1. resources incorporating
trials
RegisteR by September 24th foR Reduced PRicing
evaluating
development studies
4 Pediatric
th
pediatric
project
PROUDLY PRESENTS
strategies
Clinical Trials
regulatory
solutions
clinical
guidelines
children
safety
Successfully Balancing Regulatory Requirements While
update
Designing and Conducting Effective Pediatric Clinical Studies
November 11-12, 2010, Westin Washingtion, National Harbor, MD
F e At U r e D S e S S I O N S :
Assessing Pediatric Proposals from the EMA Perspective:
Determining Key Challenges and Opportunities in
Pediatric Studies
Gilles Vassal, MD PhD, Director Clinical and Translational Research, Institut Gustave-Roussy, Villejuif, France, EMA PDCO MEMbER
ConferenCe Kevin Connolly, FRCPI, Director, Paediatric Clinical Development, Portiuncula Hospital, Galway Ireland, EMA PDCO MEMbER
Presenters:
Abbott Laboratories
Bolstering Safety Data: Forming Networks Between
Sponsors and Investigators
AstraZeneca
Kenneth J. Cohen, MD, MBA,Clinical Director, Pediatric Oncology, SIDNEY KIMMEL COMPREHENSIVE,
Bristol-Myers Squibb CANCER CENTER AT JOHNS HOPKINS
FDA
Strategies for Dealing with Issues of Consent and Assent
Ikaria
to Reduce Patient Dropout Rates in Long Term Studies
Institut Gustave-Roussy
Brahm Goldstein, MD, Senior Medical Director, Translational Science, IKARIA
John Hopkins Robert “Skip” Nelson, MD PhD, Pediatric Ethicist, Office of Pediatric Therapeutics, FDA
Merck Laboratories
Plus! Examining Three Approved PIP Case Studies
Pfizer
Seaside Therapeutics
Vertex Pharmaceuticals SponSored By:
To Register Call 866-207-6528 or visit us at www.exlpharma.com/pediatrics

2. Who Should Attend? Dear Colleague,
Professionals from Pharmaceutical, Biotechnology and
Medical Device companies with responsibilities in the fol- It is with great pleasure that I invite you to the 4th Pediatric Clinical Trials Confer-
lowing areas: ence: Successfully Balancing Regulatory Requirements While Designing and Con-
ducting Effective Pediatric Clinical Studies.
• Clinical Operations
The development of new medications for children is a rapidly expanding area of
• Clinical Development
pharmaceutical research. While this growth has occurred partly in response to
• Regulatory Affairs evolving regulatory requirements by the FDA in 1997 and the EMEA in 2007, the
• Translational Medicine development of more marketed drugs for children is a societal imperative.
• Pediatric/ Paediatric/Perinatal
• Drug Development The successful conduct of pediatric trials involves an in-depth understanding of
• Clinical Research current regulatory mandates as well as clinical, operational, ethical, and legal chal-
• Clinical Project lenges. Furthermore, the incorporation of pediatric clinical research into an overall
• Trial Management organizational framework for drug development can be equally as daunting.
• R&D Operations
• Drug Safety In response to the increased pressure to conduct clinical trials in children, this fo-
rum will serve to provide a foundation for the successful implementation of pedi-
• Project Management
atric trials. Key industry and academic experts will address the unique concerns
• Clinical Trial Management
raised by ethics committees, investigators, and parents surrounding the conduct
• Medical Affairs
of clinical trials in children and will provide strategies for incorporating a pediatric
• Clinical Affairs clinical program into an existing global clinical trials paradigm.
• Pharmacovigilance
• Pharmacology
resources
Please join us for in-depth discussion and a shared learning opportunity.
trials
This event is also of interest to: incorporating
evaluating
Sincerely,
• CROs specializing in pediatric studies
development
Julie A. Rosenberg, MD
studies
pediatric
• Patient Recruitment Firms
Director, Oncology Global Clinical Research
project
• Consultants who can assist in the PIP scripting BRISTOL-MYERS SQUIBB
process and trial design CONFERENCE CHAIR
• Regulatory firms working in trial design
strategies
regulatory
solutions
clinical
guidelines
children
safety
update
Venue Information The Westin Washington National Harbor
171 Waterfront Street
National Harbor, Maryland 20745
For Reservations please call (301) 567-3999
Discounts End on October 20, 2010
Rising up on the shores of the scenic Potomac River, The Westin Washington Na-
tional Harbor looks out over views of our nation’s capital and its many landmarks.
