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Paediatric Clinical Trials

A large number of medicines used in paediactric care have not been tested on children and are therefore used off lable or unlicensed. This absence of suitable authorised medicinal products to treat conditions in children is an issue that has been of concern for some time, and one that is increasingly coming to the fore.

Returning for its ninth year as an industry leading event, SAE Media Group are proud to present the 9th annual Paediatric Clinical Trials Conference, 25th - 26th March 2015, London. This event will look at the paediatric therapeutic developments, directives and procedures which have been put in place to protect children taking part in clinical trials.


 

A series of interactive conference sessions, roundtables and panel discussion facilitated by leading industry experts will provide executives with useful tools and best practices to effectively submit a PIP that meets FDA and EMA regulations and improve skills and understanding of the responsibilities of conducting clinical research in the paediatric population. In addition, consideration of new approaches for clinical trial design to keep costs down whilst navigating the complexities of paediatric research, makes this a must-attend meeting for all individuals based in the world of paediatric trials and care.

 

Key topics include:
• Guidelines on the Paediactric Investigation Plan (PIP) – strategies to overcome any challenges
• Formulating meaningful statistics with a limited patient population
• Site selection and feasibility- identifying appropriate clinical trial sites
• Optimizing patient retention strategies for successful trial development

‘Some great and interesting presentations’ Allergan

FEATURED SPEAKERS

David McIntosh

David McIntosh

Head, Global Scientific Affairs, Novartis Vaccines
Deborah Lee

Deborah Lee

Senior Medical Director, Clinical Affairs, Lundbeck, USA
Klaus Rose

Klaus Rose

CEO, klausrose Consulting
Neil Parrott

Neil Parrott

Modelling & Simulation Scientist, Pharmaceutical Sciences, Roche Innovation Center Basel, Roche Pharmaceutical Research and Early Development

Cristina De Min

Chief Medical Officer, NovImmune SA
Cristina  De Min

Daniel Bar-Shalom

Associate Professor, University of Copenhagen
Daniel Bar-Shalom

David McIntosh

Head, Global Scientific Affairs, Novartis Vaccines
David McIntosh

Deborah Lee

Senior Medical Director, Clinical Affairs, Lundbeck, USA
Deborah Lee

Diana A. van Riet - Nales

Senior pharmaceutical assessor, Chemical Pharmaceutical Assessments (CFB), Member EMA Quality Working Party, , Medicines Evaluation Board in the Netherlands (MEB)
Diana A.  van Riet - Nales

Dr Elin Haf Davies

Director & Founder, Empowering Children | Enabling Research
Dr Elin Haf Davies

Jane Lamprill

Paediatric Research Consultant Specialist Medical Writer for Children and Member FP7 Ethics Panel, Paediatric Research Consultancy
Jane Lamprill

Klaus Rose

CEO, klausrose Consulting
Klaus Rose

Mette Due Theilade Thomsen

Principal Scientist, Paediatric Investigation Plan (PIP), Regulatory Affairs , Novo Nordisk A/S, Denmark
Mette  Due Theilade Thomsen

Neil Parrott

Modelling & Simulation Scientist, Pharmaceutical Sciences, Roche Innovation Center Basel, Roche Pharmaceutical Research and Early Development
Neil Parrott

Philippe Auby

Vice President of Global Clinical Research, Paediatric Neuro-Psychiatry, Lundbeck, France
Philippe Auby

Professor Koenraad Norga

Head of Clinic Paediatric Oncology,, Universitair Ziekenhuis Antwerpen, Vice Chairman, Paediatric Commitee (PDCO)
Professor Koenraad  Norga

Roy Turner

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

Sabah Attar

Divisional Head of Portfolio Operations, NIHR Clinical Research Network: Children
Sabah Attar

Solange Rohou

Director Regulatory Affairs/Policy Europe, AstraZeneca
Solange Rohou

Susan Cole

Senior Pharmacokinetics Assessor, Statistics and Pharmacokinetics Unit, MHRA
Susan Cole

