Appeal type: Medical necessity
Decision: Overturned

Diagosis: Crohn's Disease
Treatment: Stelara
The health plan denied Stelara as not medically necessary. 
The health plan's determination is overturned.

The patient is a male. He has a history of Crohn's disease. He has tried and failed Pentasa, infliximab, and Entyvio. He was started on Stelara at the standard dose, 90 milligrams (mg) every 8 weeks. However, he continued to have active disease, and his Stelara drug level was low. He was escalated to 90 mg every 4 weeks and responded well.
At issue is the medical necessity of the requested health service/treatment of Stelara.
The health plan's determination of medical necessity is overturned in whole.
The requested health service/treatment of Stelara is medically necessary for this patient. Stelara is Food and Drug Administration (FDA) approved for the treatment of Crohn's disease and ulcerative colitis in adults. Despite the lack of FDA approval for pediatric patients, there are many peer reviewed publications supporting the efficacy and safety of Stelara in children with inflammatory bowel disease.
Chavannes M. et al., reported a multi-centered cohort study supporting the safety and efficacy of Stelara in pediatric patients with Crohn's disease. A report by Dayan Jr. et al., showed that ustekinumab (Stelara) is efficacious and safe in pediatric patients with inflammatory bowel disease.
The clinical practice guidelines support the use of Stelara in pediatric Crohn's patients who failed tumor necrosis factor (TNF)-alpha inhibitors (van Rheenen, Patrick F., et al). Therefore, Stelara use in children with Crohn's disease is appropriate and well supported by peer reviewed evidence. However, some patients do not respond adequately to the standard dose of Stelara. Dose escalation is often required in biologics in order to achieve therapeutic levels. There are many peer reviewed literature reports showing that low Stelara levels were associated with poor clinical response. The requested dose of 90 mg every 4 weeks is proven to be effective in patients who fail the standard dose.
Per Ollech, Jacob E., et al.: "Shortening the ustekinumab 90 mg dose interval to 4 weeks for patients with Crohn's Disease (CD) who did not respond to doses every 8 weeks improved clinical and biological indices of disease activity. Patients who lose response to the standard dose of ustekinumab might benefit from dose interval shortening, which was effective and safe." Per Cohen, Erica et al.: "A majority of patients with moderate to severe CD benefited from ustekinumab (UST) dose escalation suggesting the clinical utility of optimizing dosing in the treatment course. " Finally, Yerushalmy-Feler, A., et al. showed that Stelara dose escalation in pediatric Crohn's disease is necessary in many cases with good safety and efficacy.
This patient has a history of refractory Crohn's disease which failed anti-tumor necrosis factor alpha (infliximab) and many other therapies. The patient also failed to respond to Stelara standard dose (90 mg every 8 weeks) and continued to have an active disease. He responded well to every 4 weeks dosing, and his drug level improved. Therefore, continuation of Stelara at 90 mg every 4 weeks is medically necessary.