The Programme

Day One
Tuesday 30 September 2008

Registration

Opening remarks from the Chair

Reviewing the regulatory environment

Jacqueline Carleer
Safety Assessor, Research and Development
FEDERAL AGENCY FOR MEDICINES AND HEALTH PRODUCTS

and Member
EMEA PAEDIATRIC COMMITTEE, PDCO

A regulatory perspective: illuminating the European wide adoption of Regulation (EC) No 1901/2006

• Assessing the uptake of the paediatric regulation
• Feedback on the quality and number of the PIPs which have been submitted
• Reviewing data discrepancies in PIPs received by the PDCO
• Offering guidance for future PIP submissions

Understanding the paediatric regulation and what it means for drug manufacturers

• Highlighting the new legislation: its pros and cons
• Emphasising the commercial and financial rewards it offers
• Exploring its impact on drug development companies
• Reviewing the progress of the legislation and how it could be adapted in the future

Paediatric regulation: scope of the paediatric obligation and paediatric rewards

• Overview of the paediatric regulation
• Scope of the paediatric obligation (new medicinal products, line extensions and variations of approved products and outstanding issues)
• Paediatric rewards (types of rewards, substantive requirements, procedural requirements and outstanding issues)
• Conclusion: is paediatric research mandatory and will it be rewarded?

A practical approach to completing a successful Paediatric Investigation Plan

• Reviewing the considerations for completing a PIP: its format and content
• Thinking strategically when completing and submitting a PIP
• Interpreting the PIP assessment process
• Achieving a successful PIP submission

Asli Santos
Head of CNS Axis, Corporate Regulatory Affairs - Europe and Portfolio Management
SANOFI-AVENTIS

EU paediatric regulation: key obligations and opportunities concerning authorised products

• Reviewing the obligations related to articles 45 and 46
• Sanofi-Aventis’ experience with submissions in relation to articles 45 and 46
• Summarising the new opportunities to be taken with a PUMA

Didem Crosby
DRA Manager, PDRM-2
F. Hoffmann-La Roche

The PIP procedure from the industry perspective

• Adjusting to the new way of thinking in the PIP era
• Working with the PDCO
• Dealing with the grey areas throughout the procedure
• Experiences from a PIP procedure

Planning a successful clinical trial for paediatrics

Assessing safety during paediatric clinical trials

• EU guideline on pharmacovigilance for medicines used by the paediatric population
• Special characteristics of paediatric pharmacovigilance
• Pitfalls in the analysis of safety data from clinical trials: signal detection in children
• Ensuring pharmacovigilance in paediatric patient populations

Model-based approaches in assessing paediatric pharmacokinetics

• Population PK of sparse data in paediatric trials: how to make more from less
• PK-PD modelling and simulation to bridge efficacy or safety
• Highlighting the ethical issues surrounding paediatric PK studies
• Designing a feasible PK study for children

Establishing the correct formulations for paediatrics

• Understanding the different factors to take into account and the challenges these may pose
• Addressing important safety and acceptability issues
• Exploring dosage forms and size, administration, taste and non-intrusive methods
• Developing simple and effective paediatric formulations

Afternoon refreshments

Focusing on paediatric trials in progress

Experiences from a paediatric clinical trial involving life threatened neonates encompassing the clinical perspectives of parents, site staff and sponsor staff

• Managing the informed consent process in a stressful situation
• Parents’ involvement in the reconstitution of IMP
• Interaction with sites having limited clinical trial experience and GCP awareness, e.g. safety reporting
• Importance of the site staff/parent relationship
• Patient recruitment and retention: challenges and lessons learned

Developing clinical trials in child psychiatry disorders: challenges and opportunities

• Mental health disorders and implications in clinical trials
• Brain and cognitive development in children and their impact on studies
• Sources of information in child psychiatry trials
• Endpoints and rating scales
• Practical considerations and conclusions

Closing remarks from the Chair

End of day one

Day Two
Wednesday 1 October 2008

Registration

Opening remarks from the Chair

Delegates will be able to attend three one-hour roundtable discussion groups from a selection of key topics. An industry expert will facilitate each session, ensuring an exchange of opinions and ideas related to an aspect of Paediatric Drug Development and Clinical Trials.

12:30

Lunch

Connecting the industry and the patient

Patient organisations and their role in supporting clinical trials in children

• Considering patient databases and their importance in paediatric clinical trials
• Designing a feasible clinical trial for children
• Best practice methods for recruiting patients
• Maintaining families in clinical trials
• Ensuring long-term follow up for safety and efficacy

Tackling the key challenges in developing medicine for children

Julie McGinnis
Senior Manager,
Study Management
AMGEN

Facilitating patient recruitment and retention: a new approach to paediatric clinical trials

• Providing the necessary training needs for the study staff
• Preparing the sites for a new approach and an increase in paediatric trials
• Outlining the difficulties of recruiting children for clinical trials
• Establishing the methods of ensuring a large enough sample size
• Deciding on acceptable procedures for children
• Successful patient recruitment and retention for paediatric trials

Afternoon refreshments

Reviewing the different approach that sites must undertake with paediatric clinical trials

• Understanding the different procedures and GCPs in trials with children
• Providing the necessary training needs for the study staff
• Preparing the sites for a new approach and an increase in paediatric trials
• Designing drug/study information for paediatricians/neonatologists, patients, parents and care givers

Chaired by

Panel discussion: the evolving paediatric environment

As a conclusion to the event a panel made up of speakers from the two days will discuss and debate questions in this interactive session.

Closing remarks from the Chair

End of conference