| 7:30 | Conference Registration and Continental Breakfast |
| 8:15 | NORD Welcome: The Time is Now to Drive Innovation, Collaboration, Advocacy and Research |
| 8:25 | Patient/Caregiver Opening Address: There is No Time Like the Present – Inspiration to Action Terry Jo V. Bichell, MPH, PhD, Tennesse Rare Action Network® Ambassador, NORD® |
| 9:00 | The Time is Now: Addressing Affordability While Sustaining Innovation
|
| 10:20 | FDA Deputy Commissioner Keynote |
| 11:05 | Networking Break |
| 11:55 | Creating a Roadmap for Collaboration Between Patient Organizations and Industry Patients benefit when industry, patient organizations, government and academic researchers work together. Models for innovative and ethical partnerships will be presented. |
| 1:00 | Networking Luncheon |
| 2:40 | Choose One of Three Breakout Sessions |
| 3:40 | Networking Break
|
| 4:30 | NIH Town Hall: A Unique Moment in Time for Rare Disease
|
| 5:30 | Cocktail Reception, sponsored by Orchard Therapeutics |
| A | Gene Therapy: Overcoming Challenges for Both Patients and Manufacturers With so much hope invested in gene therapy, this panel explores best practices for clinical trial design, inclusion criteria, ethical concerns and patient identity issues. |
| B | Social Media and Technology Platforms for Organizing Patients Potential, pitfalls and privacy — is technology a darling or a danger to rare disease organizing? Discuss options for building and convening networks and the pros and cons of using social media as that platform. |
| C | Social Determinants of Health in Rare Diseases We will explore current inequities and bias in access to diagnosis and care and how they are being addressed. |
| 7:15 | Continental Breakfast |
| 8:00 | Town Hall: A Conversation with FDA Center Directors |
| 9:00 | Acting Commissioner Keynote |
| 9:45 | Interactive Dialogue with FDA Senior Staff |
| 10:45 | Networking Break |
| 11:40 | Choose One of Three Breakout Sessions
|
| 12:35 | Update with NORD Policy Team, Networking Lunch or Lunch and Learn Discussions
|
| 2:00 | Choose One of Three Breakout Sessions
|
| 3:10 | Ahead of Its Time: Next-Generation Technology and Innovation in Rare Diseases |
| 4:15 | Close of Conference |
| 6:15 | Cocktails and Conversation with Children’s National Rare Disease Institute Summit attendees are invited to join the Children’s National team immediately following the conclusion of the NORD Summit for cocktails and conversation. Marshall Summar, MD, and members of the Children’s National Rare Disease Institute will be there to celebrate the Summit and the work going on in the Institute. Register here.
|
| I | Rare Disease Advisory Councils: Driving Progress with a Unified Voice |
| II | Patient Registries and Natural History Studies: Impact, Data Ownership and Ethical Issues |
| III | Maximizing Access to Newborn Screening, Carrier Screening and Genetic/Diagnostic Testing |
| IV | Advancing Curative Therapies for Rare Cancers |
| V | Ahead to 2020: Investment Opportunities in Orphan Products and Rare Disease Therapies |
| VI | Fostering Dialogue and Developing Partnerships Among Payers, Providers and Patients |
