Groundbreaking Medicare Directive Secures Access to Qalsody for ALS Patients

In a historic move, the Centers for Medicare & Medicaid Services (CMS) announced a groundbreaking directive in December, ensuring that people living with SOD1-ALS and enrolled in Medicare Advantage plans can access the FDA-approved treatment Qalsody®. This decision marks a significant milestone in the fight against ALS, paving the way for equitable access to life-changing therapies.

Why Qalsody Matters

Qalsody (tofersen) targets the RNA produced by mutated SOD1 genes, a known cause of a specific form of ALS. According to the pivotal phase 3 VALOR trial published in 2022, Qalsody demonstrated extraordinary promise by reducing levels of mutated SOD1 proteins in cerebrospinal fluid by 35% within eight weeks and lowering neurofilament light chain (NfL) levels—an indicator of neurodegeneration—by 50% in 12-16 weeks.

These biological improvements translated into meaningful clinical benefits, including slower disease progression, enhanced respiratory function, and improved quality of life for patients after one year of treatment. Subsequent real-world data further validated these results, with some patients experiencing stabilization or even improvements in motor function.

The Fight for Access

Despite FDA approval in April 2023, many Medicare Advantage plans denied coverage for Qalsody, deeming it “experimental.” These unjust denials left patients and families grappling with limited options for accessing a potentially life-saving treatment.

Advocates and stakeholders took swift action to address these denials, collaborating with CMS to highlight the importance of Qalsody. By presenting robust evidence of its clinical value and raising awareness of the urgent need for intervention, they ensured policymakers and legislators understood the critical importance of access to this treatment.

CMS responded by requiring Medicare Advantage plans to comply with federal rules mandating coverage of FDA-approved treatments under Medicare Part B. Plans can no longer classify Qalsody as experimental, and they must conduct case-by-case evaluations based on individual medical necessity. Additionally, plans are required to contact enrollees who were previously denied coverage to inform them of the updated policies.

A Victory for the ALS Community

This directive represents a monumental victory for individuals and families impacted by ALS. By securing access to Qalsody, CMS has set a precedent for timely approval of innovative therapies, reaffirming the principle that every person deserves access to the treatments they need.

Looking Ahead

Advocacy efforts remain vital to ensuring equitable access to all ALS treatments. This achievement is a testament to the power of collective action and determination.

As we celebrate this milestone, the work is far from over. United, we will continue to push boundaries, champion research, and fight for a future where ALS is a thing of the past. Save the date for May 9th as we plan and organize a day of Advocacy for ALS.

#EndALS #QalsodyAccess #ALSAdvocacy