Appeal type: Medical necessity
Decision: Upheld

Skin Disorders/Other
Pharmacy/Prescription Drugs

Diagnosis: Skin Disorder/ Vitiligo
Treatment: Prescription Drug (Xeljanz)
The health plan denied the requested prescription medication. The health plan's determination is upheld.

The patient is a male with Vitiligo. The patient is documented to have tried and failed topical Clobetasol and Tacrolimus, and pharmacy claims have been seen for Pimecrolimus, Alclometasone, Fluocinnide and Halobetasol. The provider reports having also tried using home/handheld light therapy, as well as systemic steroids and the Vitiligo is spreading.
The medical necessity of Xeljanz (based on its off-label use for Vitiligo) is under review.

The health plan's determination of medical necessity is upheld.

No. There are many unresolved questions with respect to both safety and efficacy for this treatment (Xeljanz) for the condition of Vitiligo, and the relative safety and efficacy (overall unknown) for a skin condition with limited to no functional impairment, supports the conclusions that Xeljanz is not appropriate for this patient's condition outside of the investigational setting.
While understandable to consider Xeljanz for this patient's condition, the requested medication is investigational at this time, and merits further study before being presumed acceptable for widespread clinical use. At this time, neither the short nor long-term safety of this medication, nor its short or long-term efficacy in the use of Xeljanz for this condition, is confirmed outside of the investigational setting. More questions of safety and efficacy exist than any consensus-supported conclusions as to the medical appropriateness of this treatment for Vitiligo.
Vitiligo is notoriously difficult and unpredictable to treat, as well as unpredictable in its response to treatment. Oral immunosuppressants, light therapy, and related therapies have all resulted in variable and unsatisfactory outcomes, and self- or limited resolution is often the only choice a patient has for severe cases.
However, while Xeljanz has some potential of promising preliminary results in open-label studies, the overall literature and reviews on Vitiligo [1-4] mention Xeljanz only as investigational. Xeljanz was denied Food and Drug Administration (FDA) approval for use in psoriasis, in large part for safety reasons, and is not without a significant risk of side effects that preclude an acceptable risk for use outside of the investigational setting for the condition of vitiligo.
At this time, Xeljanz is only FDA-approved for rheumatoid arthritis patients who have failed Methotrexate, and for psoriatic arthritis, and for ulcerative colitis--all three conditions have indications whereby the safety risks of not using treatment are significantly greater than treatment itself. Xeljanz has recently been recommended to be used with lower dosing in rheumatoid arthritis patients.
The same cannot be said for Xeljanz in the treatment of Vitiligo, where physical/functional impairment and overall health impacts are minimal to nonexistent--despite the social/psychological impacts that can occur in some patients. The proper dosing, anticipated side effects and long-term outcomes are not known at this time for Xeljanz in the treatment of Vitiligo, as well as short-term and long-term safety risks of relevance to this patient's overall health. There are no large, randomized, controlled, prospective trials demonstrating and proving the safe and effective treatment of Vitiligo with a specific dosing of Xeljanz.
Therefore, as per the evidence-based literature, Xeljanz is acceptable only within the investigational setting and is not medically necessary for less-monitored outpatient clinical use.