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Adaptive Designs in Clinical Trials
20 April - 21 April 2015
Adaptive Designs in Clinical Trials

Prior to the approval of new drugs, substantial evidence of efficacy is required and safety brought through clinical trials. A massive decline in success rates in Phase II/III of clinical trials has resulted in only 1 in 10 drugs successfully passing  through these trials in the US.

With increasing pressure to bring drugs quickly into the market and reduce the cost of drug development, the paradigms of drug dosage studies in phase II trials and the overall adaptive framework of clinical trials are being increasingly challenged. Thus, strategies such as “Seamless Adaptive Designs” and “Group –Sequential Adaptive Designs” are particular areas that have received a great deal of attention in helping to improve the efficiency of drug development.

This conference will review the drug development process and provide a detailed discussion of how adaptive designs are changing the development process. The main focus of the presentation will be to explore the role of adaptive sample sizing; in particular, why it is important to consider the need to reassess sample sizes during the course of a trial. It is designed for those looking to network and gain the latest knowledge from the academics, industry leaders and regulators.

 

FEATURED SPEAKERS

Alex Sverdlov

Alex Sverdlov

Associate Director of Biostatistics , EMD Serono
Graham Clarke

Graham Clarke

Senior Director, Head of Respiratory & Inflammation Early Clinical Development, Quintiles
Robert Clay

Robert Clay

Consultant, Highbury Regulatory Science; Chief Regulatory Officer, Kinapse; Board Director, TOPRA, Highbury Regulatory Science
Simon Wandel

Simon Wandel

Expert Statistical Methodologist, Novartis Pharmaceuticals
Sophie Carr

Sophie Carr

MD and Principal Analyst, Bays Consulting Ltd
Tamas L. Paal

Tamas L. Paal

Professor Emeritus and Regulatory Adviser, University of Szeged, Hungary and National Institute of Pharmacy

Alex Sverdlov

Associate Director of Biostatistics , EMD Serono
Alex Sverdlov

Elin Haf Davies

Director, Enabling Research
Elin  Haf Davies

Elin Haf Davies PhD – biography

Elin Haf Davies began her career as a Paediatric Nurse at Great Ormond Street Hospital before going on to gain a PhD from University College London.
After eight years of clinical experience Elin moved to research and spent six years managing clinical and academic drug trials, in metabolic, neurology, endocrinology and pain. Her PhD focused on the development of age-appropriate and disease specific biomarkers and clinical endpoints in neurometabolic.
In 2007 Elin moved to work at the European Medicine Agency Paediatric Team where, amongst other projects she evaluated over a 100 Paediatric Investigation Plans, working closely with Scientific Advice and the Orphan team. After nearly six years there Elin now works independently Empowering Children | Enabling Research.
In her free time Elin has a passion for extreme adventure to raise money for charities close to her heart. In 2007-2008 she rowed across the Atlantic Ocean, raising £190,000 for metabolic research at Great Ormond Street Hospital. Money that funded a PhD project that identified the value of Vitamin B6 for managing neonatal seizures.
www.elinhafdavies.co.uk
 

Emilie Gerard

Biostatistician provider, Hays Life Sciences
Emilie Gerard

Filippo Baldacci

Medical Doctor, Researcher, University of Pisa
Filippo Baldacci

Dr. Fillipo Baldacci, MD is a Neurologist Physician at “Department of Clinical and Experimental Medicine, Neurology Unit, University of Pisa, Italy”.

He is the author and coauthor of 38 scientific publications (34 indexed in Pubmed), with experience in GCP acquired from 7 years of dedicated training. He  is also an experienced
sub Investigator in 8 clinical trials, investigating movement disorders.
 

