Charting the Path to Treatments – 2023 RARE Drug Development Symposium

May 10, 2023

In May 2023, Global Genes and the Orphan Disease Center of the University of Pennsylvania hosted the RARE Drug Development Symposium. Global Genes CEO Charlene Son Rigby led the discussion about how the path to development of therapeutics for rare diseases may differ.

Each path toward development of rare disease therapeutics is different. In this plenary, you’ll hear from advocates who took different paths. Use their insights to get closer to approved treatments and therapeutics for your community.

Notable quotes from this session:

“My job is the master-connector. I’m really good at finding and building relationships. I’ve heard a lot of people talking about funding academics as useless. It’s not – without the science that they research, we wouldn’t be where we are today.” – Leah Schust Myers

Note: The academic work that her org funded led directly to a pharma getting a gene therapy approved.

“Apply for the big grants, fundraise from bigger foundations, and do grassroots fundraising through events and awareness campaigns.” – Simon Frost

“Hire an ED early instead of limiting your advocacy group to volunteer roles. Hiring an ED will lead to more fundraising and the role will pay for itself.” – Leah Schust Myers

“Build a skills matrix for your board of directors. Hire for your weakness, not your strength. Cycle through board members that don’t meet what you have on staff already.” – Brett Kopelan

Key Takeaways:

  • Parents are the catalyst. They have the urgency and drive to find treatments and can support industry by communicating what they’re experiencing to the FDA
  • Understand the science by collaborating with academic researchers, identify endpoints acceptable to FDA, and attract industry by knowing what they’re looking for.
  • The path isn’t linear and is different for each community, based on what’s known about the disease, availability of tools like registries and models, the skills you recruit for, etc. 

Charlene Son Rigby
CEO, Global Genes


Simon Frost
CEO, Tiber Capital Group

Brett Kopelan
Executive Director, debra of America

Leah Schust Myers
Founder and Executive Director, FamilieSCN2A Foundation

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