Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Ulcerative Colitis
Treatment: Stelara 90 mg every four weeks
The insurer denied the Stelara 90 mg every four weeks. 
The denial is overturned. 

The patient is a male with ulcerative colitis. He has failed Entyvio and Remicade.  He is being treated with Stelara 90 milligrams (mg) every eight weeks. He has had some, but not a complete response, with symptoms before eight weeks and the attending physician (AP) is requesting to increase Stelara to 90 mg every four weeks.

Yes, the proposed treatment is medically necessary.

Medical literature supports dose intensification of biologics in patients who lose response to Food and Drug Administration (FDA) approved dosing. Stelara has been found to be safe and effective at doses as high as 90 mg every four weeks. 

Results from a retrospective cohort study of patients with Crohn's disease (CD) reported that among 122 patients on ustekinumab, 21 (17%) patients on every eight week maintenance dosing required dose intensification (frequency increased to every four weeks [n=11], every six weeks [n=5], or a combination of intravenous (IV) booster and frequency increase to every six weeks or every four weeks [n=5]). A total of 11 patients (52%) had clinical response after dose escalation (two [18%] were primary nonresponders to induction while nine [82%] had poor initial clinical response). 

A multicenter, retrospective, open-label cohort study of adults with moderate to severe Crohn's disease was conducted to evaluate the long-term maintenance efficacy of ustekinumab therapy. During the maintenance therapy, dose escalation was required in 16.3% (17/104) patients and a combination strategy of reinduction and dose escalation was required in 6.7% (7/104) of patients. The most common escalated maintenance dosing was 90 milligrams subcutaneously (SC) every four or six weeks. Clinical response was regained in 52.9% (9/17) of patients who had dose escalation, and 57.1% (4/7) of patients who had combined reinduction and dose escalation. 

Results from a retrospective, multicenter, open-label study of patients with refractory Crohn's disease receiving ustekinumab, demonstrated that, among 11 patients who required a dosing interval reduction (includes 90 milligrams subcutaneously every eight weeks to every four weeks [n=8], 90 milligrams subcutaneous every eight weeks to every six weeks [n=1]), clinical improvement was seen in 73% (8/11) of patients. 

Results from a retrospective, open-label, observational study of ustekinumab for the treatment of anti-tumor necrosis factor (anti-TNF) resistant Crohn's disease patients, demonstrated that, among patients who achieved clinical response at the last followup, dose escalation was required in 35% (7/20) of patients who received ustekinumab 90 milligrams subcutaneously every eight weeks. 

While the studies were all done in patients with Crohn's disease, as Stelara is FDA approved for use in ulcerative colitis, the premise is the same, and such dose intensification of Stelara in ulcerative colitis is widely used and accepted by gastroenterologists. Therefore, as dose intensification of Stelara in patients with inflammatory bowel disease (IBD [includes Crohn's disease and ulcerative colitis]) has been found to be safe and effective at doses as high as 90 milligrams every four weeks, the requested Stelara 90 milligrams every four weeks in this patient who has lost response to Stelara 90 milligrams every eight weeks, is medically necessary.