189 UNITED STATES OF AMERICA DEPARTMENT OF HEALTH AND HUMAN SERVICES FOOD AND DRUG ADMINISTRATION + + + CENTER FOR DEVICES AND RADIOLOGICAL HEALTH + + + PEDIATRIC MEDICAL DEVICE DEVELOPMENT + + + August 14, 2018 8:30 a.m. FDA White Oak Campus 10903 New Hampshire Avenue Building 31, Room 1503 (the Great Room) Silver Spring, MD 20993 FDA: VASUM PEIRIS, M.D., M.P.H. Chief Medical Officer, Pediatrics and Special Populations Office of the Center Director Center for Devices and Radiological Health This transcript appears as received from the commercial transcribing service after inclusion of minor corrections to typographical and factual errors recommended by the DFO. Free State Reporting, Inc. 1378 Cape St. Claire Road Annapolis, MD 21409 (410) 974-0947 190 CREATING REGULATORY VALUE AND SIMPLICITY ERIC CHEN Director, Humanitarian Use Device and Pediatric Device Consortium Programs Office of Orphan Products Development FDA JAMES BAUMBERGER Senior Director in Federal Advocacy American Academy of Pediatrics LYNNE YAO, M.D. Director, Division of Pediatric and Maternal Health Center for Drug Evaluation and Research FDA ELIANE SCHUTTE Chief Development Officer, Xeltis DECLAN DINEEN Senior Regulatory Affairs Director Structural Heart Medtronic BOB KROSLOWITZ Chairman, Pediatric Medical Device Working Group AdvaMed CEO, Berlin Heart LEE GRANT Distinguished Regulatory Affairs Advisor Medtronic Spine and Biologics CHESTER KOH, M.D. Co-Founder and Co-PI Southern California Consortium for Technology and Innovation in Pediatrics (CTIP) Free State Reporting, Inc. 1378 Cape St. Claire Road Annapolis, MD 21409 (410) 974-0947 191 DEVELOPING A SUPPORTIVE MARKETPLACE CARA TENENBAUM Senior Policy Advisor Office of the Center Director Center for Devices and Radiological Health FDA GABRIEL MOVSESYAN Staff Fellow Economist Office of Planning FDA SAM MALDONADO, M.D. Vice President of Child Health Innovation and Leadership Department Johnson & Johnson ANDREW LO, Ph.D. Charles E. and Susan T. Harris Professor Director of the Laboratory for Financial Engineering Sloan School of Management Massachusetts Institute of Technology BOB McDONOUGH, M.D. Senior Director, Clinical Policy Research & Development Aetna JAMES KENNEDY WALL, M.D. Assistant Director at Biodesign Innovation Fellowship Program Assistant Professor of Pediatric Surgery Stanford University MARK THRODAHL President & Chief Executive Officer, OrthoPediatrics PEDRO del NIDO, M.D. William E. Ladd Professor of Child Surgery Harvard Medical School Chairman, Department of Cardiovascular Surgery Boston Children's Hospital TIFFANY WILSON CEO, Global Center for Medical Innovation/T3 Labs LIJIE GRACE ZHANG, Ph.D. Associate Professor, George Washington University March of Dimes funding recipient JOHN PARKER Founder and Managing Director, Springhood Ventures Free State Reporting, Inc. 1378 Cape St. Claire Road Annapolis, MD 21409 (410) 974-0947 192 MARK SCHLESINGER, Ph.D. Department Chair and Professor of Public Health (Health Policy) Yale School of Public Health PUBLIC COMMENT PETER ARMSTRONG, M.D. Chief Medical Officer, OrthoPediatrics TARA FEDERICI Vice President, Technology and Regulatory Affairs AdvaMed CLAUDIA HOYEN, M.D. Director of Innovation, Rainbow Babies and Children's Hospital HENRI JUSTINO, M.D. Director, Cardiac Cath Labs Texas Children's Hospital JANETTE STRASBURGER, M.D. Pediatric Cardiology Children's Hospital of Wisconsin-Milwaukee VICTOR GURA, M.D. Cedars Sinai UCLA MATTHEW MALTESE, Ph.D. Director, Biomechanics Research Department of Anesthesiology & Critical Care Medicine Children's Hospital of Philadelphia Free State Reporting, Inc. 1378 Cape St. Claire Road Annapolis, MD 21409 (410) 974-0947 193 ALSO PARTICIPATING BRITTANY GOLDBERG, M.D. Medical Officer Office of Device Evaluation Center for Devices and Radiological Health FDA SKIP NELSON, M.D., Ph.D. Senior Director, Pediatric Drug Development Johnson & Johnson GEORGE PANTALOS, Ph.D. University of Louisville TIMOTHY BALDWIN, Ph.D. NHLBI, NIH JUAN ESPINOZA, M.D. Co-Director, CTIP Children's Hospital of Los Angeles SUSAN ALPERT, M.D., Ph.D. MIKE BILLIG Chief Executive Officer, Experien Group GREG LICHOLAI, M.D., M.B.A. Yale PIETER KAPPETEIN, M.D., Ph.D. Chief Medical Officer/Vice President, Medtronic SUSAN McCUNE, M.D. Director, Office of Pediatric Therapeutics FDA Free State Reporting, Inc. 1378 Cape St. Claire Road Annapolis, MD 21409 (410) 974-0947 194 INDEX PAGE WELCOME AND RECAP - Vasum Peiris, M.D., M.P.H. 197 CREATING REGULATORY VALUE AND SIMPLICITY Audience Poll Question 7 199 Opening: Outline Session Topics and Goals - Eric Chen 199 Overview of Pediatric Medical Device Legislation History - James Baumberger 200 How the Medicines Market Developed for Children: What Can We Learn from Pediatric Drug Development Legislation? - Lynne Yao, M.D. 208 Pediatric Device Development Regulatory Landscape on the International Stage - Eliane Schutte 213 Question and Answer 218 Audience Poll Questions 8 and 9 226 Importance of International Regulation