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1:00pm - 7:00pm
Save time Monday morning by picking up your badge early and joining us for a welcome reception.
5:30pm - 7:00pm
7:30am
Visit the Poster Hall and Exhibit Hall
8:20am
8:30am - 9:15am
Patients and caregivers share stories that illustrate the life-changing impact of the Orphan Drug Act, the importance of leaving no one behind, and the unique relevance of medical devices for those living with rare diseases.
9:15am - 10:15am
Industry leaders share their thoughts on the current environment and outlook for the future in rare diseases and orphan products.
10:15am - 10:45am
Visit the Poster Hall and Exhibit Hall
10:50am - 11:40am
Experts on pricing, access and public policy discuss how the IRA and other current policy initiatives may affect orphan product development and patient access to care.
11:45am - 12:00pm
12:05pm - 1:30pm
1:35pm - 2:25pm
Inequities in clinical research and healthcare have been well-documented. How do we make the leap to truly effective action?
2:30pm - 3:20pm
Genome editing is emerging as a promising treatment approach for rare diseases. A panel of experts will discuss the current state of the science of these technologies and the regulatory framework of the approval process as we move into this new therapeutic area.
3:20pm - 3:30pm
Visit the Poster Hall and Exhibit Hall
3:35pm - 4:25pm
Artificial intelligence and other innovative use of data show promise for greatly reducing the diagnostic odyssey and supporting more personalized and effective care.
4:30pm - 5:00pm
Authors of the top 5 selected poster abstracts will share their key findings.
5:00pm - 6:00pm
7:30am
Visit the Poster Hall and Exhibit Hall
8:00am - 8:20am
8:30am
Track Co-Chairs: PAG and Industry Representatives
How can patient advocacy groups lay the groundwork to encourage research on their diseases?
Panelists will discuss how digital health, in-home trials and other non-traditional approaches are changing clinical trials.
Repurposing FDA-approved drugs for new indications could save time and reduce the cost of developing drugs for rare diseases. Panelists will talk about current work related to this and the outlook for more widespread application.
8:30am
Track Co-Chairs: FDA Representatives
Panelists will discuss communication strategies among stakeholders to advance rare disease product development.
Panelists from CBER, CDER, CDRH and OOPD will share wide-ranging insights on lessons learned over the years and best practices.
Panelists will discuss the unique aspects of pediatric medical product development, recent product approvals and the path forward.
12:00pm - 1:25pm
1:30pm
Track Co-Chairs: NORD Rare Disease Centers of Excellence & Industry Representatives
Our panel of mental health professionals, industry partners, researchers, and rare parents will discuss the mental health needs of our rare patients and families – and examples of programs sponsored by rare disease advocacy organizations and industry partners to meet these unique needs.
Collaboration among the nation’s leading rare disease research and treatment centers can reduce health inequities, improve diagnosis and care of rare disease patients, and foster robust research. The NORD Rare Disease Centers of Excellence Program is the first national network of U.S. hospitals and medical institutions dedicated to working collaboratively to solve the medical challenges and address unmet needs of the rare disease community. This session will highlight some of the outcomes of the Program since its inception almost two years ago.
1:30pm
Track Co-Chairs: Industry Representatives
Moderator David Scheer and a panel of investors will explore the outlook for orphan product investment over the near future and the next several years.
Initially perceived as transformational treatments, cell and gene therapies are now viewed by some through a less enthusiastic lens because of issues related to cost, access and duration of benefit. Is the original scenario still possible?
3:40pm - 4:30pm