Medicare Advantage plans in US told to cover Qalsody for SOD1-ALS
ALS Association work leads to directive that therapy no longer ‘investigational'
The Centers for Medicare and Medicaid Services (CMS) has issued a directive requiring that Medicare Advantage plans cover Qalsody (tofersen) to treat people with amyotrophic lateral sclerosis (ALS) caused by mutations in the SOD1 gene.
The directive comes after the ALS Association worked closely with the CMS, presenting evidence of widespread instances where coverage was wrongly denied patients who met all the necessary criteria for the treatment. According to the group, the order is the first of its kind to be issued by the center.
Qalsody, an injectable treatment marketed by Biogen, was conditionally approved by the U.S. Food and Drug Administration (FDA) last year under its accelerated pathway. However, many Medicare Advantage plans, run by private companies, classified the drug as “experimental and investigational,” allowing those plans to “have a blanket coverage policy that excludes Qalsody from Medicare Part B coverage,” the CMS wrote in its directive.
Medicare coverage denied based on FDA’s accelerated approval path
However, “drugs or biologicals approved for marketing by the FDA are considered safe and effective when used for indications specified on the labeling and CMS does not make a distinction between drugs that are marketed under an accelerated FDA-approval versus a traditional FDA-approval,” the center added.
Medicare is a federal health insurance program for older people and some younger people with certain disabilities in the U.S. Part B covers costs related to doctor visits, outpatient care, and prescription drugs such as Qalsody.
“This is a victory for the entire ALS community,” Calaneet Balas, president and CEO of the ALS Association, said in a press release from the organization. “It’s critical that FDA-approved treatments are made accessible rather than being labeled as ‘experimental.’ We are grateful to everyone in our community who helped make this day happen.”
The ALS Association was the first to invest in research that laid the grounds for Qalsody’s development, and it was instrumental in getting the treatment approved and accessible across the U.S. When insurance companies began denying access to Qalsody under Medicare Part B, the organization engaged with the CMS, as well as policymakers and legislators, to advocate for people with an urgent need for the treatment.
The association now urges Medicare Advantage patients denied Qalsody coverage to contact their ALS specialist and start the process of getting access.
“We hope this sends a message to the entire rare disease community and pharmaceutical industry that new treatments can be developed and made accessible to those who will benefit from them. We need to ensure more ALS treatments are developed and approved that will help everyone living with ALS,” Balas said.
ALS occurs when motor neurons — the nerve cells in the brain and spinal cord that control voluntary movement — gradually lose function and die, leading to muscle weakness. In some patients, disease-causing genetic mutations result in a faulty version of the SOD1 protein that is toxic to nerve cells.
Insurers ordered to stop classifying Qalsody as ‘experimental, investigational’
Qalsody contains a lab-made antisense oligonucleotide, a small strand of genetic material that binds to the instructions for the faulty SOD1 protein and blocks its production. By reducing the amount of toxic SOD1, Qalsody is expected to ease the ALS symptoms due to SOD1 mutations.
Its approval mainly drew on data from the Phase 3 stage of the VALOR clinical trial (NCT02623699), where Qalsody reduced the amount of faulty SOD1 protein in the cerebrospinal fluid — the liquid that flows around the brain and spinal cord — by 35% within eight weeks. It also halved the amount of neurofilament light chain, a marker of nerve damage, within four months.
These changes amounted to slower disease progression, and better respiratory function and quality of life after one year of treatment. Real-world data also supports these trial findings, showing some patients achieving stable disease or gains in motor function and life quality with Qalsody, the association reported.
“We expect that all [Medicare Advantage] plans that currently classify Qalsody as experimental and investigational for the treatment of ALS in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene will immediately discontinue use of those coverage policies and comply with the rules,” the CMS wrote.
It also stated that plans should contact patients who were inappropriately denied coverage to inform them of the new coverage policies.
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