 

 

Graham Clarke

Senior Director, Head of Respiratory & Inflammation Early Clinical Development, Quintiles
Graham Clarke

Dr. Graham Clarke PhD
 

Senior Director, Head of Respiratory & Inflammation Early Clinical Development, Quintiles Ltd
 

Dr. Clarke joined Quintiles Drug Research Unit, London in August 2008 as Therapeutic Area Head of Respiratory. He responsible for developing patient strategy, biomarker development initiatives across the Allergy and Respiratory TA, reaching out across the Quintiles business to develop an integrated approach to drug development; including patient recruitment strategy, protocol design and optimizing biomarker read-outs. After gaining initial training as a clinical physiologist at the Royal Brompton Hospital, Dr. Clarke has worked across the industry spectrum in a variety of respiratory clinical research environments. He completed a PhD in respiratory pharmacology and physiology with focus on airway and vascular hyper-responsiveness in asthma at King's College, London, with particular focus on inhaled models of inflammation including LPS and Allergen in 2006. His current focus is challenge models of inflammation, airway biomarker identification and inhaler generics/inhaler characterisation studies in broncho-protection and broncho-dilation bioequivalence programmes He has acted as a consultant in respiratory clinical trial set-up, teaching, and presenting at an international level. He is the former editor of the respiratory physiology journal, Inspire, and holds an honorary contract at Imperial College London as a senior research fellow in the department of cardiothoracic pharmacology, National Heart & Lung Institute, where he contributes to the research and medical degree teaching program.
 

Kai-Keen Shiu

FOCUS4 Trial Physician, MRC Clinical Trials Unit, UCL
Kai-Keen Shiu

Loic Darchy

Head of Statistical Methodology Group , Sanofi Aventis
Loic Darchy

Loïc has been working as Head of Statistical Methodology Group at Sanofi R&D for more than 5 years. He was previously in charge of the Internal Medicine Statistical Group for about 10 years at Sanofi too. Before joining Sanofi he worked for various pharmaceutical companies in multiple therapeutic areas such as immunology, inflammation diseases, oncology, cardiology and antibiotics. His favorite research areas are adaptive designs including biomarker-based designs, design and analysis of dose-finding studies, multiplicity issues, Bayesian statistics and data learning techniques.

Mohammad Al-Ubaydli

CEO, Patients Know Best
Mohammad Al-Ubaydli

Mohammad is founder and CEO of Patients Know Best and has over 15 years of experience in medical software. He trained as a physician at the University of Cambridge; worked as a staff scientist at the National Institutes of Health; and was a management consultant to US hospitals at The Advisory Board Company. He is the author of seven books, including Personal health records: A guide for clinicians and Streamlining Hospital-Patient Communication: Developing High Impact Patient Portals. He is also an honourary senior research associate at UCL medical school for his research on patient-controlled medical records. In 2012 he was elected an Ashoka Fellow as a social entrepreneur for the contributions he has made to patient care.

Peter Kimani

Assistant Professor in Medical Statistics, Warwick Medical School
Peter Kimani

Richard Wyse

Director of Research and Development, The Cure Parkinsons Trust
Richard Wyse

Robert Clay

Consultant, Highbury Regulatory Science; Chief Regulatory Officer, Kinapse; Board Director, TOPRA, Highbury Regulatory Science
Robert Clay

Sidath Katugampola

Biomarker Development Scientist, Cancer Research UK
Sidath Katugampola

Simon Wandel

Expert Statistical Methodologist, Novartis Pharmaceuticals
Simon Wandel

Simon Wandel holds a master in Statistics and a PhD in Medical Statistics/Epidemiology (both from University of Bern). In 2010, he joined the Early Clinical Biostatistics Group at Novartis Oncology as statistician, where he held roles with increasing responsibility (lead statistician, group manager). In 2014, he joined Novartis Oncology’s Statistical Methodology Group as Expert Statistical Methodologist. Simon Wandel has been involved in the design / analysis of numerous fully Bayesian phase I/II trials, for several of which he had close interactions with regulatory agencies. His main interest is in the use of historical and concurrent data in actual clinical trials.

Sofia Villar

Investigator Statistician , MRC Biostatistics Unit Hub for Trials Methodology Research
Sofia Villar

Sophie Carr

MD and Principal Analyst, Bays Consulting Ltd
Sophie Carr

Steve Coad

Reader in Statistics, Queen Mary, University of London
Steve  Coad

Steve Coad is a Reader in Statistics at Queen Mary, University of London, where he has worked since 2005, having previously held appointments at the Universities of Sussex, Michigan and Newcastle upon Tyne. He obtained his DPhil from the University of Oxford. He is currently an Associate Editor for Sequential Analysis and Statistical Methodology, and has been a Chartered Statistician since 1993 and a Chartered Mathematician since 1998. His research interests are mainly in the area of sequential design and analysis, with particular emphasis on estimation problems.

