Appeal type: Medical necessity
Decision: Upheld

Diagnosis: Ulcerative Colitis.
Treatment: Xeljanz 10MG (milligram) Tablet.
The insurer denied: Xeljanz 10MG (milligram) Tablet.
The denial is upheld.

This patient is a young adult male with refractory ulcerative colitis for whom the provider is requesting Xeljanz, ten milligrams orally, twice daily. The provider is requesting continuation of therapy with Xeljanz to be used along with Stelara. The patient has previously Flagyl, Apriso, and Remicade. The patient started Xeljanz in last year but had ongoing elevations of the fecal 
calprotectin. Colonoscopy showed chronic active colitis throughout the colon. At that time, it was recommended that the patient start dual therapy with Xeljanz and Stelara. Since starting 
combination therapy, the patient has had great improvement, and quality of life has improved.

The proposed treatment is not medically necessary.

Xeljanz (tofacitinib) is an oral inhibitor of Janus kinases I, II, and III, that is Food and Drug Administration (FDA) approved for the treatment of adults with moderately to severely active ulcerative colitis. 

In a double-blind, placebo-controlled, phase II trial, 194 adults with moderately to severely active ulcerative colitis were randomly assigned to receive tofacitinib at a dose of 0.5 milligrams, three milligrams, ten milligrams, or 15 milligrams or placebo twice daily for eight weeks. The primary outcome, clinical response at eight weeks, occurred in 32%, 48%, 61%, and 78% of patients receiving tofacitinib at a dose of 0.5 milligrams (P=0.39), three milligrams (P=0.55), ten milligrams (P=0.10), and 15 milligrams (P1). 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. (Sands BE, et al. Presented at the American College of Gastroenterology (ACG) Annual Scientific Meeting, Philadelphia, Pennsylvania. 510 October 2018.). 

In a study by Sands BE et al., ustekinumab as eight-week induction therapy and 44-week maintenance therapy in patients with moderate-to-severe ulcerative colitis, was studied. A total of 961 patients were randomly assigned to receive an intravenous induction dose of ustekinumab (either 130 milligrams [320 patients] or a weight-range-based dose that approximated six milligrams per kilogram of body weight [322]) or placebo (319). Patients who had a response to induction therapy eight weeks after administration of intravenous ustekinumab were randomly assigned again to receive subcutaneous maintenance injections of 90 milligrams of ustekinumab (either every 12 weeks [172 patients] or every eight weeks [176]) or placebo (175). The percentage of patients who had clinical remission at week eight among patients who received intravenous ustekinumab at a dose of 130 milligrams (15.6%) or six milligrams per kilogram (15.5%) was significantly higher than that among patients who received placebo (5.3%) (P) 95% of patients in symptomatic remission at Week 92 were corticosteroid-free (Panaccione R, et al. Aliment Pharmacol Ther. 2020 Dec). 

Dual biologic therapy may be considered in extenuating circumstances in which case patients fail monotherapy with all Food and Drug Administration (FDA) approved biologic agents or small molecule agents. This patient was failing Xeljanz and then started Stelara (as dual therapy used along with Xeljanz). The patient did not try / fail monotherapy with Stelara. Given this, it cannot be ascertained that the patient is benefiting from the dual therapy rather than the effects of Stelara alone. The patient's response to Stelara / Xeljanz dual therapy may reflect the patient's response to Stelara. Therefore, the patient does not meet extenuating circumstances as accepted in the standard of care for the request of Xeljanz to be used as part of dual therapy with Stelara.