Advocacy Announcement – June 26, 2020

The Les Turner ALS Foundation has joined with the ALS community in support of two pieces of legislation – the Promising Pathway Act (S. 3872) and the Accelerating Access to Critical Therapies for ALS Act (H. 7071), which is co-sponsored by one of our Illinois members of Congress, Rep. Mike Quigley (IL-5).

“We are grateful to partner with patients and fellow patient advocacy groups to advance these initiatives, which are desperately needed,” said Foundation CEO Andrea Pauls Backman. “One of the pillars of our mission is to advance scientific research into the causes, treatment and prevention of ALS, and we believe these bills, co-produced with people living with ALS, will provide earlier and easier access to promising therapies that could ultimately lead to a cure.”

*About These Bills

  1. Accelerating Access to Critical Therapies for ALS Act (HOUSE BILL)

○ On June 1, Congressman Jeff Fortenberry (R) and Congressman Mike Quigley (D) introduced the Accelerating Access to Critical Therapies for ALS Act (H.R.7071) in the House of Representatives.

○ ACT for ALS will create the infrastructure to fund early access to promising therapies for patients diagnosed with fast-progressing terminal diseases like ALS.

○ The bill authorizes $75,000,000 in FY2021 and 2022 as part of a pilot to provide grants at the National Institutes of Health (NIH) to support expanded access programs.

○ The bill will establish a Center of Excellence for Neurodegenerative Diseases at the FDA to accelerate the development and approval of therapies for the coming tide of neurodegenerative diseases that will cause a health and economic crisis.

○ The Center will be modeled after FDA’s Center of Excellence for Oncology that has driven forward many monumental discoveries in cancer research, including the creation of a corollary for Project Facilitate, which provides a single point of contact where FDA oncology staff will help physicians and their healthcare team through the process of submitting an Expanded Access request for an individual patient with cancer.

  1. Promising Pathway Act (SENATE BILL)

○ On June 4, Senator Mike Braun, Senator Lisa Murkowski and Senator Martha McSally introduced the Promising Pathway Act (S.3872) in the Senate.

○ The bill will speed up the regulatory process for getting drugs showing benefits to the patients who need them.

○ It ​requires the FDA to establish a real-time, priority review pathway to evaluate provisional approval applications for drugs intended to treat, prevent, or diagnose serious or life-threatening diseases or conditions.

○ Under this pathway, provisional approval would be granted by the FDA to treatments demonstrating substantial evidence of safety and relevant early evidence of positive therapeutic outcome(s).

○ Moreover, the bill would require CMS to cover therapies provisionally approved through this pathway and prohibit private insurance from denying coverage to therapies that are made available under this pathway on the basis that they are investigational therapies.

○ Finally, it would require the creation of registries for those using a provisionally approved drug and the sharing of data from these registries to help speed the overall fight.

 *Summary of bills provided by I AM ALS