Tamas L. Paal

Professor Emeritus and Regulatory Adviser, University of Szeged, Hungary and National Institute of Pharmacy
Tamas L. Paal


Tamás L. Paál, having spent 11 years in the Hungarian pharmaceutical industry was Director-General of the National Institute of Pharmacy, the Hungarian drug regulatory agency from 1984 until his retirement in 2008. He is now contracted external advisor to the Agency.
He has been promoted University professor in 1984, worked for the Postgraduate Medical University then the Semmelweis University, Budapest. He was also invited to estab-lish the Institute of Drug Regulatory Affairs in the Faculty of Pharmacy, University of Szeged (Szeged, Hungary).
He is member of The Organisation of Professionals in Regulatory Affairs, the Europe-an Forum of Good Clinical Practice and the European Academy of Sciences and Arts.
 

Conference agenda

clock

8:30

Registration & Coffee

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9:00

Chairman's Opening Remarks

Graham Clarke, Senior Director, Head of Respiratory & Inflammation Early Clinical Development, Quintiles

clock

9:10

OPENING ADDRESS: Enhancing study design and biomarker read-outs in early clinical development

Graham Clarke, Senior Director, Head of Respiratory & Inflammation Early Clinical Development, Quintiles

 

  •  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
  • clock

    9:50

    Succeeding in Bayesian Adaptive Design

    Sophie Carr

    Sophie Carr, MD and Principal Analyst, Bays Consulting Ltd

  • Understand the key components of Bayesian statistics to enhance decision- making
  • Predictive classifiers in the Bayesian belief networks
  • Bayesian survival models and predicting time events in clinical trials
  • Operational-risk management: A Bayesian approach to modelling risk and uncertainties
  • clock

    10:30

    Morning Coffee

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    11:00

    Group Sequential Enrichment Design incorporating subgroup selection – Application to the optimization of the cut-off for a continuous predictive biomarker with time-to-event endpoints

    Emilie Gerard

    Emilie Gerard, Biostatistician provider, Hays Life Sciences

     

  • 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
  • clock

    11:40

    Real world patient data in adaptive clinical designs

    Mohammad Al-Ubaydli, CEO, Patients Know Best

     

  •   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.
  • clock

    12:20

    Networking Lunch

    clock

    13:30

    Afternoon Chairman's Opening Remarks

    Tamas L. Paal, Professor Emeritus and Regulatory Adviser, University of Szeged, Hungary and National Institute of Pharmacy

    clock

    13:40

    KEYNOTE ADDRESS: 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
  • Tamas L. Paal, Professor Emeritus and Regulatory Adviser, University of Szeged, Hungary and National Institute of Pharmacy

    clock

    14:20

    Clinical trials in neurodegenerative disease

    Filippo Baldacci, Medical Doctor, Researcher, University of Pisa

     

  • 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
  • clock

    15:00

    Afternoon Tea

    clock

    15:30

    Bayesian approaches in practice: Examples from early phase Oncology trials

    Simon Wandel, Expert Statistical Methodologist, Novartis Pharmaceuticals

     

    • Prior derivation the (robust) meta-analytic-predictive approach
    • Using historical control data in phase II
    • Tailored multi-strata designs
    • Lessons learned from more than 100 Bayesian adaptive trials
    clock

    16:10

    FOCUS4: Designing and delivering an Adaptive-Biomarker driven Multi Arm Mullti Stage (MAMS) cancer trial

    Kai-Keen Shiu

    Kai-Keen Shiu, FOCUS4 Trial Physician, MRC Clinical Trials Unit, UCL

     

  • 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
  • clock

    16:50

    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.
  • Tamas L. Paal, Professor Emeritus and Regulatory Adviser, University of Szeged, Hungary and National Institute of Pharmacy

    Graham Clarke, Senior Director, Head of Respiratory & Inflammation Early Clinical Development, Quintiles

