Rare Disease Legislative Advocates

12/17/2025

Looking back, looking ahead!

Join us on Thursday, December 18, at 2pm ET for the latest RDLA State Advocacy Webinar. The December State Advocacy Webinar will recap crucial state-level rare disease legislative updates and provide insights into the legislative landscape in 2026. Four expert speakers will join us for this conversation. Each speaker will cover one aspect of this past year’s legislative accomplishments and next year’s objectives.

Erin Fitzpatrick, Manager of State Issues at Stateside Associates, will recap developments from 2025 and look ahead towards 2026. Jamie Sullivan, Vice President of Policy at the EveryLife Foundation, will discuss the Medicaid cuts and what HR1 means for the rare disease community. Lauren Edwards, Director of State Government Affairs at Blood Cancers United, will examine medical debt legislation at the state level. Finally, Hilary Gee Goeckner, Director of State & Local Campaigns at the American Cancer Society Cancer Action Network, will review Biomarker legislation at the state level.

Please register for the webinar using the link below. We look forward to seeing all of you there!

https://hubs.li/Q03Ytrsv0

12/10/2025

Rare Disease Legislative Advocates would like to give a warm welcome to our newest RDLA Advisory Committee members! These extraordinary advocates will contribute to RDLA’s mission of changing public policy through a patient-centered approach.

The RDLA Advisory Committee is comprised of rare disease advocates committed to ensuring that everyone in the rare disease community has a voice and can make an impact on legislation and policy. The committee now includes 18 community members and the Senior Director of Advocacy.

The following are the new RDLA Advisory Committee members:

-Mark Konietzko
-Carl Barnes
-Marion Jones
-Sky Collins
-Tatiana Lopez
-Dexter Sherrell
-Ann Ciombor
-Susan Alger
-Amanda Van Eps

Meet the members and learn more about their backgrounds here: https://hubs.li/Q03XGhdF0

12/05/2025

Yesterday, RDLA and the Rare Disease Congressional Caucus came together for the latest Rare Disease Congressional Caucus Briefing!

The briefing focused on the Prescription Drug User Fee Amendment's (PDUFA's) function and effectiveness, as we prepare for PDUFA VIII. Our amazing moderator, E. Cartier Esham, Ph.D., Executive Director of the Alliance For A Stronger FDA, and guest speakers came from different areas of the rare disease community to share their testimony.

Here are a few highlights of what they said:
🎤 "We knew that PDUFA was working because in the first years after it passed, we saw an influx in products being developed and approved in the U.S. And there are people that I know and love that are alive today because of the infrastructure that has come from PDUFA." - Annie Kennedy, the Chief of Policy, Advocacy, and Patient Engagement at the EveryLife Foundation for Rare Diseases

🎤 "Transparency, continuity of knowledge, and structured engagement. These are the strongest levers for accelerating safe and effective rare disease therapies." - Miro Pastrnak, PhD, Chief Strategy Officer at Rare Disease Research, LLC

🎤 "Congress has the opportunity to ensure that rare disease development is fully supported, and families benefit from that innovation without unnecessary delay." - Shion C., Senior Director of Policy & Regulatory Affairs, National Health Council

We want to express our gratitude to these patient advocacy champions for their heartfelt testimonies and expertise.

In case you missed it earlier today, you can watch the full briefing on YouTube ➡️https://www.youtube.com/live/57jrErLZNd0

12/02/2025

Rare disease advocates, you are invited!

Join us this Thursday, December 4, at 12 pm ET for the latest Rare Disease Congressional Caucus. This session will focus on how the Prescription Drug User Fee Act (PDUFA) can secure progress for rare disease drug development.

The caucus features four impactful leaders in the rare disease community. Cartier Esham, Ph.D. is the Executive Director of the Alliance for a Stronger FDA, and she will moderate the conversation. Annie Kennedy is the Vice President of Policy, Advocacy, and Patient Engagement at the EveryLife Foundation for Rare Diseases. Miro Pastrnak, Ph.D. is the Chief Strategy Officer at Rare Disease Research. Finally, Shion Chang, MPA, MSPH, is the Senior Director, Policy & Regulatory Affairs at the National Health Council.

