Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Spinal Muscular Atrophy.
Treatment: Spinraza, Pre-service.

The insurer denied the Spinraza, Pre-service.  The denial is overturned.

The patient is a female with spinal muscular atrophy. She started the treatment with Spinraza, and she was receiving this treatment every four months. Since this treatment, she has made steady gains. She can independently steer her power wheelchair, sit unassisted, communicate, and spend some time without ventilator. Her CHOP-INTEND (Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders) scores have improved from 15/64 from four years prior to 47/64 most recently.

According to the letter of medical necessity provided, without treatment with Spinraza, the patient will begin to lose key functions. The patient's prognosis is stable with continued treatment, but without Spinraza, her prognosis is very poor.

The patient was seen, and it was noted that four years prior, the patient was completely paralyzed, but then after starting Spinraza, she regained quite a bit of her function and had continued progression of improved motor skills. She was dependent on a ventilator and tracheostomy, but was able to come off her ventilator for 10 to 15 minutes.  Neurological exam showed continued improvements of motor function and stabilization of respiratory status with continued treatment with Spinraza. It was noted that it was medically necessary for the patient to continue treatment with Spinraza.

Spinraza was denied by the health plan, as the patient is dependent on her tracheostomy.

Yes, the treatment with Spinraza is medically necessary.

Medical necessity means a service, medication, or treatment is in accordance with generally accepted standards of medical practice, clinically appropriate in terms of type, frequency, extent, duration, and considered effective for the illness or symptoms, and they must not be provided primarily for the convenience of the patient or physician. The treatment or procedure must not be more intensive than the same or similar service provided by a different provider or an alternative service that is at least as likely to produce an equivalent therapeutic or diagnostic result.

Spinraza is approved for spinal muscular atrophy in order to prevent deterioration of the motor function. Use of this medicine is within the standards of medical care and is supported by the FDA (United States Food and Drug Administration) approval and by the current medical literature.

In this patient's case, the medicine is not only stopping the decline in the patient's neuromuscular status, but is also helping to gain motor abilities. This treatment is clinically appropriate in terms of the type, frequency extent and duration of the therapy and has been proven effective for the patient's symptoms of spinal muscular atrophy. The treatment is not primarily for the patient's convenience. The treatment is not more intensive than other services, since less intensive treatment alternatives able to produce similar results do not exist. Therefore, the definition of medical necessity is met.

Previously, this service was denied because the patient has tracheostomy.  However, this patient has been on respiratory support all along. Despite respiratory dependence, she nevertheless enjoys improving motor skills, ability to sit up and use upper extremities and navigate power wheelchair.  Food and Drug Administration approval and label do not make any reference to respiratory dependence as a contraindication to Spinraza.

The available medical records clearly demonstrate that Spinraza, being an FDA (Food and Drug Administration) approved medicine for the patient's diagnosis, promotes her health, improves her motor function, improves her well-being, and prevents deterioration. Therefore, the patient's best interest is to continue Spinraza.