Appeal type: Experimental/Investigational
Decision: Overturned

Diagnosis: Ulcerative Colitis. 
Treatment: Stelara 90mg/mL Syringe. 

The insurer denied the Stelara 90mg/mL Syringe. 
The denial is overturned. 

This is a male with ulcerative colitis who was started on Stelara every eight weeks earlier in the year.  He has failed Humira, Remicade, and mercaptopurine (6-MP) in the past.  He also failed Entyvio at every four weeks dosing.  He was hospitalized for more than 20 bowel movements per day and required prednisone.  He had diarrhea with blood and abdominal pain.  He was started on Stelara with improvement in bowel frequency and consistency.  However a month after starting Stelara earlier in the year, he reported increase in bowel movements with blood and abdominal pain.  Therefore, Stelara was increased to every four weeks.  He has had improvement in his bowel movements. 

Yes, the health plan should cover the proposed treatment. 

At the American College of Gastroenterology (ACG) Scientific Meeting, results from the induction phase of the Phase III UNIFI study show that treatment with a single intraveous dose of Stelara induced clinical remission in a significantly greater proportion of ulcerative colitis (UC) patients at week eight, compared with placebo, at both doses studied. 15.6% of patients receiving 130 milligrams ustekinumab and 15.5% of patients receiving approximately six milligrams per kilogram (~6 mg/kg) ustekinumab achieved clinical remission, compared with 5.3% of patients receiving placebo). Remission was defined as a Mayo score less than or equal to (</=) two points, with no individual subscore greater than (>) one. Major secondary endpoints including the proportion of patients in clinical response, endoscopic healing, as well as improvement in health-related quality of life, were also significantly higher at week eight among patients receiving Stelara compared with patients receiving placebo. About 50% of study participants are considered biologic refractory, and 17% have a history of inadequate response or intolerance to an anti-tumor necrosis factor (TNF) and/or Entyvio (vedolizumab) (article was later published in the New England Journal of Medicine [NEJM] - Sands BE et al. N Engl J Med.). 

Stelara was approved by the European Commission for use in ulcerative colitis and by the Unites States Food and Drug Administration (US FDA) two years ago. 

Dose escalation of Stelara may be required in some patients; the literature supports its use. In a retrospective multicenter cohort study was performed on patients with crohn's disease (CD), dose escalation was required in 17 patients (16.3%); response was recaptured in 9/17 (52.9%) (Ma C et al, Inflamm Bowel Dis.). In another multicenter open-label study, dose escalation was effective in eight of 11 (73%) patients (Khorrami S et al. Inflamm Bowel Dis.).  In a single center retrospective open-label observational study, dose escalation was required in 47.7% of the patients and was successful in 61.1% of them (Kapylov et al.  J Crohns Colitis.). 

In a retrospective study to determine the effectiveness of ustekinumab dose interval shortening, 506 patients with Crohn's disease (CD) who received subcutaneous ustekinumab 90 milligrams (mg) every eight weeks at a single center was reviewed.  Data from 110 patients who initially received subcutaneous ustekinumab 90 milligrams every eight weeks and then had their interval shortened to every four weeks was studied.  Harvey Bradshaw Index (HBI) scores before and after the dose interval shortening was available for 78 patients in the cohort (71%), levels of C-reactive protein (CRP) for 60 patients (55%), and levels of fecal calprotectin for eight patients (7%). Following dose interval shortening, the patients' median Harvey Bradshaw Index (HBI) decreased from 4.5 to three, the median level of C-reactive protein (CRP) decreased from eight milligrams per liter (mg/L) to three milligrams per liter (mg/L), and median level of fecal calprotectin decreased from 378 gram per gram (g/g) to 157 gram per gram (g/g). Among patients who had an Harvey Bradshaw Index (HBI) greater than ( >) four, a level of C-reactive protein (CRP) five milligrams per deciliter (mg/dL), a level of fecal calprotectin greater than (>) 250 micrograms (ug) per gram, or endoscopic evidence for disease activity before dose interval shortening, after the dose interval was shortened, 28% achieved clinical remission (an Harvey Bradshaw Index [HBI] score four), 22% had a normal level of C-reactive protein (CRP) (less than [<] five milligrams per deciliter), 50% had reduced levels of fecal calprotectin, and 36% achieved endoscopic remission (Ollech JE et al, Clin Gastroenterol Hepatol). 

Yes, the requested health service or treatment is more beneficial than any standard treatment for the insured's condition or disease.