Advancing Pediatric Research in the US

MEMBER EARLY-BIRD RATE Register by September 21, 2010 and SAVE $150! Advancing Pediatric Research in the US October 12-13, 2010 DoubleTree Hotel and Executive Meeting Center Bethesda, MD, USA PROGRAM CHAIRPERSON Chin Koerner PROGRAM COMMITTEE Bryan Boggs, PharmD Regulatory Director, Donna Helms, Takeda Global Research and Development Director, Neurosciences Medicines Development Center, Janet Jenkins-Showalter Director, FDA Liaison, Hoffmann-La Roche, Inc. M. Renee Simar, PhD Principal Strategist, Pediatrics, INC Research, Inc. Melissa Tassinari, PhD, DABT Senior Staff Fellow, Pediatric and Maternal Health Staff (PMHS), Office of New Drugs (OND), Group Director, Regulatory Affairs, Hoffmann-La Roche, Inc. WHO SHOULD ATTEND Academic health centers/investigator sites Business strategy Chemistry, manufacturing and controls/ Good manufacturing practices Clinical research and development/clinical supplies Clinical safety and pharmacovigilance Good clinical practices Nonclinical laboratory safety assessment Outsourcing Project management Regulatory affairs Statistics Co-located with Maternal and Pediatric Drug Safety Symposium Special 3-day Pricing. (See page 5 for details.) Join Pediatric Research Experts to Share Solutions to the Challenges Involved in Constructing and Conducting Global Pediatric Programs. Over the past three years industry has been busily climbing the steep learning curve of the EU Pediatric Legislation. With approved products and late-stage development programs under compliance with the Pediatric Investigation Plan (PIP), we have moved into a new era where early development projects are, for the first time, receiving input from the EU. How can the US FDA and industry dance with two partners and not step on anyone s toes? This workshop will review the global regulatory environment of pediatric research, share case studies for the successful implementation of clinical, nonclinical, and formulations development, and discuss the future of US pediatric research in the 2012 debate. FEATURED TOPICS Regulatory Challenges in an Evolving Global Environment How US and EU policies align (or not) to encourage pediatric research. Global Pediatric Strategy: Real Issues and Successes Industry, FDA and EMA Perspectives What we have learned in the last three years and challenges we still face. Implementation Challenges of Pediatric Research: From Early Considerations through Ethical Issues Recurrent challenges in appropriate preclinical studies, child-friendly formulations, efficient methods in pharmacokinetic sampling, study designs in rare pediatric populations, and ethical considerations. Lessons Learned on the Path Toward Better Medicines for Children What still needs to be solved to improve the path towards better medicines for children. Worldwide Headquarters Drug Information Association, Inc. 800 Enterprise Road, Suite 200 Horsham, PA 19044, USA Regional Offices Basel, Switzerland Tokyo, Japan Mumbai, India Beijing, China CONTACT INFORMATION Joanne Wallace, Program Manager, Phone +1.215.442.6180 Fax +1.215.442.6199 email Joanne.Wallace@diahome.org

2 MONDAY OCTOBER 11, 2010 6:00-8:00 PM CONFERENCE REGISTRATION TUESDAY OCTOBER 12, 2010 7:30-8:30 AM REGISTRATION AND CONTINENTAL BREAKFAST 8:30-8:45 AM WELCOME AND OPENING REMARKS PROGRAM CHAIRPERSON Chin Koerner 8:45-10:00 AM SESSION 1 PART 1 Pediatric Research Landscape in the US: Regulatory Environment Group Director, Regulatory Affairs, Hoffmann-La Roche, Inc. Janet Jenkins-Showalter Director, FDA Liaison, Hoffmann-La Roche, Inc. Pediatric development legislation has been in place in the US for over a decade. In 2007, the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA) were reauthorized under FDAAA. FDAAA mandated a number of changes to the legislation, including the formalization of an internal review committee to provide high level oversight of the activities conducted under the BPCA and PREA. In this session, FDA will provide a perspective on pediatrics post FDAAA, including a look behind the scenes at how the FDA Pediatric Review Committee operates and what goes into decisions and recommendations made by the committee. Industry will also provide insight on how they are working with FDA to design and implement successful programs, including case studies to