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 - 11:00 am
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
11:10 am - 12:00 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.
12:00 pm - 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 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
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 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.
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
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.