    Loic Darchy, Head of Statistical Methodology Group , Sanofi Aventis

    Mohammad Al-Ubaydli, CEO, Patients Know Best

    Kai-Keen Shiu

    Kai-Keen Shiu, FOCUS4 Trial Physician, MRC Clinical Trials Unit, UCL

    clock

    17:30

    Chairman's Closing Remarks and Close of Day One

    Tamas L. Paal, Professor Emeritus and Regulatory Adviser, University of Szeged, Hungary and National Institute of Pharmacy

    clock

    8:30

    Registration & Coffee

    clock

    9:00

    Chairman's Opening Remarks

    Robert Clay

    Robert Clay, Consultant, Highbury Regulatory Science; Chief Regulatory Officer, Kinapse; Board Director, TOPRA, Highbury Regulatory Science

    clock

    9:10

    OPENING ADDRESS: Regulatory submissions across the international landscape

    Robert Clay

    Robert Clay, Consultant, Highbury Regulatory Science; Chief Regulatory Officer, Kinapse; Board Director, TOPRA, Highbury Regulatory Science

     

  •  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.
  • clock

    9:50

    Moving from adaptive license to MAPPs (Medicine Adaptive Pathways for Patients (MAPPs)

    Elin Haf Davies, Director, Enabling Research

    clock

    10:30

    Morning Coffee

    clock

    11:00

    Adaptive designs in neuroscience drug development

    Richard Wyse

    Richard Wyse, Director of Research and Development, The Cure Parkinsons Trust

     

  • 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
  • clock

    11:40

    Pharmacodynamics biomarkers in early phase oncology trials

    Sidath Katugampola

    Sidath Katugampola, Biomarker Development Scientist, Cancer Research UK

    clock

    12:20

    Networking Lunch

    clock

    13:30

    Novel Response-Adaptive Designs to enhance efficiency of clinical trials with time-to-event outcomes

    Alex Sverdlov

    Alex Sverdlov, Associate Director of Biostatistics , EMD Serono

     

  • 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
  • clock

    14:10

    Group-sequential response-adaptive designs

    Steve Coad, Reader in Statistics, Queen Mary, University of London

     

  • 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

     

  • clock

    14:50

    Afternoon Tea

    clock

    15:20

    Bandit models for the design of adaptive clinical trials in rare disease

    Sofia Villar

    Sofia Villar, Investigator Statistician , MRC Biostatistics Unit Hub for Trials Methodology Research

     

  • 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
  • clock

    16:00

    Seamless adaptive designs: Methods for estimating treatment effect

    Peter Kimani

    Peter Kimani, Assistant Professor in Medical Statistics, Warwick Medical School

     

  • Design Strategy and motivation
  • Interim analysis of continuous long-term endpoints in clinical trials with longitudinal outcomes
  • Controlling the overall type I error rate
  • Case studies
  • Validating the end points and sample size estimation
  • clock

    16:40

    Chairman’s Closing Remarks and Close of Day Two

    Robert Clay

    Robert Clay, Consultant, Highbury Regulatory Science; Chief Regulatory Officer, Kinapse; Board Director, TOPRA, Highbury Regulatory Science


    Senior Director, Head of Respiratory & Inflammation Early Clinical Development
    Quintiles
    Director
    Enabling Research
    Director of Research and Development
    The Cure Parkinsons Trust
    Biomarker Development Scientist
    Cancer Research UK
    Associate Director of Biostatistics
    EMD Serono
    Reader in Statistics
    Queen Mary, University of London
    Investigator Statistician
    MRC Biostatistics Unit Hub for Trials Methodology Research
    Assistant Professor in Medical Statistics
    Warwick Medical School
    MD and Principal Analyst
    Bays Consulting Ltd
    Head of Statistical Methodology Group
    Sanofi Aventis
    CEO
    Patients Know Best
    Professor Emeritus and Regulatory Adviser
    University of Szeged, Hungary and National Institute of Pharmacy
    Medical Doctor, Researcher
    University of Pisa
    FOCUS4 Trial Physician
    MRC Clinical Trials Unit, UCL
    Consultant, Highbury Regulatory Science; Chief Regulatory Officer, Kinapse; Board Director, TOPRA
    Highbury Regulatory Science
    Biostatistician provider
    Hays Life Sciences
    Expert Statistical Methodologist
    Novartis Pharmaceuticals

