Appeal type: Experimental/Investigational
Decision: Overturned

Reviewer #3:

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 upheld

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

The patient has a history of amyotrophic lateral sclerosis (ALS) (superoxide dismutase1 [SOD1] mutation positive) who was prescribed Qalsody (tofersen).

An MD (doctor of medicine) note indicated that the patient presented for a lumbar puncture for the intrathecal injection. The patient was noted have a history of SOD1 mutation positive ALS, and has done well with the last administration. The patient felt that there was a dramatic slowing in the rate of progression. The patient underwent lumbar puncture. There was no examination performed.

The scientific evidence in peer-reviewed literature does not support a result of improvement in health outcomes.

The medication was approved because of a reduction in concentration of SOD1 in spinal fluid and reduce neural filament light change response in plasma over 28 weeks but did not improve clinical endpoints and was associated with adverse events. It was approved on the contingency that further data regarding its efficacy would be collected. It is therefore not associated with improved health outcomes.      

Code J3490 Qalsody (tofersen) 100 milligram (mg) pre-service is Food and Drug Administration (FDA) approved for patients with superoxide dismutase mutated amyotrophic lateral sclerosis, to allow for acquisition of additional efficacy data.     

This patient is not a good candidate for code J3490 Qalsody (tofersen) 100mg pre-service.

Additional information is needed to determine its efficacy. Such information can be obtained in a clinical trial and not from outpatient non-prospective observations.     

Code J3490 Qalsody (tofersen) 100mg pre-service is not the best available treatment for this patient at this time.

The patient is on the evidence-based medication riluzole and has been approved for the evidence-based medication Relyvrio. The patient also appears to be a candidate, based on her ALSFRS-R (Amyotrophic lateral sclerosis Functional Rating Scale) scores, for Radicava, but is not taking the medication. These medications are the best available treatments as these have known efficacy, whereas Qalsody does not.     
The adverse risks are increased over standard treatment/services.

Qalsody is known to have adverse effects that would be greater than if the patient was receiving standard of care treatment (riluzole and Relyvrio in this patient) only.     

The requested service is not likely to be more beneficial than any of the standard treatments/procedures for this patient.

Qalsody is not known to improve neurological outcomes in amyotrophic lateral sclerosis and is FDA-approved so additional data can be collected regarding efficacy. It is not likely to be more beneficial than the standard treatments the patient is receiving.

"In persons with SOD1 ALS, tofersen reduced concentrations of SOD1 in cerebrospinal fluid (CSF) and of neurofilament light chains in plasma over 28 weeks but did not improve clinical end points and was associated with adverse events. The potential effects of earlier as compared with delayed initiation of tofersen are being further evaluated in the extension phase." (Miller, T. M., et al. 2022).

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