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Pediatric Research Conference 2014: Transforming the Pediatric Research Landscape through Innovation and Forward Thinking

Nov 6 2014 1:00PM - Nov 7 2014 5:00PM | Bethesda North Marriott Hotel and Conference Center 5701 Marinelli Road, North Bethesda, MD 20852 USA

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Overview 

Tutorial:  November 6th - From the Pediatric Case Files of FDA
Meeting:  November 6-7th

Join key stakeholders within the global pediatric research community to discuss the challenges faced today and on the horizon for constructing and conducting global pediatric research programs.

There is widespread agreement that innovative pediatric legislation within the US and EU has fostered major advances in pediatric research program methodologies and designs. These significant advances include the new statutory frameworks set forth by the FDA and EMA for pediatric research to advance the health and wellness of children. 

Working together in an effort to facilitate global pediatric research planning, the US and Europe are challenging both industry and regulatory agencies to determine efficient and effective measures to expand their thinking and consider new approaches for clinical trial design to reserve scarce resources while navigating the complexities of pediatric research.

Featured Topics 

During this conference you will hear from leaders who are working on challenges such as:

  • Seeking approaches for novel trial designs for the evolving trend of smaller more targeted studies
  • Defining measurable endpoints and feasible timing for durations of studies
  • Reviewing policies to incentivize rare pediatric diseases
  • Building infrastructures to support successful pediatric research
  • Supporting pediatric patient and family engagement during pediatric medical product development
  • Ethics and Safety

Who Should Attend 

Professionals involved in:

  • Regulatory, clinical, and drug development professionals from health authorities and within industry
  • Employees from clinical research organizations (CRO) and individuals involved in pediatric clinical trials
  • Pediatricians
  • Representatives from academia, pediatric societies, and networks
  • Parents and patient advocacy organizers
  • Any stakeholder interested in the development of better pediatric research programs

Learning Objectives 

At the conclusion of this meeting, participants should be able to:

  • Discuss the recent changes in the regulatory environment for the US, EU and Japan
  • Discuss the impact of international collaborations on pediatric drug development
  • Evaluate approaches to using the Pediatric Study Plan (PSP) and Pediatric Investigation Plan (PIP) in pediatric planning
  • Discuss standards for infrastructure and elements for developing therapeutic products
  • Describe innovations in pediatric therapeutic development
  • Discuss challenges in the development of products for pediatric diseases, including ethical considerations and short- and long-term safety

Special Offers 

EARLY BIRD RATE!
DIA Industry Members who register by
October 16, 2014, save $100!


Group Discounts Available!
Register 3 and Get the 4th FREE!

Hotel & Travel 

Bethesda North Marriott Hotel and Conference Center
A limited number of rooms are available at the reduced rate shown below (rate is guaranteed until October 15, 2014, or until room block is filled).  Please note: In order to receive the reduced room rate, hotel reservations must be made with Travel Planners and not directly with the hotel.  Contact information for Travel Planners is as follows: Attendees can follow this link  to make their hotel reservations, or by calling  +1.212.532.1660 or 1.800.221.3531 in the U.S. When calling please select option 1 for “Hotel Reservations,” and inform the phone agent that you are making a reservation for Event #14020.

Standard Room Rate $229

Hotel Address: 5701 Marinelli Road, North Bethesda, MD 20852

PLEASE READ
Warning: Unauthorized Solicitation

The most convenient airport is Washington Dulles International Airport – IAD or Ronald Reagan Washington National Airport - DCA and attendees should make both airline and hotel reservations as early as possible

Contact Information 

Printable Registration Form

Registration Questions
Toll Free 1.888.257.6457
Phone +1.215.442.6100
Fax +1.215.442.6199
Monday-Friday 8:30AM-8:00PM ET
CustomerService@diahome.org

Agenda Details
Meredith Kaganovskiy, Content Lead
Phone +1.215.442.6117
Fax +1.215.442.6199
Meredith.Kaganovskiy@diahome.org

Event Logistics
Stephanie Ritter, Event Planner
Phone +1.215.442. 6149
Fax +1.215.442.6199
Stephanie.Ritter@diahome.org

Continuing Education 

Drug Information Association has been accredited as an Authorized Provider by the International Association for Continuing Education and Training (IACET).

