Appeal type: Medical necessity
Decision: Overturned

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

The patient is a female with ulcerative colitis. She has failed Entyvio and Renflexis, and started Stelara 90 mg (milligrams) every 8 weeks. On that dose, she had ongoing symptoms. Fecal calprotectin was elevated at 1122, with a CRP (C-reactive protein) of 3/64 and an ESR (erythrocyte sedimentation rate) of 28. Colonoscopy revealed ongoing active disease. Stelara was increased to 90 mg (milligrams) every 4 weeks.  She is doing well on Stelara 90 mg (milligrams) every 4 weeks. The dose was denied by the health plan, as it is higher than the FDA (United States Food and Drug Administration)-approved dose. The appeal states that discontinuing the medication would put the patient at jeopardy, as she is doing well on that dose.

Yes, the requested Stelara every 4 weeks is medically necessary.
Medical literature supports dose intensification of biologics in patients who lose response to FDA (United States Food and Drug Administration) approved dosing. Stelara has been found to be safe and effective at doses as high as 90 mg (milligrams) every 4 weeks.

Results from a retrospective cohort study of patients with CD (Crohn's disease) reported that among 122 patients on ustekinumab, 21 (17%) patients on every 8 week maintenance dosing required dose intensification (frequency increased to every 4 weeks, every 6 weeks, or a combination of IV [intravenous] booster and frequency increase to every 6 weeks or every 4 weeks). A total of 11 patients (52%) had clinical response after dose escalation (2 [18%] were primary nonresponders to induction while 9 [82%] had poor initial clinical response).

A multicenter, retrospective, open-label cohort study of adults with moderate to severe CD (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) of 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 mg (milligrams) every 4 or 6 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 CD (Crohn's disease) receiving ustekinumab, demonstrated that, among 11 patients who required a dosing interval reduction (includes 90 mg [milligrams] every 8 weeks to every 4 weeks, 90 mg [milligrams] subcutaneous every 8 weeks to every 6 weeks), 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 [tumor necrosis factor]) resistant CD (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 mg (milligrams) every 8 weeks.

While the studies were all done in patients with Crohn's disease, as Stelara is FDA (United States Food and Drug Administration) 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 IBD (inflammatory bowel disease) has been found to be safe and effective at doses as high as 90 mg (milligrams) every 4 weeks, the requested Stelara 90 mg (milligrams) every 4 weeks is clinically appropriate, accepted standard of care and is medically necessary.