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2020 Summit Registration

2019 NORD Summit Agenda

Day One-October 21, 2019


Conference Registration and Continental Breakfast


NORD Welcome: The Time is Now to Drive Innovation, Collaboration, Advocacy and Research
Peter L. Saltonstall, President & CEO of NORD


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


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.


FDA Deputy Commissioner Keynote


Networking Break


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.


Networking Luncheon
Sponsor booths open and poster presenters available at their posters; reserve your seat when registering.


Choose One of Three Breakout Sessions


Networking Break



NIH Town Hall: A Unique Moment in Time for Rare Disease



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


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.


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.


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


Continental Breakfast


Town Hall: A Conversation with FDA Center Directors
Bring your questions and observations!


Acting Commissioner Keynote
Discussion on Patient-Focused Drug Development, real-world evidence, gene therapy, pediatric drugs and devices, natural history studies and more.


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.


Networking Break


Choose One of Three Breakout Sessions



Update with NORD Policy Team, Networking Lunch or Lunch and Learn Discussions



Choose One of Three Breakout Sessions



Ahead of Its Time: Next-Generation Technology and Innovation in Rare Diseases
Learn how technology is advancing rare disease diagnosis, research and treatment.


Close of Conference


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


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.


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


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.


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.

Key Contacts