Appeal type: Experimental/Investigational
Decision: Overturned

Reviewer #2:

Diagnosis: Amyotrophic lateral sclerosis (ALS)

Treatment: code J3490 Qalsody (tofersen) 100 milligrams (mg)

The insurer denied coverage for code J3490 Qalsody (tofersen) 100 milligrams (mg)

The denial is overturned

The patient is seeking coverage for J3490 Qalsody (tofersen) 100 milligrams (mg).  

The patient was seen for follow up regarding familial amyotrophic lateral sclerosis (ALS). The patient had a positive superoxide dismutase 1 (SOD1) gene mutation. The patient was receiving monthly intrathecal Tofersen. The patient reported worsening weakness in the right lower extremity. The patient was on Riluzole and was awaiting approval of Relyvrio. The physical exam showed mild progression of the right lower extremity weakness. The plan was to continue with the treatment. 

The patient was seen by the doctor for lumbar puncture for intrathecal tofersen injection. The patient is slowing in their condition progression since starting the treatment.

The scientific evidence in peer-reviewed literature does support a result of improvement in health outcome.

The patient reported worsening weakness in the right lower extremity and the medication slowed progression of the disease.      

Code J3490 Qalsody (tofersen) 100 milligrams (mg) pre-service does have final approval from the appropriate regulatory bodies for this diagnosis.

The medication is Food and Drug Administration (FDA) approved for the patient's condition.     

This patient is a good candidate for code J3490 Qalsody (tofersen) 100mg pre-service.

Code J3490 Qalsody (tofersen) 100mg pre-service is the best available treatment for this patient at this time.

The patient has a diagnosis of SOD1 mutation ALS. The patient has reported worsening weakness in the right lower extremity and the medication slowed progression of the disease. The medication is Food and Drug Administration (FDA) approved for the patient's condition.     

The adverse risks are not increased over standard treatment/services.

The patient is tolerating the treatment.     

In this condition, there are no other alternative treatments. All the known medications should be given at the same time to help slow the progression of this lethal and progressive disease.

"FDA approved Qalsody (tofersen) to treat patients with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene (SOD1-ALS). Qalsody is an antisense oligonucleotide that targets SOD1 messenger ribonucleic acid (mRNA) to reduce the synthesis of SOD1 protein. The approval was based on a reduction in plasma neurofilament light (NfL), a blood-based biomarker of axonal (nerve) injury and neurodegeneration. Qalsody is administered intrathecally (through a spinal injection) by healthcare professionals who are experienced in performing lumbar punctures. The recommended dosage is 100 mg (15 milliliters (mL)) per administration. Patients receive three initial doses administered at 14-day intervals, followed by a maintenance dose every 28 days" (Food and Drug Administration, 2023).

In summary, the denial of code J3490 Qalsody (tofersen) 100mg pre-service as experimental/investigational and coverage for the requested service/treatment should be overturned. This treatment is likely to be more beneficial than any standard treatments for this patient's condition and the adverse risks are not substantially increased over standard treatments.