As an IACET Authorized Provider, Drug Information Association offers CEUs for its programs that qualify under the ANSI/IACET Standard. Drug Information Association is authorized by IACET to offer 1.4 CEUs <.3 CEUs for the tutorial and 1.1. CEUs for the conference) for the program. Participants must attend the entire program in order to be able to receive an IACET statement of credit. No partial credit will be awarded.

DIA’s Certificate Program
This program is part of DIA’s Certificate Program and is awarded the following:
• Clinical Research Certificate Program: 6 Elective Units
• Regulatory Affairs Certificate Program: 6 Elective Units

For more information go to www.diahome.org/certificateprograms

Name Credit Type Max Credits CEU
Tutorial 1: From the Pediatric Case Files of FDA IACET 3.25 0.300
Pediatric Research Conference 2014 IACET 10.50 1.100

Disclosure Policy:

It is DIA policy that anyone in a position to control the content of a continuing education activity must disclose to the program audience (1) any real or apparent conflict(s) of interest related to the content of their presentation and/or the educational activity, and (2) discussions of unlabeled or unapproved uses of drugs or medical devices. Disclosure statements will be included in the course materials.

Statement of Credit:

If you would like to receive a statement of credit, you must attend the program , sign in each day of the program, and complete the on-line credit request process through My Transcript. To access My Transcript, please go to www.diahome.org, select “Login to My DIA” and you will be prompted for your user ID and password. Select “My Transcript” (left side bar) and “Credit Request” to process your credit request. Participants will be able to download a statement of credit upon successful submission of the credit request. My Transcript will be available for credit requests on Friday, November 21, 2014.

To view DIA’s Grievance Policy, please visit the CE page on DIA’s website at www.diahome.org/CE

CONTINUING EDUCATION CREDIT ALLOCATION
• Tutorial: IACET .3 CEUs
• Conference: IACET 1.1 CEUs

Program Committee 

Previous Next

Agenda  

Tutorials Thursday, Nov 06, 2014

  • 8:00AM - 11:30AM

    From the Pediatric Case Files of FDA

    Ever wondered why FDA granted a waiver under PREA for one product but required studies under PREA for a similar product? Join FDA for a stimulating and challenging tutorial about issues related to pediatric product development. Cases will be presented that illustrate a variety of common and not-so-common situations that the Agency has addressed in pediatric product development since the passage of BPCA and PREA. FDA will guide the discussion but audience participation is requested! 

Day 1 Thursday, Nov 06, 2014

  • 1:00PM - 1:10PM

    Welcome Remarks and Overview of the Pediatric Research Conference

    Speaker(s):

    • Janet Jenkins-Showalter
      Senior Regulatory Group Director, Regulatory Policy and Intelligence
      Genentech, A Member of the Roche Group, United States
    • Chin Koerner, MS
      Executive Director, Regulatory Policy
      Novartis Pharmaceuticals Corporation, United States
  • 1:10PM - 1:40PM

    State of Pediatric Research: Innovation on Behalf of Children


    Session Chair(s):

    • Janet Jenkins-Showalter
      Senior Regulatory Group Director, Regulatory Policy and Intelligence
      Genentech, A Member of the Roche Group, United States
    • Chin Koerner, MS
      Executive Director, Regulatory Policy
      Novartis Pharmaceuticals Corporation, United States

    Dr. Spielberg will discuss some of the drivers of innovation and then focus on multiple spheres of innovation. It is the intersection and skillful, coordinated application of new knowledge across disciplines that will help drive benefit for children in need. Science is advancing at an incredible rate; children's needs demand that we advance our "sociology" of conversion of knowledge into practical solutions to address those needs.

    Speaker(s):

    • Stephen P. Spielberg, MD, PhD
      Editor-in-Chief, Therapeutic Innovation & Regulatory Science
      DIA, United States
  • 1:40PM - 3:10PM

    Session 1: Pediatric FDASIA: What’s working? What’s not? and What Does the Future Look Like?


    Session Chair(s):

    • Lynne P. Yao, MD
      Associate Director, Pediatric and Maternal Health Staff, OND, CDER
      FDA, United States

    Under the Food and Drug Administration Safety and Innovation Act (FDASIA), passed by Congress, and signed into law in July, 2012, the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA) were permanently reauthorized. The permanency of these important pediatric product development laws is noteworthy. Moreover, FDASIA also included changes to both BPCA and PREA that have had an impact on both FDA and Industry. This session will review the changes to pediatric product development under FDASIA from both the FDA and Industry perspective. The session will also discuss how these changes may affect future pediatric product development.

