefit from the talks of industry leaders and
CEOs in clinical leadership and learn how real
world evidence can improve adaptive trial
designs.
20th - 21st
APRIL
2015
Marriott Regents Park Hotel, London, UK
PLUS TWO INTERACTIVE HALF-DAY POST-CONFERENCE WORKSHOPS
Wednesday 22nd April 2015, Marriott Regents Park Hotel, London, UK
Bayesian Adaptive Clinical Designs and Strategies
Workshop Leader: Dr. Sophie Carr, MD and Principal Analyst,
Bays Consulting Ltd.
8.30am – 12.30pm
Winning with Adaptive Trial Strategies and Clinical Compliance
Across the Evolving International Regulatory Landscape
Workshop Leader: Robert Clay, Consultant, Highbury Regulatory
Science, Chief Regulatory Officer, Kinapse, Board Director, TOPRA
1.30pm – 5.30pm
CHAIRS FOR 2015:
Dr. Graham Clarke, Senior Director and Head of
Respiratory & Inflammation Early Clinical
Development, Quintiles
Robert Clay, Consultant, Highbury Regulatory Science,
Chief Regulatory Officer, Kinapse; Board Director,
TOPRA
Professor Emeritus Tamás L. Paál, Faculty of Pharmacy,
Institute of Drug Regulatory Affairs, University of
Szeged, Hungary and Regulatory Adviser, Hungarian
Medicines Agency
KEY SPEAKERS INCLUDE:
• Dr. Sophie Carr, MD and Principal Analyst, Bays Consulting Ltd.
• Loïc Darchy, Head of Statistical Methodology Group,
Sanofi R&D
• Dr. Steve Coad, Reader in Statistics, Queen Mary University
• Han-Joo Kim, PhD, Associate Director, Biostatistics, Oncology
PCU, Eisai Inc.
• Alex Sverdlov, Associate Director of Biostatistics, EMD Serono Inc.
@SMIPHARM
www.adaptivedesigns.co.uk
Register online or fax your registration to +44 (0) 870 9090 712 or call +44 (0) 870 9090 711
EARLY
BIRDS

2. 7th Annual Adaptive Designs in Clinical Trials
Day One | Monday 20th April 2015
8.30 Registration & Coffee
9.00 Chairman's Opening Remarks
Dr. Graham Clarke, Senior Director and Head of Respiratory &
Inflammation Early Clinical Development, Quintiles
Statistical Modelling and Adaptive Tools
to Leverage Clinical Development
OPENING ADDRESS
9.10 Enhancing study design and biomarker read-outs in early
clinical development
•Design strategies in early clinical phase - Phase I/II drug
development process
•Implementation of biomarkers and validating end points -
design protocols, optimisation and patient strategies in
precision or personalised medicine.
•Highlight the main challenges to ongoing developments and
global efforts, including biomarker collection and data loss
issues
Dr. Graham Clarke, Senior Director and Head of Respiratory &
Inflammation Early Clinical Development, Quintiles
9.50 Succeeding in Bayesian adaptive designs
•Understand the key components of Bayesian
Scientific
statistics to enhance decision - making
Spotlight
•Predictive classifiers in the Bayesian belief network
•Bayesian survival models and predicting time events in clinical
trials
•Operational-risk management: A Bayesian approach to
modelling risk and uncertainties
Dr. Sophie Carr, MD and Principal Analyst, Bays Consulting Ltd.
