Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Idiopathic short stature (ISS)

Treatment: Genotropin

The insurer denied coverage for Genotropin

The denial is overturned 

According to the clinical documentation provided for review, the treating provider is requesting plan approval for treatment with Genotropin (somatropin). The patient has the medical diagnosis of short for age. The patient has been seen and treated for a past medical history including, but not limited to short stature or small for chronological age. The patient presented with a height z-score of -3.1. The patient also has an updated bone age performed at a chronological age of seven years one month, the results were noted to be minimally delayed at six years ten months. This gives the patient a predicted adult height of four feet eight inches. The patient had a normal insulin-like growth factor 1 (IGF-1) and growth hormone (GH) levels. The treating provider prescribed therapy with Genotropin (somatropin).

This patient does not have the diagnosis of growth hormone deficiency (GHD) but has severe short stature with extremely poor height prediction that could lead to issues with daily living such as driving.      

This patient has the diagnosis of ISS. In the United States (US), children who meet Food and Drug Administration (FDA) criteria are considered candidates for GH treatment based on studies that have shown GH treatment increases the mean height of treated cohorts. GH in the treatment of ISS is supported by randomized controlled trials and consensus guidelines. The FDA approval for this indication is for "non-growth hormone-deficient short stature, defined by height standard deviation score (SDS) less than or equal to -2.25, and associated with growth rates unlikely to permit attainment of adult height in the normal range < (less than) 63 inches in boys, 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." This patient did have a pretreatment height less than or equal to -2.25 standard deviations (SD) meeting the diagnosis of ISS criteria, her initial height was at -3 SDS with a very poor height prediction thus it is recommended to start GH for the diagnosis of ISS. 

"In the first description of US children receiving recombinant human growth hormone (rhGH) from one such surveillance study, the National Cooperative Growth Study, 2331 children from 112 centers were started on treatment for GHD and other causes of short stature between October 1985 and October 1987. Seventy percent were male, which the authors suggested reflected an ascertainment bias; septooptic dysplasia was the only category in which boys did not significantly outnumber girls, and among children with idiopathic GHD, the most disparate category (male to female ratio of 2.5:1), girls had greater height deficit at diagnosis (1)." (Reference 2)

"In 2008, 32 leaders in the field of ISS published a consensus statement based on a review of the literature and clinical experience. The consensus was that recombinant growth hormone (rGH) therapy following country-specific regulatory guidelines is appropriate, and that stronger consideration should be given to shorter children. The mean increase in adult height with rGH therapy is 3.5 to 7.5 cm." (Reference 4)

"Conclusions: Thus, short-term therapy with a medium dose of rhGH was found to have a precise effect on prepubertal children with ISS. At this dose, it can significantly increase the growth rate of children and improve the expected adult height without accelerating bone maturation. No serious adverse reactions were found in association with rhGH use during the two-year study period." (Reference 3)                                                       

The health plan did not act reasonably with sound medical judgment in the best interest of the patient.

The insurer's denial of coverage for Genotropin is overturned. Medical Necessity is substantiated.