Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Crohn's Disease

Treatment: Stelara 90 milligrams (mg) / milliliter (ml)

The insurer denied coverage for Stelara 90 mg/ml

The denial is overturned

 
Patient is a teenager with a past medical history notable for refractory Crohn's disease. His course has been complicated by persistent diarrhea, poor weight gain/maintenance and persistent colonic inflammation. His disease is non-fistulizing and non-structuring. He has been managed with various medications since diagnosis with the following sequence:

-	infliximab - stopped secondary to persistent rectal bleeding
-	Repeated steroid courses bridging between different treatment regimens. 
-	vedolizumab - dose escalated to 300mg every 4 weeks - stopped secondary to failure to respond
-	Methotrexate 15mg weekly added
-	Triple antibiotic therapy - Amoxicillin, Doxycycline, and metronidazole 
-	Stelara - loading dose 300mg with reported "good response"

Last endoscopy moderate to severe chronic active colitis from transverse colon to sigmoid; moderate proctitis. Last labs - white blood cells 6.9, hemoglobin 11.6, platelets 344, C-reactive protein (CRP) 8.6. Stool calprotectin 5000.

This patient's diagnosis is medication refractory Crohn's disease (CD) partially responsive to a loading dose of Stelara. To address Stelara use in pediatric patients from a review on the subject from UpToDate the authors offer the following:

Ustekinumab - Limited data have been reported in children with plaque psoriasis, rheumatoid arthritis, and CD. Observational studies suggest that ustekinumab is efficacious in children with CD unresponsive to anti-tumor necrosis factor (TNF). 

One retrospective case series described outcomes of ustekinumab treatment of 52 pediatric patients with irritable bowel disease (IBD) (80 percent with CD), 81 percent of whom had failed other biologic agents. After 52 weeks of therapy, 75 percent of patients were still on ustekinumab therapy, the majority of whom required dose escalation. Steroid-free remission was achieved in 50 percent of patients who had failed other biologic agents and 90 percent of those who had not been exposed to other biologic agents. A second multicenter retrospective study of 44 patients demonstrated similar results, with 77 percent of patients remaining on ustekinumab for one year and 39 percent of those achieving clinical remission.

Medical necessity of off-label use of medications in pediatrics - Use of second-line biologic agents and/or escalation of doses beyond those approved by the Food and Drug Administration (FDA) may be appropriate and medically necessary for selected children with CD. The decision should be based on individual patient characteristics and clinical judgement by an expert clinician. According to a position statement by the American Academy of Pediatrics: "The purpose of off-label use is to benefit the individual patient. Practitioners use their professional judgment to determine these uses. As such, the term 'off-label' does not imply an improper, illegal, contraindicated, or investigational use. Therapeutic decision-making must always rely on the best available evidence and the importance of the benefit for the individual patient".

Substantial evidence supports a role for dose escalation, therapeutic drug monitoring, and utilization of drugs that are approved for adults in the management of pediatric CD. In many cases of refractory IBD, off-label use is medically necessary and is in the patient's best interest. 

The real-world experience with the use of Stelara in children including dose escalation for the clinical management of Crohn's patients is sufficient to support the practice as supported by the literature. 

Based on the above, the insurer's denial must be overturned. The health care plan did not act reasonably and with sound medical judgment and in the best interest of the patient. The medical necessity for Stelara 90 mg/ml is substantiated.