Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Idiopathic short stature
Treatment: Norditropin
The plan denied Norditropin.
The plan's decision is overturned. 

This is a male diagnosed with idiopathic short stature (ISS). This request is for Norditropin 1.4 milligrams (mg) 7 days a week. As reported, he has Duchene's Muscular Dystrophy at age 4. His growth hormone (GH) stimulation test with Arginine and Clonidine peaked at 10.8. His Insulin-like Growth Factor 1 (IGF1) was 355 (29-424). 
His weight was 31.7 kilograms (kg).

His height at 10.541 years old was 123.9 centimeter (cm) (-2.62 standard deviation score (SDS)) and at 11.11 years old was 126 cm (-2.66 SDS) with height velocity over 7 years old was 3.7 centimeter (cm) / year. His mid parental height is 179.07 cm (+0.31 SDS). His predicted adult height is 152.908 cm (-3.31 SDS).

At issue is the medical necessity for the requested medication, Norditropin. The health plan's determination of medical necessity is overturned, in whole. The request is medically necessary.

The peer reviewed literature including consensus clinical guidelines, randomized controlled trials are summarized below:

Classic GH deficiency criteria are outlined in the consensus guidelines for the diagnosis and treatment of GH deficiency published by the GH research society [1], the Lawson Wilkins Pediatric Endocrinology Society [2, 3] and the medical guidelines for GH use provided by the American Association of Clinical Endocrinologists. [4] The above referenced guidelines are the latest available guidelines from these respective societies. The diagnosis is based on height, height velocity, biochemical and sometimes radiological findings. The height and height velocity criteria based on consensus guidelines upon which a biochemical evaluation is to be initiated are 1) severe short stature, defined as a height more than 3 standard deviation (SD) below the mean; 2) height more than 1.5 SD below the mid parental height SD; 3) height more than 2 SD below the mean and a height velocity over 1 year more than 1 SD below the mean for chronological age, or a decrease in height SD of more than 0.5 over 1 year in children over 2 years of age; 4) in the absence of short stature, a height velocity more than 2 SD below the mean over 1 year or more than 1.5 SD sustained over 2 years. The biochemical criteria include low IGF1 or Insulin-like growth factor binding protein 3 (IGFBP3) or GH stimulation testing with a peak GH less than 10 nanograms/milliliter (ng/ml).

The patient did not meet biochemical criteria. Growth hormone stimulation testing peaked at greater than 10.

Growth hormone in the treatment of idiopathic short stature (ISS) is supported by randomized controlled trials and consensus guidelines. [4-9] FDA approval for this indication is for "non-growth hormone-deficient short stature, defined by height SDS less than or equal to -2.25, and associated with growth rates unlikely to permit attainment of adult height in the normal range, in pediatric patients whose epiphyses are not closed and for whom diagnostic evaluation excludes other causes associated with short stature that should be observed or treated by other means." [10-13]

The patient had a pretreatment height less than or equal to -2.25 SD. The patient's height velocity and bone age would not allow the patient to reach a normal adult height. The request therefore meets idiopathic short stature criteria.

The request is consistent with generally accepted standards of medical practice, and is therefore medically necessary.