Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Psoriatic Arthritis
Treatment: Xeljanz XR 11MG (milligrams)
The insurer denied the Xeljanz XR 11MG.
The denial is overturned.

This patient is a female with history of psoriatic arthritis.  The patient has previously tried and failed conventional disease modifying antirheumatic drug (DMARD) methotrexate, oral small molecule DMARD Otezla along with biologic DMARDs Stelara, Cosentyx and Tremfya.  She has been recently treated with a trial of Xeljanz with good clinical results.  The request for Xeljanz was initially denied due to policy not allowing coverage of the requested medication if it is not used in combination with a conventional DMARD such as methotrexate, leflunomide or sulfasalazine.  The reason the provider is appealing the determination is because the patient has been doing well on Xeljanz monotherapy and has already tried and failed methotrexate due to transaminitis.

Yes, the proposed treatment is medically necessary.

The patient has psoriatic arthritis and has previously tried and failed multiple medications.  The patient has been tried on Xeljanz monotherapy and overall has good clinical results.  The patient has difficult to treat psoriatic arthritis as evidenced by the multiple failed conventional DMARDs and biologic DMARDs. Xeljanz was initially denied due to patient not combining Xeljanz with a conventional DMARD.  She has already tried and failed methotrexate for conventional DMARD.  Requiring the patient to add leflunomide (Arava) or sulfasalazine (Azulfidine) before Xeljanz is approved is not definitely going to improve patient outcomes.  A conventional DMARD is not likely to control this patient's symptoms at this time given that she has already tried and failed so many biologic and targeted synthetic DMARDs.  It will likely delay necessary treatment.

Xeljanz prescribing information states that is indicated for the treatment of adult patients with active psoriatic arthritis who have had an inadequate response or intolerance to methotrexate or other DMARDs.  While the clinical trials done with Xeljanz in approval of this medication for treatment of psoriatic arthritis did combine it with a conventional DMARD, it was not studied as a secondary analysis whether this combination with a conventional DMARD was statistically significant in improving patient outcomes.

Since Xeljanz monotherapy has not been previously studied in psoriatic arthritis, a sub-study of OPAL Balance assessed Xeljanz 5 mg (milligrams) twice daily as monotherapy after methotrexate withdrawal versus with continued background methotrexate in patients with psoriatic arthritis.  OPAL Balance is an open-label, long-term extension study of the drug in patients with psoriatic arthritis who had participated in the OPAL Broaden and OPAL Beyond phase 3 trials.  This 12-month methotrexate withdrawal sub-study included 179 patients who had completed at least 24 months of Xeljanz treatment in OPAL Balance and were receiving oral methotrexate before sub-study entry.  Patients remained on open-label Xeljanz 5 mg twice daily and were randomized to receive tofacitinib monotherapy or tofacitinib plus methotrexate for 12 months.  Demographics and baseline characteristics were similar between the groups (mean age, 53.1/51.8 years; female, 52.2 percent/55.1 percent; mean disease duration, 11.3/11.2 years; low disease activity [Psoriatic Arthritis Disease Activity Score 3.2], 70 percent/62.9 percent).  Primary endpoints were changes from sub-study baseline in Psoriatic Arthritis Disease Activity Score (PASDAS) and Help Assessment Questionnaire-Disability Index (HAQ-DI) at six months.

In general, efficacy and patient-reported outcomes were similar between treatment arms at month six and month 12. Rates of minimal disease activity were maintained regardless of methotrexate withdrawal.  Rates of adverse events, serious adverse events, discontinuations due to adverse events, and adverse events of special interest were comparable between treatment arms; laboratory changes were also similar, except that elevations in liver function enzymes were more common with Xeljanz plus methotrexate. Of the patients receiving Xeljanz plus methotrexate, one met monitoring criteria for absolute neutrophil and two met monitoring criteria for absolute lymphocyte counts.

In general, no clinically meaningful differences in efficacy and safety were observed in patients with psoriatic arthritis who received open-label Xeljanz 5 mg twice daily as monotherapy after methotrexate withdrawal versus with continued methotrexate.  No new safety risks were identified.  Therefore the proposed treatment is medically necessary.

No. The health plan did not act reasonably, with sound medical judgment, or in the best interest of the patient.

While FDA (Food Drug Administration) approved prescribing information does suggest Xeljanz in combination with a conventional DMARD because that was how it was initially prescribed in the clinical studies, more recent studies such as the OPAL study as quoted above, does show that the addition of a conventional DMARD is not necessary in the treatment with Xeljanz for treatment of psoriatic arthritis and it doesn't necessarily add benefit for the patient and may just add more risk with potential side effects.  This patient has been doing well currently on monotherapy and therefore should be allowed to continue on monotherapy.  Therefore the health plan did not act reasonably, with sound medical judgment, or in the best interest of the patient.