Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Idiopathic Short Stature. 
Treatment: Norditropin Flexpro 30mg/3ml Pen Injctr, 0.067mg/kg Day Subcutaneous Injection Daily For 1 Year. 

The insurer denied the Norditropin Flexpro 30mg/3ml Pen Injctr, 0.067mg/kg Day Subcutaneous Injection Daily For 1 Year. 
The denial is overturned. 

The patient is a male diagnosed with ISS (idiopathic short stature). This request is for Norditropin. As reported; he has been on interim coverage Norditropin from Novocare for approximately six months. He has been taking 1 mg (milligram) daily and tolerating the medication well. His height velocity has improved from 5.28 centimeters per year to 11.3 centimeters per year. 

The patient's weight was 20.7 kg (kilograms). His height was 111.8 centimeters (-[minus] 3.15 SDS [standard deviation score]). It was 115 centimeters (- [minus] 3.11 SDS [standard deviation score]) with height velocity over 0.65 years of 4.9 centimeters per year. 

The patient's height on therapy was 118 centimeters (- [minus] 2.70 SDS [standard deviation score]). His mid-parental height is 172.72 centimeters (- [minus] 0.58 SDS [standard deviation score]). 

Yes, the requested Norditropin is medically necessary. 

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

Classic GH (growth hormone) deficiency 
- Classic GH (growth hormone) deficiency criteria are outlined in the consensus guidelines for the diagnosis and treatment of GH (growth hormone) deficiency published by the GH (growth hormone) research society (1), the Lawson Wilkins Pediatric Endocrinology Society (2, 3), and the medical guidelines for GH (growth hormone) 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 auxological, biochemical and sometimes radiological findings. 

The auxological criteria based on consensus guidelines upon which a biochemical evaluation is to be initiated are: 
1) Severe short stature, defined as a height of more than 3 SD (standard deviation) below the mean; 
2) Height of more than 1.5 SD (standard deviation) below the midparental height; 
3) Height of more than 2 SD (standard deviation) below the mean, and a height velocity over one year of more than 1 SD (standard deviation) below the mean for chronological age, or a decrease in height SD (standard deviation) of more than 0.5 over one year in children over two years of age; 
4) In the absence of short stature, a height velocity of more than 2 SD (standard deviation) below the mean over one year, or more than 1.5 SD (standard deviation) sustained over two years. 

The biochemical criteria include low IGF1 (insulin-like growth factor 1) or IGFBP3 (insulin-like growth factor binding protein 3) or GH (growth hormone) stimulation testing, with a peak GH (growth hormone) of less than 10 ng/ml (nanograms per milliliter). 

The patient did not meet biochemical criteria. Growth hormone stimulation testing is not reported. 

Idiopathic Short stature 
- Growth hormone in the treatment of idiopathic short stature (ISS) is supported by randomized controlled trials and consensus guidelines (4-9). FDA (Food and Drug Administration) approval for this indication is for "non-growth hormone-deficient short stature, defined by height SDS (standard deviation score) less than or equal to - [minus] 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 had a pretreatment height less than or equal to - [minus] 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 patient has benefited from growth hormone with height velocity improvement and a height SD (standard deviation) improvement. 

The request is consistent with generally accepted standards of medical practice and is  in the best interest of the patient, as the request is medically necessary as described above.