Bringing Transformational Therapies to Patients with Rare Diseases During Challenging Times
Thursday, October 8, 2020
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
10:10 am - 10:50 am
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
11:10 am - 11:55 pm
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
12:30 pm - 2:35 pm
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
3:00 pm - 3:45 pm
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
DAY 1 SUMMARY & DAY 2 PREVIEW:
Peter L. Saltonstall - President and Chief Executive Officer of NORD
9:00 am - 9:45 am
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
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
10:45 am - 11:30 am
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
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
12:45 pm - 2:15 pm
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
12:45pm to 1:30pm Diversity and Inclusion in the Rare Community 1:30pm to 2:15pm Unlocking the Power of Healthcare Data