Appeal type: Medical necessity
Decision: Upheld

Diagnosis: Growth hormone deficiency (GHD). 
Treatment: Skytrofa 6.3 mg (milligrams) weekly. 

The insurer denied coverage for Skytrofa 6.3 mg weekly.  
 
The denial is upheld. 

This patient was diagnosed with GHD. This request is for Skytrofa 6.3 mg weekly (0.24 mg x kg (kilograms) x wk (week). As reported; he has been receiving samples of Skytrofa and he is responding well. His GH (growth hormone) stimulation with clonidine and arginine peaked at 8.53. His MRI (magnetic resonance imaging) was normal. His IGF1 (Insulin Like Growth Factor 1) was 66 (80-398); Z-score (standard score) was -2.1. His height velocity is reported to be 3.924 cm (centimeters)/year however the chart shows a normal height velocity.  His testes were 1-2 ml (milliliters) bilaterally and pubic hair was at tanner 1. 
His weight was 27.2 kg. 
His height was 133.4 cm (-0.26 SDS (standard deviation scores)). 

His mother's height is 172.72 cm (centimeter) and father's height is 190.5 cm and mid parental height is 188.11 cm (+1.59 SDS). 

The health plan acted reasonably and with sound medical judgment and in the best interest of the patient as the request is not medically necessary as described below. 

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 SD (standard deviation) 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 year 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 yr (years). The biochemical criteria include low IGF1 or IGFBP3 (insulin-like growth factor binding protein) or GH stimulation testing with a peak GH less than 10ng (nanograms)/ml (milliliter). The requested medication has been shown to be safe and effective for the treatment of growth hormone deficiency [5, [6]. The study using the medication shows that the medication is clinically equivalent to daily injections of growth hormone in terms of efficacy and safety. The study showed a slight advantage of the Skytrofa over daily somatropin but only went for 1 year and did not show improved final height. It has the advantage of being a weekly medication. Other than the convenience of weekly administration, the requested medication is not superior to daily growth hormone. 

The patient did not meet height and height velocity criteria as pretreatment height and height velocity are normal. GH stimulation testing peaked at less than 10ng/ml. Although the patient's height is discordant with the midparental target height, the bone age is delayed on the height prediction is normal. 

The pediatric endocrine society recommends against making a diagnosis of growth hormone deficiency based solely on failed growth hormone stimulation testing. 

The request is not consistent with generally accepted standards of medical practice and is therefore not medically necessary. 

The references provided are the most current/relevant to the case. 

The insurer's denial of coverage for Skytrofa is upheld. Medical Necessity is not substantiated.