Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Idiopathic Short Stature, Growth Hormone Deficiency
Treatment: Norditropin Flexpro 30 milligrams (mg)/3 milliliter (ml) pen injector, 2 mg subcutaneously injected daily
The insurer denied Norditropin Flexpro 30 mg/3 ml pen injector, 2 mg subcutaneously injected daily.
The health plan's determination is overturned.

The patient is a female who is diagnosed with idiopathic short stature (ISS). She has been on growth hormone (GH) therapy. Her Insulin-like Growth Factor 1 (IGF1) was 77; and her standard score (Z-score) was minus (-)1.1 standard deviation (SD). Her Insulin-like growth factor binding protein 3 (IGFBP3) was 3.8. Her breasts were at tanner 2. The provider did not do a growth hormone (GH) stimulation test.

Her height and weight was at -0.55 standard deviation score (SDS). The pretreatment height was below the 2nd percentile. The height velocity was below the 3rd percentile. The mid parental height is 159.87 centimeters (cm) (-0.54 standard deviation score (SDS)).

The health plan did not act reasonably, with sound medical judgment and in the best interest of the patient as 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 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 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 is not reported and growth factors are normal.

Growth hormone in the treatment of idiopathic short stature (ISS) is supported by randomized controlled trials and consensus guidelines [4-9]. The 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."[10-13]

The patient had a pretreatment height less than or equal to -2.25 SD. The patient's pretreatment height velocity does not allow the patient to reach a normal adult height. The request therefore meets idiopathic short stature criteria. The patient has benefitted from growth hormone. Continuation is consistent with generally accepted standards of medical practice. The request is consistent with generally accepted standards of medical practice and is therefore medically necessary.

The health plan's determination of medical necessity is overturned.