10.30 Morning Coffee
11.00 Group Sequential Enrichment Design incorporating subgroup
selection - Application to the optimisation of the cut-off for a
continuous predictive biomarker with time-to-event endpoints
•Adapting the Group Sequential Enrichment Design (GSED)
methodology & the Combination Tests (CT) methodology to
address important problems
•Introducing new performance indicators to assess the
soundness of the subpopulation selection
•Exploring the operating characteristics of GSED and CT designs
via simulations under a wide range of scenarios
Loïc Darchy, Head of Statistical Methodology Group, Sanofi R&D
11.40 Real world patient data in adaptive clinical designs
•How real time data monitoring gives higher quality, lower costs
and happier patients
•How real world data can improve adaptive trial design
•Structure and regulatory challenges and requirements for best
clinical practices set out by the European Commission
•Case studies related to current research and the organization
Dr. Mohammad Al-Ubaydli, CEO, Patients Know Best
12.20 Networking Lunch
1.30 Afternoon Chairman’s Opening Remarks
Professor Emeritus Tamás L. Paál, Faculty of Pharmacy, Institute
of Drug Regulatory Affairs, University of Szeged, Hungary and
Regulatory Adviser, Hungarian Medicines Agency
Clinical Innovation through Design, Leadership and Management
1.40 KEYNOTE ADDRESS:
Scientific
Spotlight
•Outlook of clinical trials in the CEE region,
implementation of adaptive protocols
•Nature of regulatory framework for these markets
•Challenges for harmonization in clinical trial legislation across
the entire European Union member states
•Adaptive clinical trial design and adaptive licensing
Professor Emeritus Tamás L. Paál, Faculty of Pharmacy, Institute
of Drug Regulatory Affairs, University of Szeged, Hungary and
Regulatory Adviser, Hungarian Medicines Agency
2.20 Clinical trials in neurodegenerative disease
•Clinical trials in Neurodegenerative diseases: A zoom on
Alzheimer and Parkinson’s disease
•Patient selection and management
•Patient care and perspectives
•Challenges and ongoing research developments
Dr. Filippo Baldacci, Medical Doctor, Researcher, University of Pisa
3.00 Afternoon Tea
3.30 Diffusion of innovation through efficient, yet practical designs
and statistical leadership in drug development
•Efficient and practical trial design and strategies for leading
cross-functional team
•Multiple testing strategies
•Statistical modelling and analysis: linking this with clinical outcome
•Case studies related to research activities
Han-Joo Kim, PhD, Associate Director, Biostatistics, Oncology
PCU, Eisai Inc.
4.10 FOCUS4: Designing and delivering an Adaptive-Biomarker
driven Multi Arm Mullti Stage (MAMS) cancer trial
•Key barriers in translational research
•Designing and Delivering an Adaptive-Biomarker driven trial to
time and target
•Advantages and Challenges of implementing novel methodologies
•Regulatory compliance and challenges from an academic
perspective
Dr. Kai-Keen Shiu, FOCUS4 Trial Physician MRC Trials Unit, UCL
4.50 Panel Discussion: A rationale on adaptive
designs in clinical trials
•How are adaptive designs defined and best
identified to improve clinical trials?
•How costly are continuous trial modifications and
re-assessment in clinical trials? Does this hamper the adaptive
nature of trial design?
•What are the perceptions on real world data integration to
increase flexibility and decision making in trials.
•How significant are adaptive designs in each of the respective
research areas on the panel?
•Compare and discuss biomarker development initiatives in the
respective research areas on the panel: Trends and challenges.
Panel leader:
Professor Emeritus Tamás L. Paál, Faculty of Pharmacy, Institute
of Drug Regulatory Affairs, University of Szeged, Hungary and
Regulatory Adviser, Hungarian Medicines Agency
Panellists:
Dr. Graham Clarke, Senior Director and Head of Respiratory &
Inflammation Early Clinical Development, Quintiles
Dr. Kai-Keen Shiu, FOCUS4 Trial Physician MRC Trials Unit, UCL
Dr. Mohammad Al-Ubaydli, CEO, Patients Know Best
Loïc Darchy, Head of Statistical Methodology Group, Sanofi R&D
Han-Joo Kim, PhD, Associate Director, Biostatistics, Oncology PCU, Eisai Inc.
5.30 Chairman's Closing Remarks and Close of Day One
Want to know how you can get
involved? Interested in
promoting your services to this
market?
Contact Margaret Mugema,
SMi Marketing on +44 (0) 207
827 6072 or email:
mmugema@smi-online.co.uk
SPONSORSHIP AND EXHIBITION OPPORTUNITIES
SMi offer sponsorship, exhibition, advertising and
branding packages, uniquely tailored to
complement your company’s marketing strategy.
Prime networking opportunities exist to entertain,
enhance and expand your client base within the
context of an independent discussion specific to
your industry.
Should you wish to join the increasing number of
companies benefiting from sponsoring our
conferences please call:
Alia Malick on +44 (0) 20 7827 6168 or
email: amalick@smi-online.co.uk
Register online at: www.adaptivedesigns.co.uk • Alternatively fax

3. 7th Annual Adaptive Designs in Clinical Trials
9.00 Chairman's Opening Remarks
Robert Clay, Consultant, Highbury Regulatory Science,
Chief Regulatory Officer, Kinapse, Board Director, TOPRA
Breakthrough Trials and Streaming Through International
Regulation and Latest European Laws
9.10 Regulatory submissions across the international landscape
•Is there a global definition of “adaptive designs?”
