ALS Roundtable on Ensuring Access to New Therapies for People With ALS

With many ALS drugs now in phase II and III clinical trials, The ALS Association is considering strategies that will ensure any new treatments are accessible and affordable. We used our second ALS Roundtable to explore several important questions for our community including: How will these new therapies get paid for? How can people access them? How long will it take to get access? 

We gathered nearly 50 people at the Roundtable from the extended ALS community, including people with ALS and caregivers, representatives from the FDA, patient-centered organizations, drug companies, public and private insurers, and clinicians for a day-long focused and facilitated discussion in Washington D.C. Participants discussed the following key challenges impacting people with ALS and their families in accessing potential new treatments:

Value and Coverage Criteria

Public and private insurers (payors) require evidence to evaluate therapies and offer coverage. Sometimes what payors require does not align with drug developers’ clinical trial outcome measures (what was used in the regulatory process for approval by the FDA). Additional evidence is needed to demonstrate the value of new therapies to the ALS population. For example, they want to see that a therapy improves a person’s quality of life. Gathering information about patient preferences and experiences to understand the elements of life that matter most to people with ALS can support favorable coverage decisions by payors. Not only does this process require specific information, but it also takes time, adding to the delay a person with ALS might experience in accessing a new treatment.  

Financial Burden

ALS care is very expensive and creates a significant financial pressure for people with ALS and their families. New treatments will increase this financial burden. Additionally, not everyone is eligible for co-pay assistance and other benefits. 

Authorization Processes and Administrative Burden

There is a separate set of processes to get a prescription approved for an individual patient (pre-authorization, denial/appeals, utilization review, etc.). These processes require paperwork that can be burdensome for people with ALS, their families, clinicians, and their staff. 

Benefits Navigation

Navigating insurance coverage for existing therapies is extremely complex. New therapies that are on the horizon, when approved, will add to the complexity. Education around how the therapy works, how it is administered, who is eligible to take the drug, and clear options for insurance coverage are important to help to avoid confusion and allow access to therapies as quickly as possible. 

After the Roundtable participants identified the above challenges to accessing potential new therapies, they discussed how the Association can help the ALS community in addressing these challenges. Our solutions-based discussion was a team effort, where all the stakeholders in the room played their part both identifying solutions and suggesting how to make those solutions actionable. Further, our Roundtable helped strengthen the collaboration between people developing, approving, and funding new treatments (the representatives from pharma, the FDA, and insurers at the meeting), which will help facilitate the smooth introduction of specific new treatments to people with ALS after FDA approval. 

In the months ahead, The ALS Association will be working with key stakeholders to address these challenges in smaller groups for the entire ALS community. The Association will keep our ALS community updated on next steps. 

Thank you to all that participated under the following affiliationsADVI Health, LLC, Alliance for Regenerative Medicine, Avalere, Blue Cross Blue Shield of America, District Policy Group at Drinker Biddle, Faegre Baker Daniels, FountainHead HealthCare, Louis Stokes Cleveland VA Medical Center, MDA ALS Center of Hope at Temple University, National Health Council, WSCollaborative, LLC, representatives from theU.S. Food and Drug Administration, members of The ALS Association national and local leadership and the Board of Trustees, and most importantly people with ALS and their caregivers.

Thank you to our corporate sponsors: AveXis, Biogen, BiohavenPharmaceuticals, Genentech, Ionis Pharmaceuticals, Mitsubishi Tanabe Pharma America, Orion Pharma, and Orphazyme.

This meeting was the latest milestone for the ALS Association Roundtable Program, which launched in early 2019 to formally engage our partners in our strategic planning efforts and to identify strategies that will impact the Association’s mission-centric work – ALS care, advocacy, and research – in the near and long term.


Submitted by: Melody L. on Tue, 12/14/2021

We are seeking to learn more about the roundtable or other groups within the organization.

Submitted by: Amy L. on Thu, 12/16/2021

Hi Melody. Please feel free to reach out to us with your questions: [email protected]

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