illustrate lessons learned and best practices. US Pediatric Research Policies: Role and Impact of PeRC Lisa L. Mathis, MD Associate Director, Pediatric and Maternal Health Staff (PMHS), Office of New Drugs (OND), Implementation of FDAAA by Industry Samuel Maldonado, MD, MPH, FAAP Vice President, Pediatric Drug Development Johnson & Johnson 10:00-10:30 AM QUESTION AND ANSWER PERIOD 10:30-10:45 AM REFRESHMENT BREAK 10:45 AM-12:15 PM SESSION 1 PART 2 Pediatric Research Landscape in the US: Elements of Successful Pediatric Programs and Lessons Learned in the US Case Study/Hypothetical 1 Group Director, Regulatory Affairs Hoffmann-La Roche, Inc. Case Study/Hypothetical 2 A. J. Allen, MD, PhD, FDA Perspective on Case Studies Dr. Hari Sachs Lead Medical Officer Office of New Drugs, CDER FDA Panel Discussion CONTINUING EDUCATION This activity has been planned and implemented in accordance with the Essential Areas and policies of the Accreditation Council for Continuing Medical Education (ACCME) through the joint sponsorship of Postgraduate Institute for Medicine (PIM) and the Drug Information Association. PIM is accredited by the ACCME to provide continuing medical education for physicians. Credit Designation: PIM designates this educational activity for a maximum of 12.75 AMA PRA Category 1 Credit(s). Physicians should only claim credit commensurate with the extent of their participation in the activity. The Postgraduate Institute for Medicine (PIM) and DIA require instructors, planners, managers and other individuals who are in a position to control the content of this activity to disclose any real or apparent conflict of interest they may have as related to the content of this activity. All identified conflicts of interest are thoroughly vetted by PIM and DIA for fair balance, scientific objectivity of studies mentioned in the materials or used as the basis for content, and appropriateness of patient care recommendations. Drug Information Association has been approved as an Authorized Provider by the International Association for Continuing Education and Training (IACET), 8405 Greensboro Drive, Suite 800, McLean, VA 22102; +1.703.506.3275. Drug Information Association is authorized by IACET to offer 1.3 CEUs for this program. If you would like to receive a statement of credit, you must attend the program, scan your name badge at the DIA registration desk each day of the program, and complete the on-line credit request process through My Transcript at www.diahome.org. Participants will be able to download a statement of credit upon successful submission of the credit request. Please Note: If you do not scan your badge each day of the program, you will not be able to request continuing education credits for that portion of the program. Disclosure Policy: It is Drug Information Association 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. Faculty disclosures will be included in the course materials. Learning Objectives At the conclusion of this conference, participants should be able to: Explain and address the current state of US and EU pediatric legislation Discuss and recognize the impact of US and EU legislation on global pediatric programs Gain knowledge of how to build and implement a global pediatric program

3 12:15-1:00 PM LUNCHEON AND NETWORKING OPPORTUNITY 1:00-3:00 PM SESSION 2 PART 1 Global Pediatric Development Fact or Fiction? Challenges of Building a Global Pediatric Program Donna Helms Takeda Global Research and Development Bryan Boggs, PharmD Regulatory Director With pediatric development legislation now an integral requirement for the US and the EU drug development reviews, companies are struggling to bring a synergistic approach to planning in these separate regions. The goal is to be not only responsive to the regulatory requirements and patient needs, but also to be as efficient and effective as possible in development efforts. Companies will need to have appropriate planning processes and the infrastructure to prepare an integrated, if not harmonized, strategy. True harmonization may not be possible for many reasons, and this session will highlight some of the experiences from companies and provide key insights from the regulator perspective. A panel discussion will provide for an interesting and lively dialogue among the presenters with full