Attend in person on Capitol Hill in Washington, DC (House Rayburn Room 2044) or join us virtually on Zoom.

Please use the following link to register to attend in-person or virtually: https://hubs.li/Q03WsSCg0

12/01/2025

Rare Disease Policy Update: What Advocates Need to Know Before 2026. Join us on Tuesday, December 2, at 1 PM ET for the latest RDLA webinar. This webinar focuses on what the rare disease policy landscape looks like as the year comes to a close. Three insightful speakers will lead the discussion.

The featured speakers are:
• Annie Kennedy, Chief of Policy, Advocacy, and Patient Engagement at the EveryLife Foundation for Rare Diseases
• Jamie Sullivan, Vice President of Policy at the EveryLife Foundation for Rare Diseases
• Jennifer Dexter, Vice President of Policy at the National Health Council

Please register for the webinar here: https://hubs.li/Q03Wh-Ky0

11/20/2025

We are excited to introduce the 2025 RareVoice Awards Finalists! The RareVoice Awards honors advocates who champion and amplify the rare disease patient voice in state and federal policy. Finalists were selected by a committee from nominations made by the rare disease community.

Let’s congratulate our finalists for Federal Advocacy: Congressional Staff!

• Ruth McDonald, Office of Senator Amy Klobuchar
• Jacquelyn Gulshen, Office of Senator Markwayne Mullin
• Hannah Spengler, Office of Representative Don Davis

The 2025 RareVoice Awardees will be announced in January and featured at Rare Disease Week on Capitol Hill on February 24-26, 2026. A separate awards ceremony will not be held in 2025. To see all finalists visit: https://hubs.li/Q03VhzBx0

10/30/2025

Happening Today! Catch up on the latest legislative developments affecting the rare disease community. Join our RDLA webinar at 1 pm ET.

Register here ➡️

Webinars - EveryLife Foundation for Rare Diseases

10/23/2025

Applications closing soon! Rare Giving is an EveryLife Foundation program that supports organizations engaging patients, caregivers, and other community stakeholders in advocacy and public policy. To apply, click the link below then select either the “Events/Conferences” or “Tools/Resources” option. Applications close tomorrow, October 24.

Apply here: https://hubs.li/Q03PTH8V0

10/08/2025

Register today! Join us on Thursday, October 9, at 1 PM ET for the latest RDLA webinar titled "Share Your Story." Hear from advocates about their journeys and share your story.

We look forward to seeing all of you there! Please register here: https://hubs.li/Q03MLwgL0

10/03/2025

Apply for a Rare Giving Grant today! Rare Giving is a program of the EveryLife Foundation that supports organizations that engage patients, caregivers, and other stakeholders in the advocacy and public policy community. Applications stay open until Friday, October 24.

Apply here➡️ https://hubs.li/Q03M5_mr0

10/01/2025

⁉️ 𝗪𝗵𝗮𝘁 𝗱𝗼𝗲𝘀 𝘁𝗵𝗲 𝗙𝗲𝗱𝗲𝗿𝗮𝗹 𝗚𝗼𝘃𝗲𝗿𝗻𝗺𝗲𝗻𝘁 𝗦𝗵𝘂𝘁𝗱𝗼𝘄𝗻 𝗺𝗲𝗮𝗻 𝗳𝗼𝗿 𝘁𝗵𝗲 𝗿𝗮𝗿𝗲 𝗱𝗶𝘀𝗲𝗮𝘀𝗲 𝗰𝗼𝗺𝗺𝘂𝗻𝗶𝘁𝘆?

In anticipation of the shutdown, the U.S. Department of Health and Human Services released a contingency plan for each of its agencies outlining how it will operate in the event of a shutdown.

To learn more about how each agency (U.S. Food and Drug Administration, CDC, Medicare, Medicaid, etc.) will be impacted and what the shutdown means for the rare disease community, click the following link: https://hubs.li/Q03LJrk20

09/25/2025

Happening today! Join us at 3:00 PM ET for the latest RDLA webinar. This webinar features four insightful speakers and will focus on rare disease legislative updates and how advocates can support.

Register for the event here➡️ https://hubs.li/Q03K-HHN0