•Forecasting a global harmonization of regulatory
requirements in adaptive designs
•Major Parallels and discrepancies between the FDA, EMA
and Japan’s PDMA that impact key areas of drug
development such as decision making, bias control, pre-clinical
studies and error control.
•The processes of universal acceptance of emerging and
unconventional adaptive designs.
•The impact of regulatory time lags across international
territories on drug development.
Robert Clay, Consultant, Highbury Regulatory Science,
Chief Regulatory Officer, Kinapse, Board Director, TOPRA
9.50 Moving from adaptive license to MAPPs (Medicine Adaptive
Pathways for Patients (MAPPs)
•Why we need to think MAPPs (Medicine Adaptive Pathways
for Patients (MAPPs) not AL
•What can public-private partnership offer to support
AL/MAPPs
•Stakeholder engagement – early involvement is key!
Dr. Elin Haf Davis, Director, Enabling Research
11.00 Adaptive designs in neuroscience drug development
•Adaptive clinical developments in Parkinson’s disease
•Clinical and latest technical collaborations across the PD
community
•Overcoming challenges
•Applications of adaptive designs in relevant clinical areas
of neuroscience
Dr. Richard Wyse, Director of Research & Development,
The Cure Parkinson’s Trust
11.40 Pharmacodynamics biomarkers in early phase oncology trials
•Use of surrogate markers during dose escalation to
demonstrate proof of mechanism
•Biomarker driven early phase oncology trials to demonstrate
proof of biological principle
•Case examples and challenges
Dr. Sidath Katugampola, Biomarker Development Scientist,
Cancer Research UK
Supported by
Day Two | Tuesday 21st April 2015
8.30 Registration & Coffee
KEYNOTE ADDRESS
10.30 Morning Coffee
12.20 Networking Lunch
Discovering New Opportunities in Adaptive Designs
1.30 Novel Response-Adaptive Designs to enhance
efficiency of clinical trials with time-to-event outcomes
•Response-adaptive randomization designs for multi-armed
survival trials
•Covariate-adjusted response-adaptive randomization
designs for survival trials with predictive biomarkers
•Optimal dose finding designs for time-to-event clinical trials
•Statistical software for implementing novel designs
Alex Sverdlov, Associate Director of Biostatistics, EMD Serono
2.10 Group-sequential response-adaptive designs
•Constructing a group sequential design
•Maintaining the overall type I error rate
•Equal and unequal information levels
•The elements of response-adaptive randomisation
•Incorporating covariate information
Dr. Steve Coad, Reader in Statistics, Queen Mary University
2.50 Afternoon Tea
3.20 Bandit models for the design of adaptive clinical trials in rare
disease
•Multi-armed bandit models for the optimisation of clinical
trials in rare diseases
•Main challenges in multi-armed bandit models
•Bayesian Bandit designs to optimise treatment selection in
multiple-treatment studies
Dr. Sofia S. Villar, Investigator Statistician, MRC Biostatistics Unit
for Trials Methodology Research
4.00 Seamless adaptive designs: Methods for estimating treatment
effects
•Statistical challenges for trials that use adaptive seamless
design
•Methods for point estimation after treatment selection
•Point estimation when the adaptation is selecting the most
promising population
•Estimation when treatment selection may depend on more
than one endpoint.
Dr. Peter Kimani, Assistant Professor in Medical Statistics,
Warwick Medical School
4.40 Chairman’s Closing Remarks and Close of Day Two
your registration to +44 (0)870 9090 712 or call +44 (0)870 9090 711
Spotlight
innovation

4. HALF-DAY POST-CONFERENCE AM WORKSHOP
Wednesday 22nd April 2015
8.30am – 12.30pm
Marriott Regents Park Hotel, London, UK
Bayesian Adaptive Clinical Designs
and Strategies
Workshop Host:
Dr. Sophie Carr, MD and Principal Analyst,
Bays Consulting Ltd.
Overview of the workshop:
This workshop will provide attendees with a gentle,
broad introduction to the wonders of Bayesian
analysis and its application to the development of
Adaptive Clinical Trials and strategies. The
interactive workshop will engage attendees
through structured exercises, discussion and team
working. Attendees will leave with a fundamental
understanding of how the techniques covered will
benefit their own processes.