Terry Ernest

Formulation Team Manager, Global Formulation, Product Development , GlaxoSmithKline R&D
Terry Ernest

sponsors

Conference agenda

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10:30

Registration & Coffee

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11:00

Chairman's Opening Remarks

Klaus Rose

Klaus Rose, CEO, klausrose Consulting

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11:15

Session 1: Historical Regulatory and Clinical Background

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12:00

Session 2 Child Physiology and Clinical Pharmacology

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13:00

Networking Lunch

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14:30

Session 3 Pediatric Clinical Trials

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15:30

Session 4 The PIP Challenge: Operational Considerations

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16:30

Closing remarks

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16:45

End of workshop

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8:30

Registration & Coffee

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9:00

Chairman's Opening Remarks

Klaus Rose

Klaus Rose, CEO, klausrose Consulting

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9:10

From the origin of US and EU pediatric legislation to today’s PIP challenges

Klaus Rose

Klaus Rose, CEO, klausrose Consulting

• 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 required

 

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9:50

Paediatric clinical trials in the EU: regulatory perspective

Professor Koenraad  Norga

Professor Koenraad Norga, Head of Clinic Paediatric Oncology,, Universitair Ziekenhuis Antwerpen, Vice Chairman, Paediatric Commitee (PDCO)

• Using the EU Paediatric Regulation to develop better medicines for children

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10:30

Morning Coffee

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11:00

Revision of the ICH E11 guideline: status and update

Solange Rohou

Solange Rohou, Director Regulatory Affairs/Policy Europe, AstraZeneca

• Reviewing & analysing how an addendum to the current E11 guideline could address new scientific and technical knowledge advances in pediatric drug development
• Discussing how the ICH E11 currently covers many of the relevant issues for consideration in pediatric drug development and reviewing where there are gaps in contemporary guidance on several topics
• Discussing how to best address key specific topics such as the type of studies and methodology including the use of extrapolation of data in pediatric drug development, or modelling and simulation approaches as they might apply to pediatric subpopulations
 

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11:40

Infectious diseases – outbreaks and crises – how to include children in clinical trials

David McIntosh

David McIntosh , Head, Global Scientific Affairs, Novartis Vaccines

• Emerging viral infections – risk and transmission in children
• Therapeutics and vaccines – treatment and prevention
• Bacterial meningitis – and end in sight
 

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12:20

Networking Lunch

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13:20

Pharmaceutical development of medicine for children in the PIP and Marketing Authorisation Dossiers

Diana A.  van Riet - Nales

Diana A. van Riet - Nales, Senior pharmaceutical assessor, Chemical Pharmaceutical Assessments (CFB), Member EMA Quality Working Party, , Medicines Evaluation Board in the Netherlands (MEB)

• Basic principles of the guideline on the pharmaceutical development of medicines for paediatric use
• Selection of the dosage form
• Selection of the excipients in the preparation
• User aspects
• Regulatory science
 

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14:00

Optimising the role of the applicant to facilitate a smoother paediatric trial ethics review

Jane Lamprill

Jane Lamprill, Paediatric Research Consultant Specialist Medical Writer for Children and Member FP7 Ethics Panel, Paediatric Research Consultancy

• 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 trials

 

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14:40

Formulating for paediatric clinical trials, pitfalls and opportunities

Daniel Bar-Shalom

Daniel Bar-Shalom, Associate Professor, University of Copenhagen

• Understanding the patient, age specific problems
• Excipients, do and do not (mostly do nots)
• The oral route, organoleptics
• Where to find solutions
 

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15:20

Afternoon Tea

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15:40

Dosage form acceptability assessment & paediatric patient reported outcome instruments

Roy Turner

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

• Paediatric clinical trials provide an opportunity for formulators to evaluate the acceptability of the dosage form being developed
• The heterogeneous patient population in paediatric clinical trials makes acceptability assessment challenging, since the ability of the patients to provide feedback varies
• Paediatric Patient Reported Outcome Instruments may act as a useful tool to help obtain accurate data
 

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16:20

Innovation in paediatrics

Dr Elin Haf Davies

Dr Elin Haf Davies, Director & Founder, Empowering Children | Enabling Research

• Why we need to develop age-appropriate and disease-specific outcome measures
• Defining clinically meaningful involvement – why it’s essential
 