Harmonization, U.S. FDA/Japan Pharmaceutical and Medical Devices Agency Partnership - Declan Dineen 227 Getting a Pediatric Device to Market - Regulatory Challenges and Solutions from the Corporate Experience Small Company Perspective - Bob Kroslowitz 233 Large Company Perspective - Lee Grant 238 Pediatric Device Consortium and its Role in the Pediatric Medical Device Ecosystem - Chester Koh, M.D. 243 Question and Answer 247 Free State Reporting, Inc. 1378 Cape St. Claire Road Annapolis, MD 21409 (410) 974-0947 195 INDEX PAGE PUBLIC COMMENT Peter Armstrong, M.D. 254 Tara Federici 256 Claudia Hoyen, M.D. 257 Henri Justino, M.D. 259 Janette Strasburger, M.D. 264 Victor Gura, M.D. 266 Matthew Maltese, Ph.D. 268 DEVELOPING A SUPPORTIVE MARKETPLACE Audience Poll Question 10 270 Opening: Outline Session Topics and Goals - Cara Tenenbaum 270 The Same but Different - Commonalities and Distinctions Between the Pediatric Drug and Device Markets - Just the Facts - Gabriel Movsesyan 271 Best Pharmaceuticals for Children Act/Pediatric Research Equity Act for Devices? - The Right Incentives for the Right Market - Sam Maldonado, M.D. 277 Financial Strategies/Models for Small and Large Companies: Addressing Economic Challenges - Andrew Lo, Ph.D. 281 How Does Reimbursement Impact Pediatric Device Development? - Bob McDonough, M.D. 286 Question and Answer 290 Audience Poll Questions 11 and 12 300 Free State Reporting, Inc. 1378 Cape St. Claire Road Annapolis, MD 21409 (410) 974-0947 196 INDEX PAGE Medical Device Innovation Models The Many Roads to Successful Early Stage Health Technology Development: Incubators, Accelerators, and Other Ways to Develop Early Stage Ideas - James Kennedy Wall, M.D. 301 Small Company Business Model - Mark Throdahl 305 The Non-profit/Academic Medical Center Model - Pedro del Nido, M.D. 310 Federal-State-Private Partnerships - Tiffany Wilson 315 Philanthropic Model - Lijie Grace Zhang, Ph.D. 320 Venture Capital Perspective - John Parker 324 Dear CEOs, What Will Incentivize You to Enter, Sustain and Innovate in the Pediatric Market? - The C-Suite Survey (CEOs for Children) - Mark Schlesinger, Ph.D. 329 Question and Answer 334 THE NEXT TEN YEARS: STEPS TOWARD A MODERN MEDICAL DEVICE ECOSYSTEM FOR ADULTS AND CHILDREN DIRECTED EXPERT PANEL DISCUSSION 340 CLOSING REMARKS - Vasum Peiris, M.D., M.P.H. 379 Free State Reporting, Inc. 1378 Cape St. Claire Road Annapolis, MD 21409 (410) 974-0947 197 M E E T I N G (8:35 a.m.) DR. PEIRIS: Good morning, everybody. How is everybody doing today? Good. Excited for Day number 2 hopefully? I know there are a few of you that didn't have a chance to join us yesterday, so thank you very much for joining us today. I'll do a quick recap of some of the housekeeping things, just so that everyone that's joining us today, both in person and on the web, will know what the process is. And for those that didn't get a chance to have my introduction yesterday, my name is Vasum. I'm the Chief Medical Officer for Pediatrics and Special Populations with CDRH. So, once again, for anyone that hasn't downloaded CrowdCompass, please go ahead and download CrowdCompass. For people both on the web and in person, you can ask questions through CrowdCompass. The entire schedule is on CrowdCompass, all the speaker bios are on CrowdCompass, so please take a look at that. And, if you use the #Devices4Kids, there is a social page on CrowdCompass also that hopefully will populate. So feel free to use that. And we'll go through some instructions, but if you haven't done it yet, please go ahead and try to do it now because we'd like for you to participate in the audience poll questions that also will be conducted through CrowdCompass. As I mentioned the question, Q&As, and when you do question and answer, work through CrowdCompass, you can just hit the area of the schedule that says Question and Answer, and that'll take you to the option to file a question with us. Even if you're in person and you'd like to submit a question through CrowdCompass, feel free to do that if you don't want to come to the mic. But we definitely want to encourage everyone to come up to the mic. Another piece of housekeeping, and I'll just read this out loud again as I did yesterday, so participation in a public meeting by an individual or organization does not Free State Reporting, Inc. 1378 Cape St. Claire Road Annapolis, MD 21409 (410) 974-0947 198 imply any endorsement by the Food and Drug Administration. As all of you know, we try to maintain a neutral forum here for the exchange of ideas and education. So, I definitely welcome all of the ideas and thoughts that we'll be going through today. Let me highlight the point that if you do have comments to submit to the docket, please feel free to take a look at this website and submit comments as you'd like. If there are any questions or issues that you haven't had answered, you can definitely let our Webex and CrowdCompass team know specifically when you write in the comments to them through the question process.