smi group's 9th annual paediatric clinical trials conference

@SMIPHARM

www.paediatric-trials.co.uk BOOK BY 19TH DECEMBER AND RECEIVE £300 DISCOUNTBOOK BY 30TH JANUARY AND RECEIVE A £100 DISCOUNT

Register online or fax your registration to +44 (0) 870 9090 712 or call +44 (0) 870 9090 711

SMi presents the 9th annual...

25 - 26

MAR2015Holiday Inn Regents Park Hotel, London, UK

Paediatric Clinical TrialsReviewing latest developments in theformulation of medicines for children

PLUS INTERACTIVE PRE-CONFERENCE WORKSHOPTuesday 24th March 2015, Holiday Inn Regents Park Hotel, London, UK

Paediatric Drug Development – Vision, Challenges, Strategy Workshop Leader: Klaus Rose, CEO, klausrose Consulting

10.30am - 4.45pm

CHAIR FOR 2015: Klaus Rose, CEO, klausrose Consulting

KEY SPEAKERS INCLUDE: • Susan Cole, Senior Pharmacokinetics Assessor, MHRA• Dr Koenraad Norga, Head of Clinic Paediatric Oncology,

Universitair Ziekenhuis Antwerpen, Vice Chairman, PaediatricCommitee (PDCO)

• Dr Mette Due Theilade, Principal Scientist, PaediatricInvestigation Plan (PIP), Regulatory Affairs, Novo Nordisk A/S

• Dr Cristina De Min, Chief Medical Officer, Novimmune• Terry Ernest, Formulation Team Manager, Global Formulation,

Product Development, GlaxoSmithKline R&D• Dr Philippe Auby, Vice President of Global Clinical Research,

Paediatric Neuro-Psychiatry, Lundbeck

REGISTER BY 19TH DECEMBER AND RECEIVE A £300 DISCOUNTREGISTER BY 30TH JANUARY AND RECEIVE A £100 DISCOUNT

BENEFITS OF ATTENDING:• New for 2015 – Join us and hear the MHRA discuss guidelines

for pharmacokinetic modelling in paediatric regulatorysubmissions

• New for 2015 – Listen to GlaxoSmithKline discussingconsiderations associated with the use of foods andbeverages to assist the administration of medicines to children

• The PDCO will review Paediatric clinical trials in the EU; whatyou should know

• Case studies showcasing breakthroughs in paediatric clinicaltrials

• Hear key pharmaceutical, biotechnology, regulatory andacademic experts share their knowledge to ensure yourclinical trials have optimal outcomes

• An overview of the current practices and challenges indesigning and preparing for paediatric clinical trials and howto get yours right!

Register online at: www.paediatric-trials.co.uk • Alternatively fax y

Paediatric Clinical Trials Day One | Wednesday 25th March 2015

8.30 Registration & Coffee

9.00 Chairman's Opening RemarksKlaus Rose, CEO, klausrose Consulting

REGULATORY FRAMEWORK FOR PAEDIATRIC RESEARCH – PART 1

OPENING ADDRESS9.10 From the origin of US and EU paediatric legislation to today’s

PIP challenges • The vision of US and EU pediatric legislation• Has medical treatment of children improved since 1997?• Operational challenges in the pediatric discussion with the

regulatory authorities• What can you do if you think pediatric requirements are

exaggerated - tips and pointers• Processes and procedures to limit the number of PIP

modifications requiredKlaus Rose, CEO, klausrose Consulting

9.50 Paediatric clinical trials in the EU: regulatory perspective• Using the EU Paediatric Regulation to develop better

medicines for childrenDr Koenraad Norga, Head of Clinic Paediatric Oncology,Universitair Ziekenhuis Antwerpen, Vice Chairman, PaediatricCommitee (PDCO)

10.30 Morning Coffee

11.00 Revision of the ICH E11 guideline: status and update• Reviewing & analysing how an addendum to the current

E11 guideline could address new scientific and technicalknowledge advances in pediatric drug development