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17:00

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 clinical data analysis
• Listening to study volunteers and developing sustainable relationships throughout the trial
• Knowing what patients want and need in order to maximize retention
• Working with patients to engage them in the study and establishing the most effective methods to reach them
 

Deborah Lee

Deborah Lee, Senior Medical Director, Clinical Affairs, Lundbeck, USA

Philippe Auby

Philippe Auby, Vice President of Global Clinical Research, Paediatric Neuro-Psychiatry, Lundbeck, France

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17:40

Chairman’s Closing Remarks and Close of Day One

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8:30

Registration & Coffee

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9:00

Chairman's Opening Remarks

Klaus Rose

Klaus Rose, CEO, klausrose Consulting

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9:10

Population pharmacokinetic and physiologically based pharmacokinetic modelling in paediatric regulatory submissions

Susan Cole

Susan Cole, Senior Pharmacokinetics Assessor, Statistics and Pharmacokinetics Unit, MHRA

• 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 the extrapolation and to collate the data
• Physiologically based models offer an opportunity to mechanistically understand the drug disposition in children and to use this information to optimise clinical study design
• Population pharmacokinetic analysis affords the opportunity for sparse sampling in children and optimal use of the available data
• Pharmacodynamics may also differ in children and should be adequately described in suitable PKPD models
• It is important that size and maturation functions are adequately described in the models
• For Regulatory submissions qualification and verification of these models and the input parameters is very important
 

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9:50

Transatlantic collaboration on Paediatric Investigation Plans - recent experience with Type 2 diabetes PIPs

Mette  Due Theilade Thomsen

Mette Due Theilade Thomsen, Principal Scientist, Paediatric Investigation Plan (PIP), Regulatory Affairs , Novo Nordisk A/S, Denmark

• 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 PIPs
 

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10:30

Morning Coffee

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11:00

The challenges of developing the first drug for a life-threatening disease for which no approved drug exists

Cristina  De Min

Cristina De Min , Chief Medical Officer, NovImmune SA

• 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 approved
 

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11:40

Strategies for maximizing pediatric studies

Deborah Lee

Deborah Lee, Senior Medical Director, Clinical Affairs, Lundbeck, USA

• 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 indications
 

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12:20

Networking Lunch

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13:20

Considerations associated with the use of foods and beverages to assist administration of medicines to children

Terry Ernest

Terry Ernest, Formulation Team Manager, Global Formulation, Product Development , GlaxoSmithKline R&D

• Why are products mixed with food?
• Current practices of mixing with food
• Summary of recent EMA guidance
• Risks associated with mixing products with food
 

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14:00

NIHR clinical research network: children support and delivery of clinical research

Sabah Attar

Sabah Attar, Divisional Head of Portfolio Operations, NIHR Clinical Research Network: Children

• Current and emerging Infrastructures 
• Delivery frameworks
• NIHR Children performance to date with key examples
• Challenges and opportunities
 

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14:40

Afternoon Tea

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15:10

Practical application of physiologically based pharmacokinetic modeling to select starting doses in children

Neil Parrott

Neil Parrott, Modelling & Simulation Scientist, Pharmaceutical Sciences, Roche Innovation Center Basel, Roche Pharmaceutical Research and Early Development

• 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
 

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15:50

Learning from paediatric psychopharmacology

Philippe Auby

Philippe Auby, Vice President of Global Clinical Research, Paediatric Neuro-Psychiatry, Lundbeck, France

• Practical guidance for paediatric trials using experience from paediatric psychopharmacology
• Scientific considerations and ethical challenges
• Case studies
 

