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Learn More Posters 2020 Summit Registration

Bringing Transformational Therapies to Patients with Rare Diseases During Challenging Times

All times are Eastern Standard Time (EST)

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Thursday, October 8, 2020

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 FROM THE FDA COMMISSIONER

10:00 am -
11:00 am

PLENARY

From Rare Disease to Pandemic: What We've Learned from COVID-19 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.

11:00 am -
11:10 pm

NETWORKING BREAK

11:10 am -
12:00 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.

12:00 pm -
12:30 pm

LUNCH BREAK

12:30 pm -
2:40 pm

BREAKOUT SESSIONS

New Approaches to Advancing Diagnosis

12:30pm to 1:10pm
Update from Global Commission to End Diagnostic Odyssey
Speakers will provide an update on pilot projects launched to date by the Global Commission to End the Diagnostic Odyssey.
1:15pm to 1:55pm
Whole Genome Sequencing and Other Tools to Promote Early Diagnosis
New tools and resources offer hope for undiagnosed patients. This session will explore how these new capabilities may change the landscape for the rare community.
2:00pm to 2:40pm
Advancing Access to Diagnostic Testing
Even if new diagnostic capabilities exist, they can’t help if patients can’t access them. This session will look at the challenges and initiatives to support access.

An Era of Innovation in Research

12:30pm to 1:10pm
Designing a Regulatory-Ready Natural History Study
Panelists will discuss how to design natural history studies for maximum effectiveness in supporting research and regulatory processes
1:15pm to 1:55pm
Latest News on Gene Therapy and Genome Editing Research
Gene therapy and genome editing offer hope to many people living with rare diseases. This panel will provide an update on current research and the outlook for the future.
2:00pm to 2:40pm
Streamlining Orphan Drug Development
With only a small percent of rare diseases currently having approved treatments, this panel will explore possible ways to streamline orphan product development.

A New Era for Patient Organizations

12:30pm to 1:10pm
Moving into a Virtual World
Rare disease patient organizations have been severely affected by this year’s pandemic. This session will look at how to maintain vital communications, advocacy and fundraising functions in a virtual world.
1:15pm to 1:55pm
Designing and Implementing a Strategic Plan
Despite COVID-19, life goes on and patient advocacy organizations need to continue to strategize and plan for maximum effectiveness on behalf of their constituents.
2:00pm to 2:40pm
How Patient Organizations Can Encourage Research on their Disease
In recent years, there have been several highly publicized cases in which rare disease patient organizations played a key role in research leading to significant progress. How can organizations – even very small ones – encourage research on their disease?

2:45 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

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
Patient-reported data is contributing to research and development of treatments in ways that go far beyond what was previously possible. This panel will look at current trends and the future outlook for patient-reported data in rare oncology.
1:30pm to 2:15pm
Current Trends and Future Directions for Precision Oncology
Precision oncology is revolutionizing cancer research, seeking to improve prognosis and outcomes for patients with treatment that is targeted and individualized. This session will explore this new chapter in cancer research.

Investment & Business Development

12:45pm to 1:30pm
Impact of COVID-19 on Orphan Investment
A panel of investors will discuss the impact of COVID-19 on orphan product investment and possible future scenarios over the months to come.
1:30pm to 2:15pm
Advancing Patient Access to Care Through Drug Re-purposing
Is re-purposing of approved products a possible way to advance development of approved therapies for rare diseases that currently have no treatment? This panel will explore that question.

Patient Empowerment

12:45pm to 1:30pm
Diversity and Inclusion in the Rare Community
Inequities based on gender, race and ethnicity continue to limit clinical trial participation and access to quality healthcare. This panel will hear from individuals who have experienced inequity first-hand and will explore various approaches to promote diversity and inclusion.
1:30pm to 2:15pm
Unlocking the Power of Healthcare Data
This panel will discuss today’s increasingly data-driven healthcare environment and some specific initiatives to support research, development of treatments and disease management.

2:15 pm -
3:00 pm

DISCUSSION AND NETWORKING

7:00 pm

Rare Impact Awards

Please register here to join us for the 2020 Rare Impact Awards, a free streaming event celebrating those making extraordinary strides on behalf of the millions of American impacted by rare diseases.