audience participation. The Importance of a Global Pediatric Program Ronald Portman, MD Chair, Pediatric Drug Development Committee Group Director, Cardiovascular/Metabolics Bristol-Myers Squibb Case Studies: Part A Speaker Invited Case Studies: Part B Shamim Ruff Executive Director Global Regulatory Affairs Amgen Inc. International Collaborations: FDA Perspective Jean Temeck, MD Lead Medical Officer Office of Therapeutics Office of the Commissioner (OC) FDA International Collaborations: EMA Perspective Dr. Gunter Egger Scientific Administrator European Medicines Agency (EMA), European Union 3:00-3:30 PM REFRESHMENT BREAK 3:30-5:00 PM SESSION 2 PART 2 Global Pediatric Development: Fact or Fiction? Goals of Global Pediatric Development How Should Companies Prepare for Success A. J. Allen, MD, PhD Robert Iannone, MD Merck and Co., Inc. Sharon Olmstead Head, US Regulatory Policy Merck and Co., Inc. Panel Discussion All Session Speakers and Lisa L. Mathis, MD Associate Director Pediatric and Maternal Health Staff (PMHS) Office of New Drugs (OND) 5:00-6:00 PM NETWORKING RECEPTION WEDNESDAY OCTOBER 13, 2010 7:30-8:30 AM REGISTRATION AND CONTINENTAL BREAKFAST 8:30-10:00 AM SESSION 3 Implementation of Pediatric Development Plans: Implementation Challenges Director Neurosciences Medicines Development Center M. Renee Simar, PhD Principal Strategist, Pediatrics INC Research, Inc. The implementation of the pediatric development plans is fraught with potential pitfalls that can undermine the success of a program. This part of the workshop will address the recurrent challenges that pediatric study teams face in areas such as staging appropriate preclinical and pharmacokinetic studies, utilization of innovative pediatric study designs, ethical considerations, pediatric-specific end points, engaging pediatric investigators, and optimized approaches for program development. The format will include an overview of the recurrent challenges in the aforementioned areas, as well as case presentations to illustrate specific remedies utilized by study teams to overcome these challenges and deliver high quality, informative data. Unless otherwise disclosed, DIA acknowledges that the statements made by speakers are their own opinion and not necessarily that of the organization they represent, or that of the Drug Information Association. Speakers and agenda are subject to change without notice. Recording of any DIA tutorial/workshop information in any type of media, is prohibited without prior written consent from DIA.

4 Challenges in Early Planning of Nonclinical Studies in Pediatric Drug Development Programs Gregg D. Cappon, PhD Associate Research Fellow Pfizer Global Research and Development Pediatric Formulation Challenges Joseph Zielinski Chemist, Technical Research and Development Running Traps: Planning for Challenges in Pediatric PK Trials James D. Marshall, MD Medical Director of Research Pedia tric Critical Care Medicine Cook Children s Health Care System, TX 10:00-10:30 AM REFRESHMENT BREAK 10:30 AM-12:00 PM SESSION 3 continued Implementation of Pediatric Development Plans: Implementation Challenges Clinical Development Strategies: Special Considerations for Clinical Trials in Rare and Orphan Pediatric Diseases Charles W. Richard, III, MD, PhD Principal Medical Director Shire Human Genetic Therapies Valid and Reasonable Assessments for Efficacy and Safety Joan A. Conry, MD Professor of Neurology and Pediatrics Children s National Medical Center George Washington University School of Medicine Washington, DC Pediatric Trial Ethics: Practical Issues for Planning Studies Elora Weringer, PhD Adjunct Assistant Professor Temple University of Pharmacy, Philadelphia, PA 12:00-1:30 PM LUNCHEON 1:30-3:15 PM SESSION 4 The Path Forward Chin Koerner, MS Melissa Tassinari, PhD, DABT Senior Staff Fellow, Pediatric and Maternal Health Staff (PMHS) The Future of Pediatric Development Melissa Tassinari, PhD DABT Senior Staff Fellow Pediatric and Maternal Health Staff (PMHS) Pediatric Research Initiatives: A Global View Director, Neurosciences Medicines Development Center The Road to 2012 Chin Koerner Points to Consider in the 2012 Reauthorization Samuel Maldonado, MD, MPH, FAAP Vice President, Pediatric Drug Development Johnson & Johnson 3:15-4:00 PM PANEL DISCUSSION PANELISTS A. J. Allen, MD, PhD