    Speaker(s):

    • CDER's Perspective
      Lynne P. Yao, MD
      Associate Director, Pediatric and Maternal Health Staff, OND, CDER
      FDA, United States
    • Industry Perspective
      Albert J. Allen, MD, PhD
      Senior Medical Fellow, Bioethics & Pediatric Capabilities
      Eli Lilly and Company, United States
    • Panel Discussion
      All Session Speakers, United States
  • 3:10PM - 3:30PM

    Refreshment Break
  • 3:30PM - 5:00PM

    Session 2: International Update: Priorities and Hot Topics


    Session Chair(s):

    • Dianne Murphy, MD
      Director, Office of Pediatric Therapeutics, Office of the Commissioner
      FDA, United States

    This session will focus on the importance of international collaboration in the field of pediatric product development. Harmonization efforts will be reviewed including perspectives from the FDA, EMA and PMDA. Additionally, brief comments on areas where the ICH E-11 Pediatric document is being considered for changes will be provided.

    Speaker(s):

    • Harmonization on a Global Stage: Progress and the Common Commentary
      Jean Temeck, MD
      Lead Medical Officer, Office of Pediatric Therapeutics, OC
      FDA, United States
    • Improving Pediatric Therapeutic Information: EU Regulatory Experience - Virtual Presentation
      Paolo Tomasi, MD, PhD
      Head of Paediatric Medicines
      European Medicines Agency, European Union, United Kingdom
    • PMDA: New Pediatric Legislation
      Motoko Ishikawa, MS
      Reviewer, Office of New Drug III
      Pharmaceuticals and Medical Devices Agency (PMDA), Japan
    • Improving Pediatric Therapeutic Information: EU Regulatory Experience - Virtual Presentation
      Jordi Llinares Garcia, DrMed, MSc
      Head of Product Development Scientific Support Department
      European Medicines Agency, European Union, United Kingdom
    • Panel Discussion
      All Session Speakers, United States
  • 5:00PM - 6:00PM

    Reception

Day 2 Friday, Nov 07, 2014

  • 7:00AM - 8:00AM

    Continental Breakfast
  • 8:00AM - 8:10AM

    Welcome to Day Two

    Speaker(s):

    • Janet Jenkins-Showalter
      Senior Regulatory Group Director, Regulatory Policy and Intelligence
      Genentech, A Member of the Roche Group, United States
    • Chin Koerner, MS
      Executive Director, Regulatory Policy
      Novartis Pharmaceuticals Corporation, United States
  • 8:10AM - 8:55AM

    Session 3: Pediatric Patient Engagement


    Session Chair(s):

    • Diane D. Edquist Dorman
      Rare Diseases, Patient Advocacy; Vice President, Public Policy
      National Organization For Rare Disorders (NORD), United States

    The rare disease patient community has played an increasingly influential role in determining the direction of research and therapy development. With little research being conducted for so few rare diseases and the lack of effective therapies for most, the stakes are high. In this session we will take a closer look at how the last 30 years since the passage of the orphan drug act has shaped the research landscape. Learn from a mother who lost a child to a rare cancer and was determined to change the legislative and regulatory landscape to help all children live fulfilling and healthy lives.

    Speaker(s):

    • Advocacy of the Rare Disease Community: From Broad View to Pediatric
      Diane D. Edquist Dorman
      Rare Diseases, Patient Advocacy; Vice President, Public Policy
      National Organization For Rare Disorders (NORD), United States
    • From Reaction to Action - A Mom on a Mission: Changing the Landscape of Pediatric Research
      Nancy Goodman, JD
      Executive Director
      Kids Vs. Cancer, United States
    • Panel Discussion
      All Session Speakers, United States
  • 8:55AM - 10:25AM

    Session 4: Building an Infrastructure of Excellence in Pediatric Research


    Session Chair(s):

    • Christina Bucci-Rechtweg, DrMed, MD
      Head, Pediatric and Maternal Health Policy, Global Drug Regulatory Affairs
      Novartis Pharmaceuticals Corporation, United States

    The caliber of infrastructure to support pediatric clinical research is fundamental the development and availability of innovative and quality medicines according to the highest ethical and scientific research standards. This session will outline the critical roles that key stakeholders (patients, families, academic researchers, government and industry) play in collaborating to identify meaningful clinical endpoints or surrogates for evaluation, contributing regulatory-quality clinical data and trial design, and executing on complex clinical studies. The session will also highlight where stakeholder collaboration has struggled and succeeded on delivering on these principles. It is anticipated that the participants will take-away the need to ensure that a strong, efficient and collaborative infrastructure can enable a sustainable research environment to address pediatric needs.