One of the newest hotel additions to Washington, DC, the Westin stands on the
banks of the Potomac River, 10 minutes to Washington DC proper and close in prox-
imity to Old Town Alexandria, Arlington, and Georgetown.
Please contact the hotel directly at (301) 567-3999 to book your room. Please men-
tion ExL Pharma and the 4th Pediatric Clinical Trial conference to take advantage
of this discount. You must book your room by October 20, 2010 to be eligible for
d
the discounted rate. Please book your room early as the rooms available at this rate
are limited and review your hotel confirmation to ensure that you received the ExL
group rate.

3. Preliminary Agenda
DAy ONe | Thursday, November 11, 2010
7:30 REgiSTRaTion anD ConTinEnTal BREakfaST
8:30 Chairperson’s Opening Remarks
Julie A. Rosenberg, MD, Director, Oncology Global Clinical Research .......................................................................................................................................
BRISTOL-MYERS SQUIBB 12:30 lunChEon
8:45 R E GU L ATO RY U P DAT E 1:30 ETHICS
Evaluating Global Regulatory Perspectives in Future Pediatric Defining the Latest Ethical Pediatric Drug Trial Guidelines in
Drug Development Evaluating Design and Implementation Strategies
• Regulatory overview and update on 2012 PREA and BPCA sunset provisions • Forming frameworks to guide and monitor drugs development in children
• Update on the Best Pharmaceuticals for Children Act (BPCA) priority list • Examining safety, pharmacokinetics, and initial indications of efficacy
• Examining key issues of interpretation and effectiveness of EMA legislation • Criteria for IRB approval of research
△ Timing of discussions with coordinator/rapporteur/peer-reviewer and • Ethical issues of particular concern in drug investigations in pediatric
oral explanations to address outstanding issues populations
△ Determining the value of pre-submission discussions with the EMA • Recruitment challenges and proper ethical guidelines in child assent
△ Evaluating the core reasons for the high number of withdrawn PIP sub
missions Paul Wang, MD FAAP, Vice President, Clinical Development, SEASIDE THERAPEUTICS
• Understanding the role of WHO’s Better Medicines for Children initiative
and the new Pediatric medicines Regulators Network (PmRN) 2:15 C O R P O R AT E S T RU C T U R E S D I S C US S I O N
Thomas Hassall, Senior Director, Global Pharmaceutical Regulatory Affairs Assessing Organizational Structures to Best Incorporate Pediatric
ABBOTT LABORATORIES Studies into the Product Development Process
Emma Du Four, Director, Regulatory Policy & Intelligence, Global Pharmaceutical Regu- • Assessing the scientific and biologic rationale in considering pediatrics
latory and Medical Sciences, ABBOTT LABORATORIES, UK during the drug development process
• Incorporating pediatric plans in each development program from the
9:30 E U R E GU L ATO RY P E R S P E C T I V E Leadership level
Assessing Pediatric Proposals from the EMA Perspective: • Involving pediatricians throughout development
Determining Key Challenges and Opportunities in Pediatric Studies • Determining how early to evaluate pediatric populations
• Examining an historical background of the PIP and it’s progression Jeffrey Skolnik, MD, Director of Clinical Development, ASTRAZENECA
• Understanding the PDCO perspective on PIP timelines and stages .......................................................................................................................................
• Assessing the impact of the PIP process to pediatric studies 3:00 nETWoRking BREak
o Approval trends in the EU
o Evaluating the causes for incomplete PIP submissions 3:30 A A P I N I TAT I V E
• Discussing the negotiation and facilitating process with PDCO
Gilles Vassal, MD PhD, Director Clinical and Translational Research, Institut Gustave-
Examining the Developing AAP Initiative on Medical Advances In
Roussy, Villejuif, France, EMA PDCO Member Pediatrics
Kevin Connolly, FRCPI, Director, Paediatric Clinical Development • Evaluating the gaps between pediatrician abilities and understandings and
Portiuncula Hospital, Galway Ireland, EMA PDCO Member the increasingly stringent requirements of the pharmaceutical industry
• Bolstering the ability to uncover qualified pediatrician investigators do
....................................................................................................................................... mestically by raising the profile of industry-focused pediatricians
10:15 nETWoRking BREak
Charles A Thompson, Medical Director, Infectious Diseases, PFIZER (Chair, AAP SoMAP)
Brahm Goldstein, MD, Senior Medical Director, Translational Science, IKARIA
10:45 PIP CASE STUDIES Paul Wang, MD FAAP, Vice President, Clinical Development, SEASIDE THERAPEUTICS
Examining Three Approved PIP Applications
We have brought together three different Paediatric Investigational Plans (PIPs)
4:15 RO U N D TA B L E D I S C US S I O NS
recently approved by the EMA Roundtable Discussions On Specific Therapeutic Areas
Roundtable discussions are an opportunity to break into smaller, concentrat-
• Discuss the challenges with and approaches taken ed groups and focus on specific issues. Each table is moderated to facilitate
• Examine the negotiation process with the EMA deeper discussion.