• Discussing how the ICH E11 currently covers many of therelevant issues for consideration in pediatric drugdevelopment and reviewing where there are gaps incontemporary guidance on several topics

• Discussing how to best address key specific topics such asthe type of studies and methodology including the use ofextrapolation of data in pediatric drug development, ormodelling and simulation approaches as they might applyto pediatric subpopulations

Solange Rohou, Director Regulatory Affairs/Policy Europe,AstraZeneca

PREPARING FOR CLINICAL TRIALS

11.40 Infectious diseases – outbreaks and crises – how to includechildren in clinical trials• Emerging viral infections – risk and transmission in children• Therapeutics and vaccines – treatment and prevention• Bacterial meningitis – and end in sightDr David G. McIntosh, Head, Global Scientific Affairs, NovartisVaccines and Diagnostics

12.20 Networking Lunch

1.20 Optimising the role of the applicant to facilitate a smootherpaediatric trial ethics review• Understanding what the EC/IRB look for in your

application and protocol• Reviewing the aspects of legal and ethical frameworks

relevant to paediatric trials• Addressing key ethical issues e.g. information provision,

minimising distress etc.• Realising the benefits of GEP© (Good Ethical Practice) for

successful paediatric trialsJane Lamprill, Paediatric Research Advisor, SpecialistMedical Writer, Children, Paediatric Research Consultancy

2.00 Formulating for paediatric clinical trials, pitfalls andopportunities• Understanding the patient, age specific problems• Excipients, do and do not (mostly do nots)• The oral route, organoleptics• Where to find solutionsPharmacist Daniel Bar-Shalom, Associate Professor, Universityof Copenhagen

2.40 Afternoon Tea

3.10 Dosage form acceptability assessment & paediatric patientreported outcome instruments• Paediatric clinical trials provide an opportunity for

formulators to evaluate the acceptability of the dosageform being developed

• The heterogeneous patient population in paediatricclinical trials makes acceptability assessment challenging,since the ability of the patients to provide feedback varies

• Paediatric Patient Reported Outcome Instruments may actas a useful tool to help obtain accurate data

Dr Roy Turner, Formulation Project Leader, Paediatric &Geriatric Network Leader, Technical Research &Development, Novartis

INNOVATIONS AND ADVANCES

3.50 Innovation in paediatrics• Why we need to develop age-appropriate and disease-

specific outcome measures • Defining clinically meaningful involvement – why it’s essential Dr Elin Haf Davies, Director & Founder, Empowering Children/Enabling Research

PANEL DISCUSSION4.30 Optimizing patient retention strategies for successful trial

development – how to keep patients on board• Making sure patients stay on course with all study

requirements to ensure the statistical validity of the clinicaldata analysis

• Listening to study volunteers and developing sustainablerelationships throughout the trial

• Knowing what patients want and need in order tomaximize retention

• Working with patients to engage them in the study andestablishing the most effective methods to reach them

Professor Koenraad Norga, Head of Clinic PaediatricOncology, Universitair Ziekenhuis Antwerpen, ViceChairman, Paediatric Commitee (PDCO)Dr Deborah Lee, Senior Medical Director, Clinical Affairs,Lundbeck, USADr David G. McIntosh, Head, Global Scientific Affairs, Novartis Vaccines and DiagnosticsDr Philippe Auby, Vice President of Global Clinical Research, Paediatric Neuro-Psychiatry, Lundbeck

5.10 Chairman's Closing Remarks and Close of Day One

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Paediatric Clinical Trials Day Two | Thursday 26th March 2015

8.30 Registration & Coffee

9.00 Chairman's Opening RemarksKlaus Rose, CEO, klausrose Consulting

REGULATORY FRAMEWORK FOR PAEDIATRIC RESEARCH – PART 2

9.10 Population pharmacokinetic and physiologically based pharmacokinetic modelling in paediatric regulatory submissions• Determination of posology in paediatric patients

usually involves an initial extrapolation from adult data.There is then an iterative process with results from one study informing the design of further studies

