The Programme

Day One
Tuesday 17th November 2009

Registration

Opening remarks from the Chair
Practical advice to comply with the paediatric legislation and submit a successful paediatric investigation plan (pip)

Hendrik van den Berg
DUTCH MEDICINES EVALUATION BOARD and Member Division 3
PDCO

View from the regulators: clarifying our interpretation of the regulations to ensure an accurate and fast PIP submission

• Reviewing the number and quality of PIP submissions to date to determine key factors leading to an accurate PIP
• Assessing waiver and deferral processes to highlight which strategy is most appropriate for your indication
• Establishing best practice approaches to completing a PIP accurately to ensure a timely approval process
• Ensuring all documentation surrounding the PIP is included in the submission to support the plan and minimise the number of queries
• Increasing the transparency of clinical trials information and results through the introduction of the EudraCT database

Michael Christiensen
Senior Regulatory Affairs Project Manager NOVO NORDISK

View from the industry: overcoming the regulatory challenges for a successful PIP application

• Highlighting the key points of the paediatric legislation to aid understanding and compliance throughout the submission process
• Reviewing how the legislation is effecting feasibility by increasing research costs to assess how the regulators can help
• Assessing how the industry and authorities can facilitate the submission process through the introduction of guidelines and support materials
• Approaching the PDCO for a deferral: advising best practice techniques and support materials to uphold the decision
• Ensuring the legislation does not effect adult trials: implementing the theory in practice
• Overcoming the challenge of writing detailed PIPs at an early stage where information may be sparse in order to meet the legislation

Morning refreshments

Panel discussion: overcoming the challenges surrounding the paediatric legislation to find a useable solution for both the industry and the authorities

• The regulator’s perspective: clarifying the most common pitfalls in order to improve submissions and minimise queries
• The industry’s perspective: applying the legislation in practice to produce a feasible paediatric trial
• Finding a compromise between the authorities and the industry to introduce a workable protocol
• Striking a balance between what is necessary and what is reasonable in order to run a practical and financially viable paediatric study
• Establishing successful negotiation between parties to address problems and find solutions and compromises

Uncovering a paediatric strategy: developing a successful indication

Dr Albert J Allen
Medical Director, Neuroscience Platform Team
ELI LILLY

Trying to satisfy diverse stakeholders: factors to consider when integrating paediatric drug development strategy into the overall strategy

• Recognising and understanding the legitimate objectives and concerns with regards to paediatric drug development of legislators, regulators, clinicians, patients, industry and other stakeholders to create a workable protocol
• Highlighting the need to integrate the planning and implementation of paediatric drug development within drug development as a whole to ensure it is effectively carried through
• Striking a "good enough" balance in paediatric planning that is somewhere between unrealistic idealism and obstructionist stonewalling in order to develop a realistic strategy

Dr Ensio Norjavaara
Senior Research Physician Clinical Pharmacology
ASTRAZENECA

Developing a global paediatric study which conforms to both FDA and EMEA regulations

• Highlighting the regulations and their differences to ensure that global paediatric plans are compliant across the board
• Determining the different considerations and definitions when applying for a waiver or a deferral
• Clarifying when a global paediatric plan should start by reviewing the timelines issued by the US and European authority bodies
• Assessing the number of patients necessary for pharmacokinetic research to establish accurate toxicity and safety assessments
• Clarifying the challenges when using different comparator drugs in a global trial to ensure consistency throughout
• Ensuring successful negotiations to develop a global study between the FDA and EMEA to ensure trial timelines are adhered to

Carrying out successful trials: pointers to follow to crease a winning formula

Charles Schmidt
Medial Expert
MEDPACE BRASIL

Conducting successful paediatric clinical trials in Latin America

• Outlining the regional expertise and benefits
• Identifying regulatory and bioethical aspects that needs to be considered when running trials in Latin America
• Discussing efficient methods to identify appropriate sites
• Uncovering successful risk management strategies
• Outlining opportunities and future trends

Dr Luc De Schaepdrijver
Research Fellow, Head of Reproductive/Juvenile and General Toxicology Late Development, Global Preclinical Development
JOHNSON & JOHNSON PHARMACEUTICAL R&D

Exploring the role of preclinical safety studies in paediatric drug development to establish their added value

• Designing preclinical safety studies in function of the paediatric trial to ensure they correspond to the end target
• Conducting juvenile toxicity studies: assessing the why, when and how for targeted trials
• Revealing key discussion points determining the design and conduct of juvenile toxicity studies
• Determining the correspondence of organ development between different species and man to provide accurate comparisons
• Ensuring that juvenile animal toxicity studies are appropriately designed to facilitate adequate identification of potential safety and pharmacokinetic issues
• Assessing examples of successful preclinical toxicity studies to uncover best practice
• Reviewing the common misunderstandings of preclinical toxicity studies to fully understand why they are necessary
• Highlighting the added value these studies may bring to the clinical trials

Stephen Heaton
Safety Risk Management Advisor for Pharmacovigilance
BAYER HEALTHCARE

Understanding the impact of safety risk management on paediatric drug development to maximise safety throughout the process

• Clarifying factors to take into account when producing a safety risk management plan for paediatric trials to provide an accurate evaluation
• Highlighting how safety risk management can influence paediatric trials to maximise the visibility of safety risks during clinical development
• Determining how a PIP application will affect the safety risk management process to better understand how the two are linked
• Reviewing best practice techniques to fully understand the emerging benefit and risk profile of a product

Establishing the correct formulations to use in paediatric trials to ensure they are acceptable and suitable for the age of the patient