• Evaluate lessons learned • Oncology
• Neonatology/Critical Care
Each PIP will be discussed for 30 minutes, followed by a group Q&A and panel • CNS
discussion. • Infectious Disease
.......................................................................................................................................
Liz Morgan, Regulatory Affairs, UK, PFIZER
5:30 CloSE of Day onE
Mark De Roche, Senior Director, Regulatory Affairs, VERTEX PHARMACEUTICALS
Samuel C Blackman, Associate Director, Experimental Medicine/Oncology,
resources
trials
MERCK RESEARCH LABORATORIES
incorporating
aluating
development studies
pediatric resources
project
rials
incorpora
studie
ies
development
ions
al

4. Preliminary Agenda
DAy TWO | Friday, November 12, 2010
7:30 ConTinEnTal BREakfaST
11:00 I N V E S T I GAT O R P E R S P E C T I V E
8:30 Chair’s Welcome and Roundtable Recap Bolstering Safety Data: Forming Networks Between
Julie A. Rosenberg, MD, Director, Oncology Global Clinical Research Sponsors and Investigators
BRISTOL-MYERS SQUIBB • Building regional and global ‘sub-specialty’ networks to further
investigation into a wide class of initiatives
9:00 I N F O R M E D C O NS E N T • Evaluating the investigator initiated approach to pediatric trials
Strategies for Dealing with Issues of Consent and Assent to • Examining strategies unique to pediatrics
Reduce Patient Dropout Rates in Long Term Studies Kenneth J. Cohen, MD, MBA, Clinical Director, Pediatric Oncology,
SIDNEY KIMMEL COMPREHENSIVE, CANCER CENTER AT JOHNS HOPKINS
• Writing effective consent form information that covers tangible and con
ceptual aspects of the trial Gilles Vassal, MD PhD, Director Clinical and Translational Research,
• Building effective trial information that can best inform patients and Institut Gustave-Roussy, Villejuif, France, EMA PDCO Member
families to changes during trials
• Determining when you do and do not need assent forms 11:45
Brahm Goldstein, MD MCR FAAP FCCM, Senior Medical Director, Translational Science, Building Effective Pediatric Clinical Trials Unit
IKARIA
• Evaluating the Cost to Benefit in Determining the Number of Clinical Sites
Robert “Skip” Nelson, MD PhD, Pediatric Ethicist, Office of Pediatric Therapeutics • Establishing pediatric administrative staff
FOOD AND DRUG ADMINISTRATION • Assessing the number of sites necessary from an administrative viewpoint
Ron Portman, MD, Group Director, Pediatric Drug Development Program,
9:45 PA RT N E R S H I P D I S C US S I O N BRISTOL-MYERS SQUIBB
Forming Global Patient Recruitment and Retention .......................................................................................................................................
Strategies to Increase Pediatric Trial Involvement 12:30 CloSE of Main ConfEREnCE
• Best and emerging practices for communicating with patients and family
members about participation
• Examining age group specific support methodologies
• Understanding and overcoming special retention and compliance obstacles
• Engaging stakeholders to maximize trial participation Take advantage of our group discount rates to
Ann Kottcamp, Vice President, CNS & Mental Health, MMG
strengthen your entire team’s pediatric strategies.
.......................................................................................................................................
10:30 nETWoRking BREak
Post-Conference Workshop
POST-CONFereNCe WOrkSHOP
Developing Formulation and Dose Selection Strategies that
ensure High Levels of efficacy and Safety in Pediatric Studies
Pharmacological Strategies in Pediatric Drug Development: Getting to the Right Dose
Evaluating current understanding in pediatric pharmacology • Use of M&S in the evaluating pediatric study results
• Reviewing the regulatory environment • Physiologically-based pharmacokinetic modeling
• Drug safety studies
• Extrapolating data from adult studies Formulation Development Strategies
• Strategies to increase drug product palatability
Modeling and Simulation Examples in PK/PD • Tase masking, texture, smell in developing dosage forms
• Use of M&S in pediatric trial design
.................................................................................................................................................................................................................................
