Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Growth hormone deficiency
Treatment: Skytrofa 9.1 MG Cartridge
The insurer denied the Skytrofa 9.1 MG Cartridge
The denial is overturned

This is a male patient diagnosed with growth hormone deficiency (GHD). This request is for Skytrofa 9.1 milligrams (mg). As reported; the patient finds the daily shots extremely painful and refuses to take a shot; he will be going to dormitory high school and his parents are worried that he will not take it if has to take it every day. He started growth hormone therapy approximately three years ago; he is taking Norditropin 1.3 milligrams (0.251 milligrams per kilograms per week [mg/kg/wk]). His height velocity is reported to be improved from 5.52 centimeters per year to seven centimeters per year. His growth hormone (GH) stimulation test with arginine and clonidine peaked at 6.17. His magnetic resonance image (MRI) was normal. 

Prior to treatment, the patient's insulin-like growth factor one (IGF1) was 145; insulin-like growth factor binding protein three (IGFBP3) was 3.3.  Approximately three years ago, the patient's insulin-like growth factor one (IGF1) was 125 (37-459); insulin-like growth factor binding protein three (IGFBP3) was 2.98.  Most recently, the patient's testes were four to five milliliters bilaterally and pubic hair was at tanner I.

The patient's weight most recently was 39.5 kilograms.  His mid parental height is 178 centimeters.  His pretreatment height was 127.4 centimeters (- [minus] 2.08 standard deviation scores [SDS]) and approximately a year later was 130.1 centimeters (- [minus] 2.15 standard deviation scores [SDS]) with height velocity over nine months 3.5 centimeters per year.  His height on therapy was 148.6 centimeters (- [minus] 1.23 standard deviation scores [SDS]).

Yes, the proposed Skytrofa is medically necessary.
The peer reviewed literature including consensus clinical guidelines and randomized controlled trials are summarized below:

Classic growth hormone deficiency:
Classic growth hormone deficiency criteria are outlined in the consensus guidelines for the diagnosis and treatment of growth hormone deficiency published by the growth hormone research society [1], the Lawson Wilkins Pediatric Endocrinology Society [2, [3], and the medical guidelines for 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 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 3.0 standard deviations (SD) below the mean; 2) height more than 1.5 standard deviations below the mid parental height standard deviation; 3) height more than 2.0 standard deviations below the mean and a height velocity over one year more than 1.0 standard deviations below the mean for chronological age, or a decrease in height 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 more than 2.0 standard deviations below the mean over one year or more than 1.5 standard deviations sustained over two years. The biochemical criteria include low insulin-like growth factor one (IGF1) or insulin-like growth factor binding protein three (IGFBP3) or growth hormone stimulation testing with a peak growth hormone less than ten nanograms per milliliter (ng/mL).

This patient met biochemical criteria. Growth hormone stimulation testing peaked at less than ten nanograms per milliliter. The patient met height criterion #2 as his height is more than 1.5 standard deviations below target height standard deviation.  The patient also met height criterion #3.  The patient met both height and height velocity and biochemical criteria for the diagnosis of growth hormone deficiency. Growth hormone in the treatment of growth hormone deficiency is medically necessary. 

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.  It has the advantage of being a weekly medication. 

Given the patient's difficulties with daily injections and the fact that the requested medication is weekly, makes the requested medication superior to the formulary alternatives which are daily.  The request is consistent with the generally accepted standards of medical practice and is therefore medically necessary.

No, 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 as described above.