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Learn More 2020 Summit Registration

Bringing Transformational Therapies to Patients with Rare Diseases During Challenging Times

Thursday, October 8, 2020

8:50 am

NORD’S WELCOME & SUMMIT PREVIEW

Peter L. Saltonstall, President and Chief Executive Officer, NORD

9:00 am -
9:30 am

PATIENT/CAREGIVER OPENING ADDRESS

Innovation Transforms Lives
A panel of patients and caregivers will describe how their lives have been changed by medical
capabilities that didn’t exist until recently.

9:30 am -
10:10 am

KEYNOTE ADDRESS

10:10 am -
10:50 am

PLENARY

From Rare Disease to Pandemic: What We’ve Learned and How We Can Apply It
Public health experts, patient advocates, CEOs and medical directors will discuss how to build a stronger healthcare system from what we’ve learned this year.

10:55 am -
11:10 pm

NETWORKING BREAK

11:10 am -
11:55 pm

POLICY PLENARY

Perspectives From The Hill: The Year to Date and What’s Ahead
NORD’s Vice President for Policy & Regulatory Affairs will moderate a panel of Capitol Hill staff members discussing policy developments during 2020 and looking ahead to possible post-election scenarios.

11:55 am -
12:30 pm

LUNCH BREAK

12:30 pm -
2:35 pm

BREAKOUT SESSIONS

New Approaches to Advancing Diagnosis

12:30pm to 1:10pm
Update from Global Commission to End Diagnostic Odyssey
1:15pm to 1:50pm
Whole Genome Sequencing and Other Tools to Promote Early Diagnosis
1:55pm to 2:30pm
Advancing Access to Diagnostic Testing

An Era of Innovation in Research

12:30pm to 1:10pm
Designing a Regulatory-Ready Natural History Study
1:15pm to 1:50pm
Latest News on Gene Therapy and Genome Editing Research
1:55pm to 2:30pm
Streamlining Orphan Drug Development

A New Era for Patient Organizations

12:30pm to 1:10pm
How Patient Organizations Can Encourage Research on their Disease
1:15pm to 1:50pm
Designing and Implementing a Strategic Plan in the Wake of a Pandemic
1:55pm to 2:30pm
Moving into a Virtual World: Conferences, Fundraisers & Grassroots Advocacy

2:35 pm -
3:00 pm

NETWORKING BREAK

3:00 pm -
3:45 pm

PLENARY

International Perspectives on Access, Affordability and Pricing
A panel of global patient advocates, industry CEOs, and finance/policy experts will discuss challenges and opportunities related to creating sustainable healthcare for all

Friday, October 9, 2020

8:50 am

DAY 1 SUMMARY & DAY 2 PREVIEW:

Peter L. Saltonstall - President and Chief Executive Officer of NORD

9:00 am -
9:45 am

PLENARY

Telehealth and Rare Diseases
Panelists from patient organizations, industry and academic health centers and government will discuss the current status and particular relevance of telehealth to those living with rare diseases.

9:45 am -
10:30 am

NIH PLENARY

Changing the Clinical Development Paradigm
This panel of NIH senior staff will explore new frontiers in research and how to advance the development of treatments by moving beyond one-disease-at-a-time.

10:30 am -
10:45 am

NETWORKING BREAK

10:45 am -
11:30 am

FDA PLENARY

A New Era for Regulatory Science
A panel of FDA speakers will discuss how the landscape has changed this year; there will be time for audience Q & A.

11:30 am -
12:15 pm

PLENARY

A Conversation with FDA Center Directors
NORD’s CEO will moderate this interactive session with the directors of FDA centers, which will include audience Q & A.

12:15 pm -
12:45 pm

LUNCH BREAK

12:45 pm -
2:15 pm

BREAKOUT SESSIONS

Innovative Approaches to Rare Cancer Therapies

12:45pm to 1:30pm
Increasing Application of Patient-Reported Data in Rare Oncology
1:30pm to 2:15pm
Current Trends & Future Directions for Precision Oncology

Investment & Business Development

12:45pm to 1:30pm
Impact of COVID-19 on Investment
1:30pm to 2:15pm
Advancing Patient Access to Care Through Drug Repurposing

Patient Empowerment

12:45pm to 1:30pm
Diversity and Inclusion in the Rare Community
1:30pm to 2:15pm
Unlocking the Power of Healthcare Data

2:15 pm -
3:00 pm

DISCUSSION AND NETWORKING