• Pharmacokinetic models are important to inform theextrapolation and to collate the data

• Physiologically based models offer an opportunity tomechanistically understand the drug disposition in childrenand to use this information to optimise clinical study design

• Population pharmacokinetic analysis affords theopportunity for sparse sampling in children and optimal useof the available data

• Pharmacodynamics may also differ in children and shouldbe adequately described in suitable PKPD models

• It is important that size and maturation functions areadequately described in the models

• For regulatory submissions qualification and verification ofthese models and the input parameters is very important

Susan Cole, Senior Pharmacokinetics Assessor, MHRA

9.50 Transatlantic collaboration on Paediatric Investigation Plans -recent experiences with Type 2 diabetes PIPs• EU Paediatric Investigation Plan vs US Paediatric Study Plan • What to do with divergent regulatory requirements to

paediatric trials• Challenges with Type 2 Diabetes Mellitus PIPsDr Mette Due Theilade, Principal Scientist, PaediatricInvestigation Plan (PIP), Regulatory Affairs, Novo Nordisk A/S,

DESIGNING PAEDIATRIC CLINICAL TRIALS

10.30 Morning Coffee

11.00 The challenges of developing the first drug for a life-threatening disease for which no approved drug exists • Creation of alternative ways to determine dose and dose

frequency• Limitations of conventional study designs in rare diseases• Challenges in the recruitment of paediatric patients with a

rare disease • Definition of a regulatory pathway for a disease for which

no drug has ever been approvedDr Cristina De Min, Chief Medical Officer, Novimmune

11.40 Strategies for maximizing pediatric studies• Challenges of conducting paediatric trials in orphan

indications and part of life cycle management• Use of PK modelling for establishing paediatric dosing and

to support efficacy in orphan populations• Use of patient advocates in protocol development in

paediatric orphan indicationsDr Deborah Lee, Senior Medical Director, Clinical Affairs,Lundbeck, USA

12.20 Networking Lunch

CASE STUDIES

1.20 Considerations associated with the use of foods and

beverages to assist administration of medicines to children

• Why are products mixed with food?

• Current practices of mixing with food

• Summary of recent EMA guidance

• Risks associated with mixing products with food

Terry Ernest, Formulation Team Manager, Global Formulation,

Product Development, GlaxoSmithKline R&D

2.00 NIHR clinical research network: children support and delivery

of clinical research

• Current and emerging Infrastructures

• Delivery frameworks

• NIHR children performance to date with key examples

• Challenges and opportunities

Dr. Sabah Attar, Divisional Head of Portfolio Operations, NIHR

Clinical Research Network: Children

2.40 Afternoon Tea

3.10 Practical application of physiologically based

pharmacokinetic modeling to select starting doses in

children

• Advantages and limitations of a physiologically based PK

modelling approach

• A strategy for applying PBPK in drug development projects

• Several case studies from Roche projects will be shared

Dr Neil Parrott, Modelling & Simulation Scientist,

Pharmaceutical Sciences, Roche Innovation Center Basel,

Roche Pharmaceutical Research and Early Development

3.50 Learning from paediatric psychopharmacology

• Practical guidance for paediatric trials using experience

from paediatric psychopharmacology

• Scientific considerations and ethical challenges

• Case studies

Dr Philippe Auby, Vice President of Global Clinical Research,

Paediatric Neuro-Psychiatry, Lundbeck, France

4.30 Chairman’s Closing Remarks and Close of Day Two

KEYNOTE

FULL-DAY PRE-CONFERENCE WORKSHOPTuesday 24th March 2015

10.30am - 4.45pmHoliday Inn Regents Park Hotel, London, UK

Paediatric Drug Development – Vision,Challenges, Strategy

Workshop Leader: Klaus Rose, CEO, klausrose Consulting

Overview of the workshop:This workshop will be an interactive walk throughdifferent aspects of pediatric drug development,starting with the historical background of thediscussion about on-label and off-labelprescription of drugs for children; ethical &operational challenges of clinical trials in children;the different meanings of the term 'pediatric drugdevelopment' as used by different players;pediatric pharmaceutical legislation in the USAand the EU, and an assessment to what degreepediatric legislation has improved child healthand child research over the past 18 years. Theworkshop will include an interactive walk throughthe EMA pediatric website.

