Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Endocrine/Metabolic/Nutritional/Hypopituitarism
Treatment: Pharmacy/Prescription Drugs/Skytrofa 3.6 mg SC (subcutaneous) CART (cartridge).
The health plan denied the requested prescription medication, Skytrofa 3.6 mg SC CART as not medically necessary. 
The health plan's determination is overturned.

This is a female diagnosed with hypopituitarism. This request is for Skytrofa 3.6 mg (milligrams). As reported; she has severe needle phobia. She was started on Skytrofa and she is responding well on it. Her growth hormone (GH) stimulation test with Clonidine and Arginine peaked at 9.44. Her height velocity is reported to be 1.922 cm (centimeters)/year. Her Insulin-like Growth Factor 1 (IGF1) was 24 (37-272); Z-score was -2.3 standard deviation (SD). Her breasts and pubic hair were at tanner 1. Her weight was 17.2 kilograms.

The patient's pretreatment height was 97 cm (-0.94 standard deviation score (SDS)) and later was 103.3 cm (-2.07 SDS) with height velocity over 1.798 years of 3.5 cm/year. Her mid parental height is 157.48 cm (-0.90 SDS).

The health plan's determination of medical necessity is overturned, in whole.

Yes. The peer reviewed literature including consensus clinical guidelines, randomized controlled trials are summarized below.

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 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 Insulin-like growth factor binding protein 3 (IGFBP3) or GH stimulation testing with a peak GH less than 10ng/ml (nanograms/milliliter).
This patient met biochemical criteria. GH stimulation testing peaked at less than 10ng/ml. the patient had a low IGF1. The patient met height criterion 3 as his height is more than 2 SD below the mean and height velocity is more than 1 SD below the mean. The patient met both height and height velocity and biochemical criteria for the diagnosis of GH deficiency. GH in the treatment of GH 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 but only went for 1 year and did not show improved final height. It has the advantage of being a weekly medication.
The patient is reported to have needle phobia making a weekly administration of medication with a hidden needle superior to daily administration of medication using the formulary alternative Norditropin. The request is consistent with generally accepted standards of medical practice and is therefore medically necessary.