UPDATE: Promising Pathway Act: Senate Hearing Sheds Light on the Challenges of Getting Access to Treatments for Diseases like ALS – October 26, 2023

On October 26, 2023, the United States Senate Special Committee on Aging held a hearing: Unlocking Hope: Access to Therapies for People with Rare, Progressive, and Serious Diseases. Here’s what unfolded and what it means for people living with ALS, their caregivers and loved ones, and access to future medical advancements.

I AM ALS advocates took a prominent role in the bipartisan Congressional press conference and subsequent Senate Aging Committee hearing. Senator Braun took a moment to observe that this was one of the most crowded Aging hearings that he’d seen. That’s because over 50 ALS community members took the time to attend the press conference, while hundreds more watched online.

In “the room where it happened,” I AM ALS’ Promising Pathway Act subsquad co-chair Tim Tobin addressed the critiques of PPA  at the press conference with Senators Gillibrand & Braun. Co-founder Brian Wallach and Board member Peggy Plews-Ogan, MD were official witnesses at the hearing, presenting testimony on the need for PPA in the ALS community and answering questions from the Senators.

The first hand experiences, personal stories, and passionate advocacy of Tim, Brian and Peggy during the Congressional events provided a deep, human context to the discussion, emphasizing the urgent need for accelerated treatments.

Also testifying were:

Maureen Bell, Galactosemia Patient and Advocate
Anish Bhatnagar MD, CEO of Soleno Therapeutics
Holly Fernandez-Lynch JD, MBe, Assistant Professor of Medical Ethics and Law, University of Pennsylvania
Keith Desserich, Chairman/Co-Founder of The Cure Starts Now Foundation, President of Pediatric Brain Tumor Consortium Foundation

The Promising Pathway Act is legislation aimed at expediting access  to potentially life-saving treatments for life-threatening diseases, like ALS, while further study occurs and full FDA is sought.  

Key takeaways from the hearing

A Bipartisan Push for Faster Approvals: The central theme of the hearing was the need for speed. With diseases like ALS, where time is of the essence, waiting years for a potential treatment is devastating, and thousands of lives are lost while potential treatments slog through the regulatory process that can often take over 15 years to get FDA approval.    

Safety and Efficacy: While speed is crucial, it’s equally important to ensure that these treatments are both safe and effective. The I AM ALS community and other experts participating in the day’s proceedings highlighted the importance of a balanced approach.

Collaborative Approach: The hearing underscored the necessity of collaboration between the medical community, policymakers, and patient advocates. Only through a unified effort can the process be swift, safe, and effective.

Next Steps 

Because of the tenacious efforts of many I AM ALS advocates (THANK YOU!) that met with and wrote every member of the Senate Aging committee prior to the hearing, Senators Braun (IN), Gilibrand (NY), Casey (PA), Ricketts (NB), Warren (MA), Blumenthal (RI), Warnock (GA), Vance (OH), and Scott (FL) all attended the hearing. This important bill is on their radar because of you.

The Senate hearing on the Promising Pathway Act, with I AM ALS advocates playing a central role, marks a significant milestone in the movement against ALS. As the legislative process unfolds, it’s crucial for all stakeholders to continue the dialogue, ensure patient safety, and work towards the ultimate goal: a world without ALS.

A gigantic thank you to the community, to I AM ALS’ PPA and Legislative teams for their engagement, continued tenacity for educating members of Congress about the importance of speeding up access to new treatments for unmet, 100% fatal diseases such as ALS. And thank you to everyone that took time to get to the Dirksen building to watch the hearing or took time to watch the hearing online. It was a hopeful day.

You can help make the Promising Pathway Act a law in three ways:

  1. Contact your senators and representative about the Promising Pathway Act: https://www.iamals.org/action/promising-pathway-act/
  2. Join the legislative team: https://www.iamals.org/action/legislative-affairs-team/ 
  3. Help the legislative team by becoming a constituent: https://www.iamals.org/action/help-the-legislative-team

We will find cures for ALS. The only question is when. That answer depends on you.

Stay informed on I AM ALS’ progress and what you can do to help us make hope real.

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