Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Endocrine/Metabolic/Nutritional/Idiopathic Short Stature
Treatment: Pharmacy/Prescription Drugs/Norditropin Flexpro
The health plan denied the requested prescription medication Norditropin Flexpro as not medically necessary. 
The health plan's determination is overturned.

This is a male diagnosed with idiopathic short stature (ISS). This request is for Norditropin. His growth hormone (GH) stimulation test with Arginine and Glucagon peaked at 19. His Insulin-like Growth Factor 1 (IGF1) was 54. His height velocity is reported to be 3.9 centimeters/year (cm/yr). His bone age was 6 at chronological age (CA) 7 years 3 months. His IGF1 was 70; Insulin-like growth factor binding protein 3 (IGFBP3) was 2.87.
His weight was 21.9 kilograms. His height at 8.6 years old was 119 cm (-2.14 standard deviation score (SDS)) and later at 9.1 years old was 120.7 cm (-2.26 SDS) with height velocity over 0.5 years of 3.4 cm/yr. His mid parental height is 175.4 cm (-0.20 SDS). His predicted adult height is 159.57 cm (-2.40 SDS)

The determination is overturned, in whole, as the request is medically necessary, as described above.

Yes. The requested health service/treatment of Norditropin Flexpro is medically necessary.
The peer reviewed literature including consensus clinical guidelines, randomized controlled trials are summarized below:
Classic GH deficiency
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 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.
Idiopathic Short Stature
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 (standard deviation score) 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 (standard deviation). 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.