    Speaker(s):

    • Conducting Clinical Trials in Small Populations: Study Design and Endpoint Considerations
      Anne R. Pariser, MD
      Lead Medical Officer, OTS, CDER
      FDA, United States
    • Need for Regulatory Level Data Quality from Academic Investigators
      Anne Zajicek
      National Institute of Child Health and Human Dev, United States
    • A Model for Trial Excellence in Pediatric Research: Delivering on Stakeholder Collaboration in an Environment of Competing Goals and Objectives
      Mark Turner
      Associate Director
      University of Liverpool, Institute of Child Health, United Kingdom
    • Panel Discussion
      All Session Speakers, United States
  • 10:25AM - 10:45AM

    Refreshment Break
  • 10:45AM - 12:15PM

    Session 5: Innovation in Pediatric Research: Novel Clinical Trial Design and Regulatory Framework


    Session Chair(s):

    • Janet Jenkins-Showalter
      Senior Regulatory Group Director, Regulatory Policy and Intelligence
      Genentech, A Member of the Roche Group, United States

    In order to obtain the necessary data to determine the safety and efficacy of new drugs, biologics and devices for children, innovative and feasible studies must be designed and executed. Often, this will involve the careful collaboration and partnership of key stakeholders. Presentations in this session will feature: (1) an innovative trial design framework that uses a mechanism of action (MOA) approach to evaluate molecules to facilitate early access to innovative therapies for children with cancer. The approach matches the biology of tumors that occur in childhood to the MOA of drugs under development, rather than relying upon the disease targets chosen in adult development programs; (2) efforts currently ongoing to develop a partnership of industry, academia, regulators, clinicians, children's hospitals, and professional societies across the globe to establish a pediatric clinical research network; and (3) FDA's perspective on innovative approaches for pediatric drug development -- what is in place, what is needed -- to ensure that important therapies are, and will continue to be, available for pediatric use?

    Speaker(s):

    • Innovative Approaches to Drug Development in Pediatric Oncology: The Matrix Trial Concept
      Raphael Rousseau, MD, PhD
      Assoc. Group Medical Director and Leader, Global Pediatric Development Team
      Genentech, A Member of the Roche Group, United States
    • Creation of an International Pediatric Clinical Trial Network: Novel Approaches to Precompetitive Collaboration
      Ronald Portman, MD
      Pediatric Drug Development Lead
      Bristol-Myers Squibb, United States
    • Applying Current Regulatory Framework to Scientific Advances for Pediatric Clinical Trials
      Lynne P. Yao, MD
      Associate Director, Pediatric and Maternal Health Staff, OND, CDER
      FDA, United States
    • Panel Discussion
      All Session Speakers, United States
  • 12:15PM - 1:30PM

    Lunch
  • 1:30PM - 3:00PM

    Session 6: New Trends in Pediatric Science


    Session Chair(s):

    • Stephen P. Spielberg, MD, PhD
      Editor-in-Chief, Therapeutic Innovation & Regulatory Science
      DIA, United States

    We will be focusing on several areas of science impacting pediatric drug development. Modeling and simulation are increasingly being used to improve pediatric clinical trials, taking advantage of all prior knowledge about a compound to develop efficient, informative, and practical pediatric studies. "Big data" offers the prospect of new sources of information about the safety and effectiveness of medications in children in the real world. Exploration of the complex underpinnings of common diseases down to the genomic level offers the hope of development of targeted therapeutics with greater efficacy. The complexity of the relationship between genotype and phenotype suggests that future therapeutics is likely to involve several interventions aimed at several targets, and the need for further evolution of clinical study design.

