Karin Hoelzer’s Post

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Senior Director, Patient Advocacy at BIO

Just about a week to go and counting! I am excited to see everyone in San Diego at #DIA2024. What a treat to discuss "Sustainability of Rare Disease Drug Development" with an esteemed panel of FDA experts including Julie Tierney, Patroula Smpokou, MD, and Katherine Donigan - of course expertly moderated by Darcy Frear, PhD. Current incentives for #raredisease #drugdevelopment including the #orphandrugact #ODA have changed to world for many #raredisease patients and families. We went from fewer than 40 FDA approved therapies in 1983 before the #ODA to #orphandrugs consistently making up more than 50% of new drug approvals (#NMEs) at #FDA. But more than 95% of rare diseases still lack any FDA-approved therapies. So how do we sustainably improve rare disease drug development and bring life-changing therapies to those communities that have for too long been left behind? There are so many promising approaches, pilots, and policy priorities to make this dream a reality, and I can't wait to discuss them with this esteemed panel of experts!

Mike Zhou M.D.

CEO at ACA Pharma | New Drug Macao 30-90 Day Registration for Asia Access

7mo

I missed your session at BIO2024, but your summary was excellent. Your dedication and hard work in the rare disease field are truly commendable. Your efforts inspire us all to continue working towards our mission of helping children with rare diseases worldwide receive the treatment they need.

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Carie Pierce

Business Development Executive | Corporate Strategy | Public Relations | Global Marketing

6mo

Thanks for your participation

Darcy Frear, PhD

Regulatory Policy at BridgeBio

7mo

I'm excited to moderate this session next week!

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