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16:30

Chairman’s Closing Remarks and Close of Day Two


Chief Medical Officer
NovImmune SA
Associate Professor
University of Copenhagen
Head, Global Scientific Affairs
Novartis Vaccines
Senior Medical Director, Clinical Affairs
Lundbeck, USA
Senior pharmaceutical assessor, Chemical Pharmaceutical Assessments (CFB), Member EMA Quality Working Party,
Medicines Evaluation Board in the Netherlands (MEB)
Director & Founder
Empowering Children | Enabling Research
Paediatric Research Consultant Specialist Medical Writer for Children and Member FP7 Ethics Panel
Paediatric Research Consultancy
CEO
klausrose Consulting
Principal Scientist, Paediatric Investigation Plan (PIP), Regulatory Affairs
Novo Nordisk A/S, Denmark
Modelling & Simulation Scientist, Pharmaceutical Sciences, Roche Innovation Center Basel
Roche Pharmaceutical Research and Early Development
Vice President of Global Clinical Research, Paediatric Neuro-Psychiatry
Lundbeck, France
Head of Clinic Paediatric Oncology,
Universitair Ziekenhuis Antwerpen, Vice Chairman, Paediatric Commitee (PDCO)
Formulation Project Leader, Paediatric & Geriatric Network Leader, Technical Research & Development
Novartis Pharmaceuticals
Divisional Head of Portfolio Operations
NIHR Clinical Research Network: Children
Director Regulatory Affairs/Policy Europe
AstraZeneca
Senior Pharmacokinetics Assessor, Statistics and Pharmacokinetics Unit
MHRA
Formulation Team Manager, Global Formulation, Product Development
GlaxoSmithKline R&D

Sponsors and Exhibitors

Official Media Partner

Supporters

Workshops

Interview with Conference Chairman, Klaus Rose, CEO from klausrose Consulting

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Q&A with Lundbeck

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Attendees at Paediatric Clinical Trials 2015

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Sponsors and Exhibitors


Molecular Profiles

Sponsors and Exhibitors
http://www.molprofiles.co.uk/

Molecular Profiles is a contract drug product development and manufacturing organization. We assist clients globally by providing pharmaceutical development services and clinical trial manufacturing, with proven expertise in development of paediatric products. We are also renowned for our expertise in materials characterization to resolve some of the toughest issues during development and in relation to intellectual property litigation.

Media Partners


Samedan

Official Media Partner
http://www.samedanltd.com/magazine/13.

International Clinical Trials (ICT) is a quarterly magazine edited by Dr Graham Hughes, and written by specialists at the forefront of clinical research. ICT’s coverage of operational developments ranges from adaptive designs in early phase trials through to post marketing research. ICT’s targeted readership consists of key decision makers across the pharmaceutical and biotech industries, along with contract partners and consultants. The magazine is distributed to pharmaceutical professionals in Europe and North America, and is accessed globally online. Read the latest issues, explore the ICT archive and subscribe at www.samedanltd.com/magazine/13.


Paediatrics and International Child Health

Official Media Partner
http://www.Maneyonline.com/pch

Published by Maney, Paediatrics and International Child Health is an international forum for all aspects of paediatrics and child health in developing and low-income countries. The international, peer-reviewed papers cover a wide range of diseases in childhood and examine the social and cultural settings in which they occur. www.maney.co.uk


Trials

Official Media Partner
http://www.trialsjournal.com

Open access, peer-reviewed, online journal that encompasses all aspects of the performance and findings of randomized controlled trials in health. The journal publishes articles on general trial methodology as well as protocols, commentaries and traditional results papers - regardless of outcome or significance of findings.

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PharmaVOICE

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Antibodies Online

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Absave

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Journal of Negative Results in Biomedicine

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Open access, peer-reviewed, online journal that provides a platform for the publication and discussion of unexpected, controversial, provocative and/or negative results in the context of current tenets. The journal strongly promotes and invites the publication of clinical trials that fall short of demonstrating an improvement over current treatments. The aim of the journal is to provide scientists and physicians with responsible and balanced information in order to improve experimental designs and clinical decisions.


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Drug Discovery Today

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SelectScience

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http://www.contract-biotechnology.com

Contract-Biotechnology.com is a web-based platform for laboratory outsourcing solutions. It is an online R&D matching tool that connects Scientifics and service and product providers worldwide. The platform Contract-Biotechnology.com would help you in the process of finding the right partner saving time and money, because with one single and secure application you would be able to receive multiple quotes quickly, keeping your contact information confidential. Contract-Biotechnology.com is the new Collaboration Network Model for Discovery Research and Development. Contract-Biotechnology.com team has extensive experience working for pharmaceuticals, biotechs, universities and academic research institutes and can help you addressing your key gaps.


