Appeal type: Medical necessity
Decision: Upheld

Diagnosis: Crohn's Disease. 
Treatment: Skyrizi (Risankizumab-rzaa), procedure code J2327. 
The insurer denied coverage for Skyrizi (Risankizumab-rzaa), procedure code J2327. 
The denial is upheld. 

The patient has a past medical history notable for inflammatory disease (Crohn's disease). The patient's presenting symptoms at that time included abdominal pain, weight loss and growth/puberty delay. The patient's stool calprotectin was 1297. The patient underwent an upper and lower endoscopy notable for numerous esophageal ulcers and terminal ileal mucus and deep ulcerations. A magnetic resonance (MR) enterography reportedly showed inflamed 50 centimeters (cm) of terminal ileum. The patient was treated with infliximab with initial response that was lost after conversion to a biosimilar. The patient was transitioned to Humira and escalated to weekly dosing with clinical remission. However, a video capsule showed active ileal disease. On an office visit the patient was without symptoms and their weight was 62.6 kilograms and height was 163.3 centimeters. The patient reportedly has developed loose stools without blood but averages one bowel movement per day. In the setting of an overseas trip the patient was treated with a course of budesonide without improvement. 

The patient's treating gastroenterologist is requesting off-label treatment with Skyrizi based on adult study results, dosing and Food and Drug Administration (FDA) approval. The justification for this agent after failure of tumor necrosis factor (TNF) inhibitors in this patient is the selective interleukin-23 (IL-23) blockade mechanism of action, perceived superior response rates to Skyrizi in TNF-experienced patients, the potential for stricturing disease, and the treating physician's unpublished clinical experience. 

The insurer is denying coverage for Skyrizi since the patient is less than 18 years of age as per the insurer's policy on monoclonal antibodies to IL-23. The issues to be addressed are whether the denial was appropriate and whether this agent is medically necessary for this patient. 

This patient's diagnosis is medication refractory Crohn's disease of the terminal ileum. The denial appears to be principally based on the patient's age. 

One retrospective case series described outcomes of ustekinumab treatment of 52 pediatric patients with 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. 

This review was based on the records submitted for review and the review of the medical treatment of Crohn's disease in children and adolescents from UpToDate cited above. 

Based on the above, the insurer's denial must be upheld. The health care plan did act reasonably and with sound medical judgment and in the best interest of the patient. 

The medical necessity for Skyrizi (Risankizumab-rzaa), procedure code J2327 is not substantiated.