Bryan Boggs, PharmD Regulatory Director Dr. Gunter Egger Scientific Administrator European Medicines Agency (EMA), European Union Donna Helms Takeda Global Research and Development Director, Neurosciences Medicines Development Center Janet Jenkins-Showalter Director, FDA Liaison Hoffmann-La Roche, Inc. Samuel Maldonado, MD, MPH, FAAP Vice President, Pediatric Drug Development Johnson & Johnson Lisa L. Mathis, MD Associate Director, Pediatric and Maternal Health Staff (PMHS) Office of New Drugs (OND), M. Renee Simar, PhD Principal Strategist Pediatrics, INC Research, Inc. Melissa Tassinari, PhD, DABT Senior Staff Fellow, Pediatric and Maternal Health Staff (PMHS) Jean Temeck, MD Lead Medical Officer, Office of Therapeutics Office of the Commissioner (OC), FDA Group Director, Regulatory Affairs Hoffmann-La Roche, Inc. 4:00-4:15 PM CLOSING REMARKS 4:15 PM WORKSHOP ADJOURNED

CO-LOCATED WITH Advancing Pediatric Research in the US Special 3-day Pricing. Register on page 5. Maternal and Pediatric Drug Safety Conference October 13-14, 2010 DoubleTree Hotel and Executive Meeting Center Bethesda, MD, USA Join Global Leaders from Industry, Academia, CDC, FDA, EMA, and WHO to Discuss Emerging Topics in Maternal and Pediatric Drug Safety. Maternal and pediatric drug safety are becoming increasingly important areas in the life cycle of medical compounds. Recent regulatory guidelines and scientific findings have emphasized the need to collect, analyze, and report data of the highest quality to health care providers and patients. This conference will bring together international leaders in the fields of maternal and pediatric drug safety to discuss emerging topics in these areas. CONFERENCE GOALS Discuss the pros and cons of various methods for drug safety assessment in pregnancy, lactation, and pediatrics Provide an update on recent regulatory actions in the maternal and pediatric areas Assess the safety and efficacy of vaccines used during pregnancy Recognize the effects of medicinal drug use on breastfeeding Review how different pediatric age groups may pose different safety risks DESIRED OUTCOMES Build consensus on how to assess maternal and pediatric drug safety Identify situations in which the benefits of vaccination during pregnancy outweigh the potential risks Classify situations when a lactation study is needed and how to communicate the results to the public Improved understanding on how to treat the pediatric population CALL FOR POSTER ABSTRACTS DEADLINE: September 14, 2010 For abstract submission guidelines, visit www.diahome.org, keyword 10032 WHO SHOULD ATTEND This program will benefit those involved in Academic health centers Clinical safety and pharmacovigilance Clinical research and development Regulatory affairs Managed health care Medical scientific writing Nonclinical safety and efficacy/toxicology PROGRAM CHAIRPERSON Diego F. Wyszynski, MD, MHS, PhD Global Safety Medical Director and Head Maternal and Pediatric Safety, Amgen Inc. PROGRAM COMMITTEE Elizabeth B. Andrews, PhD, MPH Vice President, Pharmacoepidemiology and Risk Management, RTI Health Solutions Christina Chambers, PhD, MPH Associate Professor, Pediatrics and Family and Preventive Medicine, University of California, San Diego Karen B. Feibus, MD, FACOG Medical Team Leader, Maternal Health Team Office of New Drugs, Karen D. King, MBA Global Safety Manager, Maternal and Pediatric Safety, Amgen Inc. Sonja A. Rasmussen, MD, MS Senior Scientist, Division of Birth Defects and Developmental Disabilities, NCBDDD, CDC Kristine Shields, MSN, MPH Director, Clinical Risk Management and Safety Surveillance, Merck & Co., Inc. Robert M. Ward, MD, FAAP, FCP Director, Pediatric Pharmacology Program, University of Utah

