Discussion Group Topics: *Space is limited, Advanced registration required.
*session offered on both days, please register for one
DEI: From Theory to Practice
Dive further into the results of the NORD RDDC Diversity Survey and Hispanohablantes Listening Sessions to drive DEI into action within your organization
Telling your Story to Advance Awareness
Storytelling provides connection, education and awareness. Learn how to effectively tell your story to support advocacy efforts and promote understanding and awareness.
Partnering for Progress *
A strong relationship between patient advocacy groups and industry is critical to patient centric rare disease drug development. Explore ways to build and strengthen this partnership while maintaining good ethical guidelines.
How Patient Data is Helping Drive Research Innovation: RDCA-DAP
Discuss the role of data in rare disease research with a case study example of how FARA has leveraged the Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP)’s data sharing and data-analysis capabilities to accelerate the development of treatments for FA.
Reimagining Pediatric Medical Product Development
Developing a product for pediatric use comes with significant scientific, clinical and ethical challenges. Continue the discussion on how we can reimagine pediatric drug development to advance treatments and cures for children with rare diseases.
Selecting Clinical Trial Endpoints *
Join this facilitated discussion on the challenges and opportunities surrounding the selection of clinical trial endpoints and resources available for support.
Examining the Role of AI in Rare R&D
Continue the discussion on how advances in AI and Digital Health are transforming rare disease drug development and care.
Newborn Screening Considerations
This group will discuss the basics of newborn screening and how decisions are made regarding which diseases are included.