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Day One-October 15, 2018

7:00

Conference Registration and Continental Breakfast

7:50

NORD’s Welcome & Summit Preview
Peter L. Saltonstall, President & CEO of NORD

8:00

Patient/Caregiver Opening Address

8:45

How Patients Are Helping Drive Research and Drug Development
The role of patients, caregivers and patient organizations in research and drug development has undergone fundamental changes in recent years. This panel will look at innovative collaborations and how these new patient roles are helping to advance research and development of therapies.

9:30

Solving the Diagnosis Challenge
Delay in diagnosis remains a serious problem for all stakeholders in the rare disease community. What is the outlook for reducing time to diagnosis and what are the specific challenges – scientific and financial – that need to be overcome?

10:15

Networking and Refreshment Break

10:45

Keynote Address: Scott Gottlieb, M.D., FDA Commissioner

 

11:30

The Growing Impact of Grassroots Advocacy
Decisions made at the state level are having an increasing impact on rare disease patients and families. Representatives of NORD’s Rare Action Network will talk about their experiences and the challenges and opportunities of advocacy at the state level today.

12:15

Networking Lunch or Lunch & Learn Roundtable Discussions

 

 

1:30

Choose One of Three Breakout Sessions
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3:00

Networking & Refreshment Break

 

3:30

Value, Pricing and the Patient Experience: How to Achieve an Effective Balance
With gene therapies and other treatment advances, the conversation regarding value and pricing has taken on new significance. This panel will examine how to achieve a balance in orphan product pricing that reflects the value of therapies in the lives of patients.

 

4:30

What We All Can Learn from Advances in Cancer Therapies
More than 40% of orphan drug approvals are for rare types of cancer. How can this success and the principles adopted by the oncology community be more broadly applied to other diseases?

5:30

Close of Day One-Networking Reception

Day One Breakout Sessions

A

What’s Next for the Rare Cancer Community?

B

Addressing Access Challenges for Orphan Products

C

Patient Advocacy for Diagnosis & Drug Development

Day Two-October 16, 2018

7:00

Continental Breakfast Opens

7:45

Day 1 Summary and Day 2 Preview

8:00

Update from HHS

8:30

Patient Perspectives on Gene Therapy
With more than 150 gene therapy Phase I, II and III clinical trials in progress, patients and caregivers representing several rare disease communities share their thoughts, hopes and concerns regarding this promising new avenue to treatment.

9:00

Choose One of Three Breakout Sessions
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10:00

Networking and Refreshment Break

 

10:30

Current Topics from the FDA
Senior FDA staff will present an overview of current priorities, with particular emphasis on Patient-Focused Drug Development. This session will include FDA Leadership Perspective and FDA Patient Voice Perspective segments.

 

12:30

Lunch & Learn Roundtables

2:00

Federal Policy Priorities & Election Implications
Members of NORD’s federal policy team and advocacy partners will discuss current issues and policy priorities for the community. They will also talk about possible scenarios following the November elections.

3:00

What’s Ahead for Orphan Drug Development?
In a fireside chat format, orphan drug investors and corporate CEOs will talk about gene therapy, personalized medicine, the impact of mergers & acquisitions, scientific innovation, and other topics that will play a role in emerging directions for orphan product development.

4:00

Predicting the Pipeline
The division heads of FDA CBER, CDER AND CDRH share their insights and predictions for orphan product trends, opportunities and challenges over the coming months.

5:00

Close of Conference

6:00

Music, Medicine and Moving Forward
A cocktail reception hosted by Children’s National Rare Disease Institute to celebrate the NORD Rare Summit

 

Day Two Breakthrough Sessions

I

Prior Authorization, Step Therapy and Other Access Challenges

II

Clinical Trial Success: Patient Access, Recruitment, & Retention

III

Embracing Innovation to Advance Science

Key Contacts

General Inquiries
events@rarediseases.org

Sponsorship Inquiries
Derek Gavin
dgavin@rarediseases.org