REGISTRATION FORM Register online or fax this page to +1.215.442.6199 Advancing Pediatric Research in the US Event #10027 October 12-13, 2010 DoubleTree Hotel and Executive Meeting Center, Bethesda, MD, USA Registration Fees If DIA cannot verify your membership, you will be charged the nonmember fee. Registration fee includes refreshment breaks, luncheons, and reception (if applicable), and will be accepted by mail, fax, or online. Member Early-bird Opportunity On or before After Available on nondiscount member fee only SEPT. 21, 2010 SEPT. 21, 2010 Member Fee US $1310 US $1460 Join DIA now to qualify for the early-bird member fee! www.diahome.org/membership MEMBERSHIP US $140 To qualify for the early-bird discount, registration form and accompanying payment must be received by the date above. Does not apply to government/academia/nonprofit members. Nonmember Fee US $1600 A one-year membership to DIA is available to those paying a nonmember registration fee. If paying a nonmember fee, please indicate if you do, or do not, want membership. I want to be a DIA member I do NOT want to be a DIA member Discount Fees MEMBER NONMEMBER Government (Full-time) US $580 US $720 Charitable Nonprofit/Academia (Full-time) US $730 US $870 *If paying a nonmember fee, please check one box above, indicating whether you want membership. Co-located Conferences Take advantage of the discounted pricing at a special 3-day registration fee accordingly. Register for this workshop (#10027) and 1 day for the Maternal and Pediatric Drug Safety Symposium (#10032) 3-day Fees MEMBER NONMEMBER Industry US $1810 US $1950 Government (Full-time) US $725 US $865 Charitable Nonprofit/Academia (Full-time) US $925 US $1065 GROUP DISCOUNTS* Register 3 individuals from the same company and receive complimentary registration for a 4th! All 4 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. Group registration is not available online and does not apply to the already-discounted fees for government or charitable nonprofit/academia. To take advantage of this offer, please make a copy of this 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. Please indicate that this form is part of a group registration by checking this box and list below the names of the other three registrants from your company. 1. TRAVEL AND HOTEL The most convenient airport is Ronald Reagan Washington National Airport and attendees should make airline reservations as early as possible. The DoubleTree Hotel and Executive Meeting Center is holding a block of rooms at the reduced rate below until September 20, 2010, for the DIA event attendees. Room availability at this rate is guaranteed only until this date or until the block is filled. Single $169 Double $169 Attendees must make their own hotel reservations. Contact the Double- Tree Hotel and Executive Meeting Center by telephone at +1.240.235.1670 and mention the DIA event. The hotel is located at 8120 Wisconsin Avenue, Bethesda, MD 20814, USA. CANCELLATION POLICY: On or before OCTOBER 5, 2010 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 by the cancellation date above. Registrants who do not cancel by that date and do not attend will be respons ible for the full registration fee paid. Registrants are responsible for cancelling their own hotel and airline reservations. You may transfer your registration to a colleague at any time but membership is not transferable. Please notify DIA of any such substitutions as soon as possible. Substitute registrants will be responsible for nonmember fee, if applic able. 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. Participants with Disabilities: DIA event facilities and overnight accommodations are accessible to persons with disabilities. Services will be made available to sensory-impaired persons attending the event if requested at least 15 days prior to event. Contact the DIA office to indicate your needs. EVENT INFORMATION Contact Constance Burnett, Program Developer, Phone +1.215.293-5800 Fax +1.215.442.6199, email Constance.Burnett@diahome.org Contact Joanne Wallace, Program Manager, Phone +1.215.442.6180 Fax +1.215.442.6199, email Joanne.Wallace@diahome.org Please check the applicable category: Academia Government Industry CSO Student (Call for registration information) Last Name 2. 3. First Name M.I. Payment options: Register online at www.diahome.org or check payment method. CREDIT CARD number may be faxed to: +1.215.442.6199. You may prefer to pay by check or bank transfer since non-u.s. credit card payment will be subject to the currency conversion rate at the time of the charge. Degrees Dr. Mr. Ms. Job Title Visa MC AMEX Exp Date Card # Company Name (printed) Signature Address (As required for postal delivery to your location) Mail Stop CHECK drawn on a US bank payable to and mailed along with this form to: Drug Information Association Inc, P.O. 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