Maney Publishing

Supporters
http://maneypublishing.com/

Maney Publishing delivers personalised service to authors, societies, readers and libraries for the publishing and international dissemination of high quality, peer-reviewed scholarship and research. Specialising in print and electronic journal publishing, Maney is committed to technical and editorial innovation combined with traditional values of quality and collaboration.


pharmaphorum

Supporters
http://www.pharmaphorum.com

pharmaphorum drives innovation within the pharmaceutical industry, by bringing healthcare together through a suite of media services that help produce and disseminate thought leadership, combined with an online platform for communicating messages to a global audience.Visit www.pharmaphorum.com


Journal for Clinical Studies – Your Resource for Multisite Studies & Emerging Markets

Supporters
http://www.JforCS.com

Published bi-monthly.JCS is a unique journal, dedicated to providing information to the global pharmaceutical, biotechnology, medical devices and contract research organisations. JCS details practical and theoretical operational procedures, challenges, validatory and regulatory guidelines when conducting trials on a multisite basis and particularly within the emerging markets, naïve patient population, and remote access areas. JCS provides country by country objectives and uniquely brings you experiences in therapeutic areas of Liver diseases, kidney diseases, insect borne diseases, malnutrition and under nutrition. JCS is led by a strong editorial advisory board sourced out for their experiences; you will get the most practical insight for your global studies. JCS invites you to join us, write for us, feature your experiences with us, advertise your capabilities with us, and ask our advisory board for suggestions and guidelines. Let us make health care available to all.


Cutting Edge

Supporters
http://www.cuttingedgeinfo.com

Cutting Edge Information provides customized research and benchmarking reports to pharmaceutical, biotechnology and other life science companies. We provide off-the-shelf and customized research for key areas including medical affairs, clinical development, market access, regulatory affairs, marketing management, sales and HCP fair market value.


Bioportfolio Limited

Supporters
http://www.bioportfolio.com

BioPortfolio.com is a leading news, information and knowledge resource covering the global life science industries impacted by biotechnology. The site aims to provide the lay person, the researcher and the management executive with a single location to source core information on specific bio-related topics, to collate relevant data associated with each topic and to point the user to relevant knowledge resources


British Pharmacological Society

Supporters
http://www.bps.ac.uk

The British Pharmacological Society (BPS) is the primary UK learned society concerned with research into drugs and the way they work. Our members work in academia, industry, regulatory agencies and the health services, and many are medically qualified. The Society covers the whole spectrum of pharmacology, including laboratory, clinical, and toxicological aspects. Clinical pharmacology is the medical speciality dedicated to promoting safe and effective use of medicines for patient benefit. Clinical pharmacologists work as consultants in the NHS and many hold prominent positions in UK Universities.

Holiday Inn Regents Park

Carburton Street
London W1W 5EE
United Kingdom

Holiday Inn Regents Park

Choose the well-connected Holiday Inn London-Regent's Park hotel, with a superb central London location and speedy transport links. Holiday Inn London-Regent's Park is in a leafy and cosmopolitan area of central London, a 10-minute walk from bustling Oxford Street. Leave your car in our NCP managed underground car park, and explore London by Tube. Great Portland Street Tube station is 25 metres from the hotel, from where you can reach the City and Canary Wharf in 30 minutes, and London Heathrow Airport in 45 minutes.

Wireless Internet is available throughout the hotel, and you can invite up to 300 people to events at the Academy Conference Centre, with an IT technician and break-out zones. Holiday Inn London-Regent's Park is a 10-minute walk from Santander's offices and businesses in the BT Tower. Stroll 5 minutes to Regent's Park, where you'll find London Zoo and pretty Primrose Hill. We're a 10-minute walk from Bond Street boutiques and 20 minutes from Buckingham Palace and cruises on the River Thames.

Ask our Concierge to plan your day out and book West End theatre tickets. Room Service is available 24 hours at Holiday Inn London-Regent's Park, or dine in the vibrant Junction Restaurant. Our Junction Bar has a menu of light bites, and a hot breakfast buffet is served daily.

HOTEL BOOKING FORM

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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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