Appeal type: Experimental/Investigational
Decision: Upheld

Diagnosis: Alopecia Areata Universalis
Treatment: Xeljanz 10 mg (milligrams) tablet
The insurer denied: Xeljanz 10 mg tablet
The denial is: Upheld

The patient is a male with alopecia areata universalis affecting the hair of his scalp, eyebrows, eyelashes, head, and body. The patient has been well-controlled on Xeljanz (10 milligrams [mg] twice-daily). The patient has tried and failed topical, intralesional, and systemic steroids, as well as psoralen-ultraviolet A (PUVA). The patient has maintained nearly 100% (percent) clearance of his disease while on Xeljanz.

Health plan coverage of further treatment with Xeljanz is under review.

Treatment with Xeljanz is not likely to be more beneficial than any standard treatment for this patient.

While it is understandable to consider Xeljanz (tofacitinib) 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, the long-term safety of this medication is not as confirmed as short-term efficacy in the use of Xeljanz for this condition.

Alopecia areata is notoriously difficult and unpredictable to treat, as well as unpredictable in its response to treatment. Oral immunosuppressants, light therapy, contact allergen treatment and related therapies have all resulted in variable and unsatisfactory outcomes, and self-resolution is often the only choice a patient has (along with hairpieces) for severe cases.

However, while Xeljanz has some promising preliminary results in open-label studies [5, 6], the overall literature and reviews on alopecia areata [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 alopecia areata.

At this time, Xeljanz is only FDA-approved for rheumatoid arthritis patients who have failed methotrexate, for juvenile idiopathic arthritis, for psoriatic arthritis, for ankylosing spondylitis, and for ulcerative colitis--all five conditions have indications whereby the safety risks of not using treatment are significantly greater than treatment itself. Accordingly, the health plan policy only covers Xeljanz for these four conditions. There is no significant history rheumatoid arthritis failing methotrexate to support medical necessity for Xeljanz for that indication. On a related note, Xeljanz has been recommended to be used with lower dosing in rheumatoid arthritis patients because of concerns about excessive blood clotting.

The same cannot be said for Xeljanz in the treatment of alopecia areata, where physical/functional impairment and overall health impacts are relatively 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 alopecia areata, 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 alopecia areata with a specific dosing of Xeljanz.

However, if the concerns of quality of life impacts of this patient's alopecia areata are of concern to the provider and patient (and it to be noted that alopecia areata universalis is not just "cosmetic" but disfiguring and potentially threatening towards one's ability to find employment and interact with society), it should be pointed out that another Janus Kinase inhibitor, Olumiant (baricitinib), has been FDA-approved for moderate to severe alopecia areata. Unlike Xeljanz, Olumiant has demonstrated safety and efficacy that support use as a viable treatment option for patients with this challenging disease [7,8].
Therefore, as per the evidence-based literature, Xeljanz is acceptable only within the investigational setting and not appropriate for less-monitored outpatient clinical use.