Key Benefits of Attending:
Not heard of Bayesian analysis? Not sure how
Bayesian analysis is used strategically within
Adaptive Clinical Trials? Come along to this
workshop and gain a broad understanding of how
the powerful and elegant Bayesian analysis can
support your work and enhance your
understanding of uncertainty and statistics.
Programme
8.30 Registration and Coffee
9.00 Introduction of main principles and identify
key areas of research that benefit from
Bayesian modelling
9.30 Learn core skills of how to adapt Bayesian
statistics in research
10.30 Coffee Break
11.00 Structured exercises and team work
11.30 Discussion and evaluation
12.30 Close of Workshop
About the workshop host:
Sophie Carr has over fifteen years’ experience
working with clients to deliver key insights in areas
as diverse as policy formulation through to the
development of detailed technical models.
Sophie’s knowledge in statistics and facilitating
structured workshops began during her doctoral
research which focused on the elicitation of
opinion and values, the results of which were used
as the basis for Bayesian Belief Networks.
Across all sectors of industry, Sophie as undertaken
statistical analysis including: forecasting, time
series analysis and Bayesian inference; and
supported decision-making relating to capability
investments and strategic planning.
About the organisation:
Bays Consulting’s specialty is the application of
Bayesian analysis but can also draw upon a broad
range of mathematical and statistical techniques,
to build datasets, develop models and create
analysis plans to give the trends, insights, values…
the nugget of information needed to impact the
bottom line and meet objectives. Our regular
customers particularly value our commitment to
deliver clearly explained results to audiences at
every level of the business.

5. HALF-DAY POST-CONFERENCE PM WORKSHOP
Wednesday 22nd April 2015
1.30pm - 5.30pm
Marriott Regents Park Hotel, London, UK
Winning with Adaptive Trial Strategies and
Clinical Compliance Across the Evolving
International Regulatory Landscape.
Workshop Host:
Robert Clay, Consultant, Highbury Regulatory Science,
Chief Regulatory Officer, Kinapse, Board Director,
TOPRA
Overview of the workshop:
The modern adaptive trial design is now well established
in the design of drug development programmes and
accepted by regulatory agencies. However, the
application of these designs brings interesting
challenges not only in the design and operation of such
trials but also the integration of the results from studies
into a global regulatory and submission strategy. This
workshop will examine some of these challenges and
review examples from practice to encourage the
optimal use in development programmes.
Key Benefits of Attending:
This workshop is intended for researchers looking utilise
adaptive designs and gain optimal use out of these
methods. It is anticipated that learning professionals from
various backgrounds of medical research and industry
will be able to understand how adaptive designs plays a
definitive role within the framework of clinical research
and healthcare. It will also promote greater dialogue
among industry, regulators and academia.
Programme
1.30 Registration and Coffee
2.00 Opening
3.00 Addressing the challenges, perspectives
and solutions
3.30 Coffee Break
4.00 Case studies
5.00 Discussion & Q+A
5.30 Close of Workshop
About the workshop host:
Bob Clay established his own regulatory consultancy
practice through Highbury Regulatory Science, prior to
this he was a VP Global Regulatory Affairs at
AstraZeneca with responsibility for oncology, infection
and personalised healthcare. Bob is Chief Regulatory
Officer at Kinapse and a member of the board of
TOPRA (The Organisation of Professional Regulatory
Affairs). He is a member of the Expert Scientific Advisory
Committee for Medicines for Malaria Venture (MMV)
and several working groups at CPTR (Critical Path to TB
Regimens). Bob is a pharmacist with more than 30
years’ experience in drug development, leading the
global regulatory approval of many products across a
range of therapy areas including metabolic diseases,
neuroscience, cancer and infection. Bob has held
significant leadership roles in regulatory affairs in regional
and global functions at several companies including
AstraZeneca and Pfizer.
About the organisation:
Highbury Regulatory Science
provides consulting support to
small and mid-size growing
biotech companies on
regulatory and development strategies across a
range of therapy areas and regions. We also provide
advice on organisational development and
outsourcing key regulatory activities.
Kinapse provides expert
advisory, capability building and
operational services to the life
sciences industries

6. ADAPTIVE DESIGNS IN CLINICAL TRIALS
Conference: Monday 20th & Tuesday 21st April 2015, Marriott Regents Park Hotel, London, UK Workshops: Wednesday 22nd April 2015, London
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