| 3:30 |
Summit Chair’s Welcome Richard Moscicki, M.D. Deputy Center Director for Science Operations, CDER, FDA |
| 3:45 |
Current State of Expanded Access Richard Klein Patient Liaison Program Director, Office of Health and Constituent Affairs (OHCA), FDA |
| 4:15 |
Ethical Issues Surrounding Expanded Access Including How Drugs are Made Available Kevin A. Prohaska, D.O., M.P.H., Captain (USPHS), Medical Officer Office of Good Clinical Practice, FDA |
| 4:35 |
Impact of Drug Importation Paras M. Patel, R.Ph., M.B.A., CDR U.S. Public Health Service , CDER Drug Shortage Staff/ Office of the Center Director, Senior Regulatory Review Officer, FDA
|
| 5:05 |
Patient Experience in Expanded Access Ron Bartek President and Co-Founder, Friedreich’s Ataxia Research Alliance (FARA) NORD, Board of Directors |
| 5:25 |
Expanded Access in Oncology Pediatrics Meredith Chuk Medical Officer, Office of Hematology and Oncology Products, CDER, FDA |
| 5:45 |
Close of Track I |
| I |
Expanded Access |
| II |
Facilitate Early Diagnosis and Treatment |
| III |
Be Visible, Be Active, Be Heard |
| IV |
| V |
| VI |
| 3:30 |
Summit Chair’s Welcome Marshall L. Summar, M.D. Division Chief, Genetics and Metabolism, Children’s National Heatlh System, NORD Board of Directors |
| 3:35 |
Newborn Screening-Advanced Capabilities and Ethical Considerations Tim Boyd Associate Director of State Policy, NORD Mike S. Watson, Ph.D., FACMG Executive Director, ACMG
|
| 4:20 |
Rare Cancer Treatment Challenges Jim Palma Executive Director, TargetCancer Foundation Mary Smith Vice President, Public Development, United Therapeutics |
| 5:05 |
Optimizing Chances of Clinical Trial Success-Patient Engagement from Start to Finish Jeanne Hecht, MBA, PMP Senior Vice President, Global Head of Site and Patient Networks, Quintiles Dan Bobear Principal-Managing Director, The Patient Experience Project Pamela Gavin Chief Operating Officer, NORD Bray Patrick-Lake, MFS Director of Stakeholder Engagement, Clinical Trials Trasformation Initiative Tina Schlecht, PharmD, MBA President, Center Point Clinical Services |
| 5:45 |
Close of Track II |
| 3:30 |
Summit Chair’s Welcome Bert Bruce Vice President of Commercial Development Rare Disease, Pfizer |
| 3:45 |
The Rebrand Effect: Accelerating Fundraising Through Communications Lisa Phelps, MPH Director of Marketing and Community Relations, NORD Farra Trompeter Vice President, Big Duck Will Nolan Director of Communications and Administration, Parent Project Muscular Dystrophy |
| 4:30 |
Navigating the Social Media Landscape Leigh Fazzina Social Media Counselor, Sam Brown Health Communications Berna Diehl Senior Vice President, JPA Health Communications Lauren Smith Dyer Director of Communications, Melanoma Research Foundation |