12:30 lunChEon foR WoRkShoP PaRTiCiPanTS
.................................................................................................................................................................................................................................
1:30 WoRkShoP BEginS
.................................................................................................................................................................................................................................
2:45 nETWoRking BREak
.................................................................................................................................................................................................................................
4:30 WoRkShoP EnDS
To Register Call 866-207-6528 or visit us
at www.exlpharma.com/pediatrics

5. resources
trials
incorporating
evaluating
sPonsorshiP anD exhibiting oPPortunities
development studies
Do you want to spread the word about your organization’s solutions and services to potential clients
who will be attending this event? Take advantage of the opportunity to exhibit, present an education-
pediatric
al session, host a networking event, or distribute promotional items to attendees. ExL Pharma will
work closely with you to customize a package that will suit all of your needs. To learn more about
project
these opportunities, please contact:
Steve Decker
strategies
Business Development Manager
regulatory
solutions
clinical
212.400.6234, sdecker@exlpharma.com
guidelines
children
five Ways to register
Fax: 888-221-6750 Phone: 866-207-6528
safety
Mail: ExL Events, Inc. Online: www.exlpharma.com/pediatrics
update
555 8th Ave, Ste 310
New York, NY 10018
Email: register@exlpharma.com
MeDia Partners
registration fees for attenDing exL Make checks payable to ExL Events, Inc. and write code
P1902 on your check.
PharMa’s 4th PeDiatriC CLiniCaL triaLs: You may also use Visa, MasterCard, Discover or American
Express.
Payments must be received in full prior to the commence-
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6. resources
trials
incorporating
RegisteR by September 24th foR Reduced PRicing
evaluating
development studies
4th Pediatric
pediatric
project
PROUDLY PRESENTS
strategies
Clinical Trials
regulatory
solutions
PLEASE MENTION PRIORITY CODE: P1902
clinical
Exp. Date:
guidelines
children
safety
555 8th Avenue, Suite 310
Successfully Balancing Regulatory Requirements While
Designing and Conducting Effective Pediatric Clinical Studies
update
New York, NY 10018
November 11-12, 2010, Westin Washingtion, National Harbor, MD
F e At U r e D S e S S I O N S :
Assessing Pediatric Proposals from the EMA
Perspective: Determining Key Challenges and
Name on Card
Card Number
Opportunities in Pediatric Studies
Signature
Gilles Vassal, MD PhD, Director Clinical and Translational Research,
Institut Gustave-Roussy, Villejuif, France, EMA PDCO MEMbER
Kevin Connolly, FRCPI, Director, Paediatric Clinical Development, Portiuncula Hospital,
ConferenCe Galway Ireland, EMA PDCO MEMbER
Presenters:
Please contact me: I’m interested in marketing opportunities at this event
Abbott Laboratories
Bolstering Safety Data: Forming
Networks Between Sponsors and Investigators
register@exlpharma.com
I wish to receive email updates on ExL Pharma’s upcoming events
AstraZeneca
www.exlpharma.com
Kenneth J. Cohen, MD, MBA,Clinical Director, Pediatric Oncology, SIDNEY KIMMEL
Zip
Yes! Register me for the conference and workshop:
COMPREHENSIVE, CANCER CENTER AT JOHNS HOPKINS
Bristol-Myers Squibb
888-221-6750
FDA Strategies for Dealing with Issues of
Consent and Assent to Reduce Patient
Ikaria
State
Dropout Rates in Long Term Studies
Title
Fax
Institut Gustave-Roussy Brahm Goldstein, MD, Senior Medical Director, Translational Science, IKARIA
Robert “Skip” Nelson, MD PhD, Pediatric Ethicist, Office of Pediatric Therapeutics, FDA
Register me for the conference only
John Hopkins
Merck Laboratories
Plus! Examining Three Approved PIP Case Studies
New York, NY 10018
Pfizer
TO R E G I S T E R
555 8th Avenue,
ExL Events, Inc.
866-207-6528
Seaside Therapeutics
Suite 310
Vertex Pharmaceuticals
SponSored By:
Telephone
Company
Address
Name
Email
Dept.
City
To Register Call 866-207-6528 or visit us at www.exlpharma.com/pediatrics