• Reviewing different methods by which to administer the drug (eg syrups, liquids or chewable tablets) to facilitate compliance and guarantee it is ethically acceptable
• The taste test: introducing a drug which is palatable for children to ensure it will be accepted by the child and patient organisations
• Ensuring that the different dosage forms and sizes will not affect the drug’s properties to ensure safety is maximal
• Allowing an adequate lead-time for reformulation efforts has been included into the trial timeline to allow for necessary changes

Robert Webster
Director, Global Research and Development, Pharmacokinetics, Dynamics and Metabolism,
PFIZER UK

Overcoming the challenges of pharmacokinetic (PK) modelling in paediatric populations to ensure accurate safety and toxicology reports

• Clarifying the ethical issues with paediatric PK studies to determine best practice approaches
• Tackling the changing age, size and metabolism of paediatric patients to adjust the PK model accordingly
• Meeting ethical standards by defining the type of sampling fluid (blood, urine or saliva)
• Determining the number of patients required for a PK study to ensure accurate results
• Key factors to address when designing a feasible PK study for children where limited samples are available

The ethical roadmap: ensuring paediatric trials meet the approval of Ethics Committee

Francis P Crawley
Executive Director
GOOD CLINICAL PRACTICE ALLIANCE – EUROPE

Considering best practices for ethics in paediatric clinical trials to facilitate the ethics approval process

• Exploring the European and International Regulations and Guidance on Ethics in Paediatric Research to fully determine what needs to be covered
• Defining ethical review procedures for paediatric clinical trial proposals to establish best practice methodology
• Increasing transparency for children and their families as engaged participants in clinical trials for better understanding of the ethics behind the study
• Clarifying age-appropriate information and informed consent procedures to ensure the process meets the highest ethical standards

Day Two
Wednesday 18th November 2009

Registration

Opening remarks from the Chair

Roundtable morning
Delegates will be able to attend three one-hour roundtable discussion groups from a selection of key topics. Each session will be chaired by an industry expert who will facilitate an exchange of opinions, essential experiences and learning related to a current aspect of paediatric drug development and clinical trials

Roundtable 1 
Highlighting the key requirements for the paediatric regulations to ensure compliance

This session will cover the key elements to the paediatric regulation to fully understand what is necessary and how to go about it.

Roundtable 2
Exploring the different factors in pharmacokinetic studies for children to ensure the most informative data commensurate with optimal benefit-risk

The discussion will include, but not be limited to, such aspects as age stratification, numbers of patients, numbers of samples, population PK in paediatric patients, micro-assays etc.

Roundtable 3   
Meeting the authorities requirements by clarifying their interpretation of the paediatric regulation

During this session you will gain more insights into what the EMEA/PDCO expect when Sponsor's are applying for a PIP, deferral or a waiver.

Roundtable 4  
Developing  preclinical trials for use in paediatric studies to uncover key information they bring to paediatric trials

During this discussion you will explore the key factors involved in preclinical studies for paediatric trials such as their timing, the study end points, duration of dosing and ensuring that they correspond to the target.

Highlighting success stories: factors ensuring trials with children are effective and efficient

Dr Philippe Auby
Senior Medical Director
CORPORATE ICR FRANCE AND PAEDIATRICS LUNDBECK

Reviewing key factors to consider when writing an effective protocol for your paediatric trial to ensure the study will be efficient and the data will be reliable

• Determining the study endpoints for different age groups throughout the trial to ensure they reflect equivalent stages in the trial
• Ensuring accurate comparator studies: can the comparator drug be used across all age ranges and on a global basis? How can you validate the studies if different comparators are used?
• Effective strategies to ease the informed consent and assent process, also taking into account long term trials where children may change age groups and additional forms may be necessary
• Considering cultural and country specific differences to increase trial uptake and to ensure that accuracy is globally consistent across all sites
• Maximising patient compliance by working with families to introduce convenient visit times, frequency levels and other communication methods (eg phone or email)

Sylvie Jouve
Associate Director, Clinical Research and Development
WYETH

Overcoming challenges during the informed consent/assent processes to ensure that parents and children wholly understand the whys and whats of the clinical study

• Guidelines to establish appropriate informed consent/assent forms: what is mandatory? How can you facilitate this for parents and children?
• Establishing communication with the parents from an early stage to guarantee they fully understand and support the trial
• Developing approaches to facilitate children's understanding of the trial
• Tackling barriers to literacy/reading ability
• Uncovering winning methods to achieve a successful informed consent and assent process for fast ethics approval

Bruce Morland Consultant Paediatric Oncologist, Department of Oncology
BIRMINGHAM CHILDREN’S HOSPITAL NHS TRUST

Facilitating patient recruitment and retention for paediatric trials to produce a successful study with accurate data

• Establishing where best to approach patients to ease recruitment: working with patient groups and associations
• Maximising patient uptake by overcoming the stigma of clinical trials in paediatrics
• Designing a study that increases participation: planning visit frequency and time around family life
• Determining effective methods in which to approach the target population and their patents through different media
• Ensuring clear and concise information is provided to help parents and their children understand what is needed of them
• Tailoring communication appropriately to study participants and parents, and providing an excellent level of service to greatly increase trial retention

Dr Eric Hoedemaker Medical Director,
SANOFI AVENTIS

F.W. Durian
Independent Medical Advisor
DURIAN MEDICAL SERVICES

Case study: highlighting practical experiences in Belgium and the Netherlands, the challenges faced and solutions found

• Outlining trials which have been undertaken in the Benelux region to highlight the successes and challenges
• Reviewing patient recruitment and retention: clarifying best practice techniques
• Exploring appropriate approaches to adverse event reporting to determine the who, where and when for accurate reporting
• Analysing what was successful to ensure that future paediatric trials are carried out within the expected timeframe