    Official Media Partner

    Supporters

    Interview with Graham Clarke, Senior Director, Head of Respiratory & Inflammation Early Clinical Development, Quintiles

    Download

    Adaptive Designs Speaker Interview for Loic Darchy

    Download

    Interview with Alex Sverdlov, Associate Director of Biostatistics, EMD Serono

    Download

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    Marriott Regents Park

    128 King Henry's Road
    London NW3 3ST
    United Kingdom

    Marriott Regents Park

    This 4 star north London hotel in zone 2 is the perfect destination for the astute business traveler as well as the leisure guest that knows how convenient north London hotels are, as a base from which to explore the city .Bond Street is just 3 stops from Swiss Cottage underground station on the Jubilee Line, so you can be shopping, exploring the sights and taking in one of London’s world-renowned West End shows in less than 15 minutes when you stay at this hotel near central London. At the same time, the hive of activity that is Camden Town, the chic shops, cafes and restaurants of Primrose Hill and ZSL’s London Zoo in Regents Park are all just a short walk from this hotel in north London.

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    WHAT IS CPD?

    CPD stands for Continuing Professional Development’. It is essentially a philosophy, which maintains that in order to be effective, learning should be organised and structured. The most common definition is:

    ‘A commitment to structured skills and knowledge enhancement for Personal or Professional competence’

    CPD is a common requirement of individual membership with professional bodies and Institutes. Increasingly, employers also expect their staff to undertake regular CPD activities.

    Undertaken over a period of time, CPD ensures that educational qualifications do not become obsolete, and allows for best practice and professional standards to be upheld.

    CPD can be undertaken through a variety of learning activities including instructor led training courses, seminars and conferences, e:learning modules or structured reading.

    CPD AND PROFESSIONAL INSTITUTES

    There are approximately 470 institutes in the UK across all industry sectors, with a collective membership of circa 4 million professionals, and they all expect their members to undertake CPD.

    For some institutes undertaking CPD is mandatory e.g. accountancy and law, and linked to a licence to practice, for others it’s obligatory. By ensuring that their members undertake CPD, the professional bodies seek to ensure that professional standards, legislative awareness and ethical practices are maintained.

    CPD Schemes often run over the period of a year and the institutes generally provide online tools for their members to record and reflect on their CPD activities.

    TYPICAL CPD SCHEMES AND RECORDING OF CPD (CPD points and hours)

    Professional bodies and Institutes CPD schemes are either structured as ‘Input’ or ‘Output’ based.

    ‘Input’ based schemes list a precise number of CPD hours that individuals must achieve within a given time period. These schemes can also use different ‘currencies’ such as points, merits, units or credits, where an individual must accumulate the number required. These currencies are usually based on time i.e. 1 CPD point = 1 hour of learning.

    ‘Output’ based schemes are learner centred. They require individuals to set learning goals that align to professional competencies, or personal development objectives. These schemes also list different ways to achieve the learning goals e.g. training courses, seminars or e:learning, which enables an individual to complete their CPD through their preferred mode of learning.

    The majority of Input and Output based schemes actively encourage individuals to seek appropriate CPD activities independently.

    As a formal provider of CPD certified activities, SAE Media Group can provide an indication of the learning benefit gained and the typical completion. However, it is ultimately the responsibility of the delegate to evaluate their learning, and record it correctly in line with their professional body’s or employers requirements.

    GLOBAL CPD

    Increasingly, international and emerging markets are ‘professionalising’ their workforces and looking to the UK to benchmark educational standards. The undertaking of CPD is now increasingly expected of any individual employed within today’s global marketplace.

    CPD Certificates

    We can provide a certificate for all our accredited events. To request a CPD certificate for a conference , workshop, master classes you have attended please email events@saemediagroup.com

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    Contact SAE Media Group

    UK Office
    Opening Hours: 9.00 - 17.30 (local time)
    SAE Media Group , Ground Floor, India House, 45 Curlew Street, London, SE1 2ND, United Kingdom
    Tel: +44 (0) 20 7827 6000 Fax: +44 (0) 20 7827 6001
    Website: http://www.smgconferences.com Email: events@saemediagroup.com
    Registered in England - SMi Group Ltd trading as SAE Media Group




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