Appeal type: Medical necessity
Decision: Upheld

Diagnosis: Idiopathic Short Stature

Treatment: Medication Norditropin Flexpro 10 milligrams (mg) per 1.5 milliliter (ml) syringe

The insurer denied coverage for medication Norditropin Flexpro 10 milligrams per 1.5 milliliter syringe.

The denial is upheld. 

This is a male diagnosed with short stature. This request is for Norditropin 1.8 milligrams (mg) daily. As reported, he is on growth hormone (GH) therapy, 1.8 mg daily (0.28 mg/kg [kilogram]/wk [week]). His GH stimulation test with arginine and clonidine peaked at 23.50. 

The health plan acted reasonably, with sound medical judgment, and in the best interest of the patient. The request is not medically necessary as described 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 SD (standard deviations) 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; 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 (insulin-like growth factor 1) or IGFBP3 (insulin like growth factor binding protein 3) or GH stimulation testing with a peak GH less than 10 ng/ml (nanograms per milliliter).

The patient did not meet biochemical criteria. Growth hormone stimulation testing peaked at greater than 10. The patient had a normal IGF1 and IGFBP3. Furthermore, there is no evidence of growth hormone resistance as the growth factor levels are all normal.

Growth hormone in the treatment of idiopathic short stature (ISS) is supported by randomized controlled trials and consensus guidelines  [4-9]. Food and Drug Administration (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."

The patient does not have a pretreatment height less than or equal to -2.25 SD. The patient therefore does not meet idiopathic short stature criteria. 

The request does not meet any nationally accepted criteria for use of growth hormone. The request is therefore not medically necessary. The health plan acted reasonably, with sound medical judgment, and in the best interest of the patient.

The insurer's denial of coverage for the medication Norditropin Flexpro 10 milligrams per 1.5 milliliter syringe is upheld. Medical necessity is not substantiated.