2019 Summit Agenda - NORD (National Organization for Rare Disorders)

Day One-October 21, 2019

7:30	Conference Registration and Continental Breakfast
8:15	NORD Welcome: The Time is Now to Drive Innovation, Collaboration, Advocacy and Research Peter L. Saltonstall, President & CEO of NORD
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® Karen Pignet-Aiach, Founder, Chief Executive Officer and Board of Directors Chairperson, Lysogene; mother of a child with MPS IIIA
9:00	The Time is Now: Addressing Affordability While Sustaining Innovation Cutting-edge solutions that address affordability while sustaining innovation to make drugs and other therapies affordable without compromising innovation and effectiveness will be examined.
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 Sponsor booths open and poster presenters available at their posters; reserve your seat when registering.
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 Research
5:30	Cocktail Reception, sponsored by Orchard Therapeutics All attendees are invited to join NORD immediately following the first day of the Summit for cocktails and fun at our reception. Fun collaboration is sure to be had by all with special thanks to Orchard Therapeutics.

Day One Breakout Sessions

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.

Day Two-October 22, 2019

7:15	Continental Breakfast
8:00	Town Hall: A Conversation with FDA Center Directors Bring your questions and observations!
9:00	Acting Commissioner Keynote Discussion on Patient-Focused Drug Development, real-world evidence, gene therapy, pediatric drugs and devices, natural history studies and more.
9:45	Interactive Dialogue with FDA Senior Staff Discussion on Patient-Focused Drug Development, real-world evidence, gene therapy, pediatric drugs and devices, natural history studies and more.
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 Learn how technology is advancing rare disease diagnosis, research and treatment.
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.

Day Two Breakthrough Sessions at 11:20 am

I	Rare Disease Advisory Councils: Driving Progress with a Unified Voice Rare Disease Advisory Councils have been implemented in many states to advise legislators on issues related to access and coverage for rare disease patients. Hear about their impact to date and future focus.

Patient Registries and Natural History Studies: Impact, Data Ownership and Ethical Issues
As more organizations develop patient registries and natural history studies, they are faced with ethical and legal consideration around data and data ownership. This session is designed to address these challenges, the ramifications and the solutions being proposed.

III	Maximizing Access to Newborn Screening, Carrier Screening and Genetic/Diagnostic Testing Exome/genome sequencing, carrier screening and other diagnostic capabilities offer great promise. Investigate why many patients still don’t have access to them.

Day Two Breakthrough Sessions at 2:00 pm

IV	Advancing Curative Therapies for Rare Cancers Explore advances in precision oncology, personalized combination therapies, cell and gene therapies and other breakthrough areas within the rare cancer space.

V	Ahead to 2020: Investment Opportunities in Orphan Products and Rare Disease Therapies Investors reveal anticipated trends and make predictions in orphan product development.

Fostering Dialogue and Developing Partnerships Among Payers, Providers and Patients
Providers and payers often have challenges getting what they need from each other while the patient is left frustrated. This session puts communication barriers under a microscope and gives novel examples of partnerships that exist among these three groups.

2019 NORD Summit Agenda

Day One-October 21, 2019

Day One Breakout Sessions

Day Two-October 22, 2019

Day Two Breakthrough Sessions at 11:20 am

Day Two Breakthrough Sessions at 2:00 pm

Key Contacts

Alone we are rare. Together we are strong.®