Appeal type: Medical necessity
Decision: Upheld

Diagnosis: Growth Hormone deficiency  

Treatment:  Norditropin  

The insurer denied the medication Norditropin.  The denial was upheld.  

This patient is a male child with a diagnosis of growth hormone deficiency.  He was evaluated by his pediatric endocrinologist, and the medication Norditropin was recommended.  His GH stimulation test in late 2019 with clonidine peaked at 0.1, and L-dopa peaked at 2.3, respectively.  His MRI was normal.  His height velocity is reported to be 2 cm/year.  His bone age was 10 years at greater chronological age (CA).  His testes are 2-3 cc bilaterally and pubic hair is at tanner 1.  

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 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 more than 3 standard deviations (SD) below the mean; 2) height more than 1.5 SD below the mid-parental height; 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 10ng/ml.  

The patient did not meet auxological criteria as pretreatment height, and height velocity do not meet the above criteria.  The patient has familial short stature, as his height and height prediction are consistent with his mid-parental target height.  

GH stimulation testing peaked at less than 10ng/ml.  

The Pediatric Endocrine Society recommends against making a diagnosis of growth hormone deficiency based solely on failed growth hormone stimulation testing.

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 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 pre-treatment height less than or equal to -2.25 SD.  Therefore, the patient does not meet idiopathic short stature criteria. 

The request does not meet any nationally accepted criteria for use of growth hormone.  Therefore, the request is not medically necessary.  

Based on the above, the medical necessity for Norditropin is not substantiated.  The insurer's denial is upheld.