09/11/2025
Today, RDLA hosted the latest Rare Disease Congressional Caucus Briefing. The briefing focused on regulatory urgency and therapeutic opportunity in the drug approval process. Our amazing guest speakers came from different areas of the rare disease community to share their testimony. Here's what they had to say:
"The rare disease community has been advocating for greater predictability, consistency, and transparency." - James Valentine, an FDA lawyer and Director at Hyman, Phelps & McNamara, P.C.
"We must advance regulatory consideration and patient testimonies to be beyond anecdotes, but rather to be included as critical data in the compressive and thorough review of new drug applications." - Emily Milligan, Executive Director at the Barth Syndrome Foundation
"Delayed approvals cost the government more money. We need Congress to ensure the FDA has resources, incentives, and structures to serve our children. " - Jessica Haywood, a board member at the Cure Sanfilippo Foundation
"As programs are being paused muscles are wasted and families are losing hope. With your action, Congress can make a difference." - Kelly Brazzo, the Co-Founder/CEO of the Curelgmd2i Foundation
"Our rare disease community wants, needs, and deserves safe and effective therapies as quickly as possible. We don't have time to wait; we need therapies for this generation of patients." - Annie Kennedy, the Chief of Policy, Advocacy, and Patient Engagement at the EveryLife Foundation for Rare Diseases
We want to express our gratitude to these patient advocacy champions for their heartfelt testimonies and stories.
In case you missed it earlier today, you can watch the full briefing on YouTube ➡️ https://youtube.com/live/touiS3iV-yk
