Archived Events

2024 RARE Drug Development Symposium

Global Genes and the Orphan Disease Center of the University of Pennsylvania hosted the 8th Annual RARE Drug Development Symposium April 29-May 1, 2024.

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Innovative Ideas from Next Generation Change-Makers

The RARE Drug Development Symposium, hosted by Global Genes and the Orphan Disease Center of the University of Pennsylvania, equips advocates with the knowledge, skills and connections they need to advance therapy development for their communities.

Keep scrolling to see more content from the 2024 event, including videos of the sessions, posters, and the RARE Research roadmap.

Sessions & Key Takeaways from the 2024 RARE Drug Development Symposium

Welcome and Opening Keynote: Shifting the Paradigm to Push Past Limits

Advocates are dramatically altering the landscape of rare research and reducing the timeline for rare disease therapy development. What is it that allows some organizations to move faster, be more nimble, use resources effectively and blaze new paths? Is there a matrix that can help you determine what will work for you?

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The Critical Need for Patient-Led Data Initiatives: Does Size Matter?

Researchers and regulators need data. But what kind? Who is the end user? What is “enough data?” Do you need certain types of data for specific research projects? Why? Most importantly how do you evaluate your data set to be certain it’s useful?

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Rethinking Clinical Trials: What’s Doable? What’s Approvable?

Researchers are overcoming barriers posed by small populations using decentralized and basket trials, platform science, wearables, videos, natural history data and engaging diverse patients. But what does FDA guidance say? How can patient communities prepare by ensuring their data is well-structured?

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Day 2 Welcome

Charlene Son Rigby, CEO of Global Genes, gives a recap of the first day and gives an introduction of what to expect for Day 2.

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What will $100K Buy You? Emerging Commercial & Non-Profit Financing Models

You’ve raised some money and have a strategy (or are about to develop one). The next question is, “What will your funds realistically buy for your organization?” Are there creative ways to make the most of what you have? How do you choose partners and negotiate?

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Closing Keynote and Remarks

What new solutions are emerging to accelerate translational research in rare disease? What can patients, caregivers, researchers and organizations do to help move the needle? Dominique Pichard talks about her unique experience as a physician, rare Mom and advocate has informed her approach to leading innovation in rare disease research at NCATS.

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RARE-X Exchange Sessions

These sessions were available to any representatives of patient advocacy groups
participating in RARE-X data collection programs

Partnerships in Action

This session was moderated by
Charlene Son Rigby, Global Genes CEO
Panelists included Emily Bonkowski, Ph.D. Candidate, Genetic Counselor & Research Program Coordinator at St. Jude Children’s Research Hospital; Alexandra Gillett, Ph.D., Board Member, Wiedemann-Steiner Syndrome Foundation; and Jeff De’Angelo, President & Founder, CHAMP1 Foundation

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Unlocking Insights Together: The Power of Collaboration

This session was moderated by Meagan Perry, Head of Global Public Affairs, Sanofi. Panelists included Chandler Crews, Patient Advocate; Founder of The Chandler Project; Karmen Trzupek
Senior Director, Scientific Programs for Global Genes; and Maddie Crowley,
Pompe Advocate.

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Integrating Clinical & Patient Reported Data

This session was moderated by Zohreh Talebizadeh, Ph.D., Sr Director, RARE-X Research Program. Panelists included Scott Demarest, M.D., Pediatrics & Neurology for Children’s Hospital Colorado; Yssa DeWoody, Ph.D., RING14 USA; Vanessa Vogel-Farley, Sr Director, Research & Data Analytics; and Kelly Wentworth, Associate Manager, RARE-X Research Program.

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2024 RARE Drug Development Symposium Posters

Coming Soon!


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Looking Back at the 2024 RARE Drug Development Symposium

2024 Recap

Check out some stats from our event this year.

2023 RARE Drug Development Symposium Stats