Appeal type: Medical necessity
Decision: Overturned

Diagnosis:  Ulcerative Colitis.
Treatment:  Stelara subcutaneous (SQ) 90 milligrams (MG).

The insurer denied the Stelara subcutaneous (SQ) 90 milligrams (MG).  The denial is overturned. 

The patient is a female with ulcerative colitis.  She had failed treatment with mesalamine, prednisone, Remicade and Entyvio and was started on Stelara 90 milligrams (MG) every eight weeks.  She has had some, but not a complete response, with breakthrough symptoms.  The attending physician is requesting to increase Stelara to 90 milligrams (MG) every four weeks. 

Yes, the proposed treatment with Stelara is medically necessary.

The patient has ulcerative colitis and has lost response to Stelara 90 milligrams (MG) every eight weeks.  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 milligrams (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 (number) =11], every six weeks [n (number) =5], or a combination of intravenous (IV) booster and frequency increase to every six weeks or every four weeks [n (number) =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 (CD) 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 (MG) subcutaneous (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 (CD) receiving ustekinumab, demonstrated that, among 11 patients who required a dosing interval reduction (includes 90 milligrams [MG] subcutaneous [SC] every eight weeks to every four weeks [n (number) =8], 90 milligrams [MG] subcutaneous [SC] every eight weeks to every six weeks [n (number) =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 (CD) 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 (MG) subcutaneous (SC) every eight weeks. 

While the studies were all done in patients with Crohn's disease, as Stelara is Food and Drug Administration (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) has been found to be safe and effective at doses as high as 90 milligrams (MG) every four weeks, the requested Stelara 90 milligrams (MG) every four weeks is clinically appropriate, accepted standard of medical care, and medically necessary, and the health plan did not act reasonably, with sound medical judgment and in the best interest of the patient by denying it.