Appeal type: Experimental/Investigational
Decision: Overturned

Reviewer #1:

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 presented for lumbar puncture for intrathecal tofersen injection.  The patient had superoxide dismutase 1 (SOD 1) positive amyotrophic lateral sclerosis (ALS).  The patient did very well with the last tofersen administration without post lumbar puncture headache.  Since starting the treatments, the patient felt that there had been a dramatic slowing of the rate of progression.  The patient's last spinal fluid results remained benign.  The protein was mildly elevated at 84.  The nucleated cells were 4.  The provider recommended continuation of intrathecal infusion of tofersen.  

In an undated appeal letter, the provider requested reconsideration for the patient's J3490 Qalsody (tofersen) 100mg.  The provider felt that the most appropriate treatment for the patient fell outside of the established policy plan.  The patient had a diagnosis of amyotrophic lateral sclerosis with the SOD1 gene mutation.  Denying the medication placed the patient at risk for quicker progression of disease.  There were no other formulary alternatives.  The request was continuation of therapy as the patient had been receiving the medication in the past year.  There was a goal to slow progression of disease for the patient.

The Food and Drug Administration (FDA) states that Qalsody (tofersen) is indicated for the treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene.  The maintenance dosage is 100 mg every 28 days.  The medication is approved under the accelerated approval pathway, under which Food and Drug Administration (FDA) may approve drugs for serious conditions where there is an unmet medical need, and the drug is shown to have an effect on a surrogate endpoint that is reasonably likely to predict the clinical benefit to patients.  In this case, the patient was being treated for SOD 1 positive amyotrophic lateral sclerosis (ALS).  The patient had been receiving tofersen with reported benefit.  Continuation of the treatment was recommended. 

The Food and Drug Administration approves tofersen for the patient's clinical presentation and diagnosis. 

There is support for expectation of improvement in health outcomes, the medication has approval from the appropriate regulatory bodies for the diagnosis, and the patient is a good candidate for the medication.  

Given the paucity of available treatment options, the treatment is likely to be more beneficial than prior available options and the benefit of treatment outweighs risk.  As such, the denial of coverage for J3490 Qalsody (tofersen) 100mg is overturned.

In summary, the denial of code J3490 Qalsody (tofersen) 100mg pre-service and coverage for the requested service 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.