Key benefits of attending:People working in life science industry, regulatoryaffairs, academia, ethics committees and peoplewith a general interest in the challenge of bettermedicines for children.

Programme:10.30 Registration and Coffee

11.00 Opening Remarks

11.15 Session 1 Historical Regulatory and ClinicalBackground

12.00 Session 2 Child Physiology and ClinicalPharmacology

1.00 Break

2.30 Session 3 Pediatric Clinical Trials

3.30 Session 4 The PIP Challenge: OperationalConsiderations

4.30 Closing remarks

4.45 End of workshop

About the workshop host:Dr. Klaus Rose is CEO of klausrose Consulting,Switzerland, advising on pediatric drugdevelopment and how to comply with FDA &EMA pediatric requirements. Mostly his clients

are pharmaceutical companies and academicinsitutions. He first studied Latin languages &psychology and then medicine. After postgraduateclinical training in General Medicine in Germany andEngland hejoined pharmaceutical industry in 1991. Hejoined various positions in R&D and medical affairs,was Global Head Pediatrics Novartis 2001 - 2005 andGlobal Head Pediatrics Roche 2005 - 2009. After ayear with a regulatory consultancy he established hisown business. Dr. Rose is a frequent speaker oninternational conferences on pediatric drugdevelopment and publishes on a regular basis. Atextbook on pediatric formulations, co-edited withProf. Bar-Shalom, University of Kopenhagen, DK, wasreleased February 2014. Married with two daughters,his private interests include Mediterranean cooking,wine, gardening, Latin languages, and classical guitar.

About the organisation: klaurose Consulting, Switzerland supportspharmaceutical companies, CROs and otherhealth care providers in all aspects of pediatricdrug development.

SMi PharmaceuticalForward Planner

2014-2015

SPONSORSHIP AND EXHIBITION OPPORTUNITIES

SMi offer sponsorship, exhibition, advertisingand branding packages, uniquely tailored tocomplement your company’s marketingstrategy. Prime networking opportunities existto entertain, enhance and expand your clientbase within the context of an independentdiscussion specific to your industry.

Should you wish to join the increasing numberof companies benefiting from sponsoring ourconferences please call:

Alia Malick on +44 (0) 20 7827 6168 or email: [email protected]

DECEMBER

Cold Chain Distribution2nd – 3rd December 2014

Marriott Regents Park, London

JANUARY

Pharmaceutical Microbiology21st – 22nd January 2015


Social Media in the Pharma Industry21st – 22nd January 2015


Pre Filled Syringes28th – 29th January 2015


FEBRUARY

Parallel Trade9th – 10th February 2015

Holiday Inn Regents Park, London

Advances and Progress in Drug Design16th – 17th February 2015


RNA Therapeutics16th – 17th February 2015


MARCH

Superbugs & Superdrugs– A focus on Antibacterials

25th – 26th March 2015Holiday Inn Regents Park, London

Paediatric Clinical Trials25th – 26th March


APRIL

Asthma & COPD 13 – 14 April 2015


Adaptive Designs20th – 21st April 2015, London

Pre Filled Syringes USA27th – 28th April 2015, Iselin, USA

Lyophilisation and Freeze Drying USA29th – 30th April 2015

Iselin, USA

PAEDIATRIC CLINICAL TRIALS Conference: Wednesday 25th & Thursday 26th March 2015, Holiday Inn Regents Park Hotel, London, UK Workshop: Tuesday 24th March 2015, London, UK

4 WAYS TO REGISTER

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If you have any further queries please call the Events Team on tel +44 (0) 870 9090 711 or you can email [email protected]

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smi group's 9th annual paediatric clinical trials conference

Education