Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Digestive System/Gastrointestinal/Crohn's Disease
Treatment: Pharmacy/Prescription Drugs - Stelara
The health plan denied the requested medication, Stelara, as not medically necessary. 
The health plan's determination is overturned.

This is a review for the use of Stelara (Ustekinumab) to treat severe Crohn's Disease (CD). This male patient has severe ileocolonic stricturing Crohn's disease. He has previously tried and failed steroids and antibiotics. He was initially doing well on Stelara at standard dosing every 8 weeks but recently lost response as manifested with elevated inflammatory markers (fecal calprotectin). Therefore, the attending provider would like to escalate the dose of Stelara to 90 mg (milligrams) every 4 weeks to treat his severe Crohn's Disease.

At issue is the medical necessity of the requested treatment of Stelara.

I overturn, in whole, the health plan's determination of medical necessity.

Yes, the requested health service/treatment of Stelara SQ (subcutaneous) 90 mg every 4 weeks is medically necessary for this patient. Dose escalation in treating inflammatory bowel disease (IBD) patients who are not responding to standard dose of the medication is a well-accepted practice and is standard of care.

Since this patient was not responding to standard dosing of Stelara, it is reasonable to increase the dose of Stelara to 90 mg every 4 weeks. Dose escalation in biologic therapy used in inflammatory bowel disease can be done either based on clinical symptoms or laboratory markers or endoscopic or radiological features with or without therapeutic drug monitoring. Dose escalation is done either by increasing the dose of the medication or shortening the interval between doses or sometimes a combination of both. There are only a finite number of biological agents used to treat inflammatory bowel disease and the standard of care is to optimize the dose of the biological agent before switching to another agent. Therefore, it is medically necessary to use Stelara 90 mg SQ every 4 weeks to treat this patient's severe Crohn's disease.

While therapeutic levels of Infliximab and Adalimumab are well established in the medical literature, there are less data on appropriate therapeutic levels of Ustekinumab since this medication and therapeutic drug monitoring of Ustekinumab have been available for only a few years as opposed to over a decade for the anti-tumor necrosis factor (TNF) agents. Current guidelines for therapeutic drug monitoring for patients with inflammatory bowel disease address only the anti-TNF agents and not Vedolizumab or Ustekinumab. The current evidence supporting the role of TDM (therapeutic drug monitoring) regarding Ustekinumab is based on two exposure-response relationship studies showing that higher Ustekinumab concentrations correlate to better therapeutic outcomes. At this time, there are still no studies comparing either proactive or reactive TDM with empiric Ustekinumab optimization. Therefore, although there are emerging data that may show an association between drug concentrations and outcomes, they are not sufficient to guide specific induction and maintenance drug concentrations for Vedolizumab and Ustekinumab other than confirming that there is detectable drug.

Crohn's disease results from a pathological immune response to luminal antigens in genetically predisposed individuals. Since the causes of Crohn's disease are complex and only partially understood, treatment is based on the empiric use of anti-inflammatory drugs. Although multiple therapies for Crohn's disease currently exist, a substantial proportion of patients are unresponsive to conventional agents. 

Based on the clinical data provided, Stelara at the escalated dose of 90 mg SQ every 4 weeks is medically necessary.