    Speaker(s):

    • Modeling and Simulation: Using All Available Knowledge to Optimize Clinical Trials
      Jeffrey S Barrett, PhD
      Vice President
      Sanofi Pharmaceuticals , United States
    • Big Data
      Eric D. Perakslis
      Executive Director, Center for Biomedical Informatics & Countway Library of Med
      Harvard Medical School, United States
    • Systems Biology: Understanding Complex Pathways of Disease Pathogenesis and Implications for Therapeutics
      Hakon Hakonarson
      Director, Genomics
      The Children's Hospital of Philadelphia, United States
    • Panel Discussion
      All Session Speakers, United States
  • 3:00PM - 3:15PM

    Refreshment Break
  • 3:15PM - 4:30PM

    Session 7: Pediatric Safety


    Session Chair(s):

    • Andrew E. Mulberg, MD
      Division Deputy Director, Gastroenterology and Inborn Errors Products, OND, CDER
      FDA, United States

    The goal of this session is to focus on safety and ethical considerations in developing short- and long-term studies of medical products in children. There will be discussion of obstacles in developing short- and long-term medical product safety assessments in children, for example, safety of long term antibiotics for UTI suppression; long term proton-pump inhibitors for GERD; long term antipsychotics; ADHD meds and asthma medications. There will also be discussion of the ethical framework for conducting pediatric clinical trials, including newborns, focusing on early phase first-in-human trials and assessment of risk and potential benefit in pediatric clinical trials.

    Speaker(s):

    • FDA's Approach to Assessing Postmarketing Safety Issues in Pediatrics
      Ann McMahon, DrMed, MS
      Director of KidNet, Deputy Dir of Science, Office of Pediatric Therapeutics, OC
      FDA, United States
    • Long Term Safety Considerations: Why are they so Difficult?
      Robert L Davis, MD, MPH
      Governor's Chair and Director, Center for Biomedical Informatics
      University of Tennessee Health Sciences Center , United States
    • Risky Business: When Children are First-in-Line for Experimental Treatments
      Robert M Nelson, MD, PhD
      Pediatric Ethicist, Office of Pediatric Therapeutics, Office of Commissioner
      FDA, United States
  • 4:30PM - 5:00PM

    Closing Keynote Address

    Speaker(s):

    • Clay Alspach
      Majority Counsel
      House Energy and Commerce Committee, United States

Registration Fees 

Member

Charitable Nonprofit/Academia Member
$710.00
Member Government
$535.00
Member Industry
$1085.00
Member Industry (As of 10/17/2014)
$1185.00

Non-Member

Charitable Nonprofit/Academia Nonmember
$935.00
NonMember Government
$760.00
NonMember Industry
$1410.00
Registration Fees for Additional Offerings
Tutorial 1: From the Pediatric Case Files of FDA - Standard Rate
$405.00
Group Discounts

Register three individuals from the same company and receive complimentary registration for a fourth! All four individuals must register and prepay at the same time – no exceptions. DIA will apply the value of the lowest applicable fee to this complimentary registration; it does NOT include fees for optional events or DIA membership. You may substitute group participants of the same membership status at any time; however, administrative fees may be incurred.

To take advantage of this offer, please print a registration form for EACH of the four registrants from your company. Include the names of all four group registrants on each of the forms and return them together to DIA.

Group registration is not available online and does not apply to the already-discounted fees for government or charitable nonprofit/academia.

Register Online

CANCELLATION POLICY: All cancellations must be received in writing two weeks before
the start of the event. Administrative fee that will be withheld from refund amount:

  • Member or Nonmember = $200
  • Government or Academia or Nonprofit (Member or Nonmember) = $100
  • Tutorial (if applicable) = $50

Cancellations must be in writing and be received two weeks before the start of the event. Registrants who do not cancel two weeks before the start of the event and do not attend the event will be responsible for the full registration fee. Registrants are responsible for cancelling their own hotel and airline reservations. DIA reserves the right to alter the venue, if necessary. If an event is cancelled, DIA is not responsible for any airfare, hotel or other costs incurred by registrants.

Unless otherwise disclosed, the statements made by speakers represent their own opinions and not necessarily those of the organization they represent, or that of the Drug Information Association. Speakers, agenda and CE information are subject to change without notice. Recording of any DIA educational material in any type of media is prohibited without prior written consent from DIA.

Participants with Disabilities:
Reasonable accommodations will be made available to persons with disabilities who attend an educational activity. Contact the DIA office in writing at least 15 days prior to event to indicate your needs.

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