Appeal type: Experimental/Investigational
Decision: Overturned

Diagnosis: Alopecia Universalis
Treatment: Xeljanz
The insurer denied Xeljanz.
The determination is upheld.

This is a male patient with alopecia areata universalis, with hair loss including his entire scalp, eyelashes and eyebrows. The patient has tried and failed topical steroids, tofacitinib and minoxidil. At issue is whether Xeljanz 5 milligram (mg) tablet is likely to be more beneficial than any standard treatment or treatments for the insured's life threatening or disabling condition or disease.
Xeljanz 5 mg Tablet is not likely to be more beneficial than any standard treatment or treatments for the insured's life threatening or disabling condition or disease.
While it is understandable to consider Xeljanz (tofacitinib) for this patient's condition, and the life-altering and psychologically dramatic impacts of this child's condition are not to be underestimated, the requested medication 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. Threatening the physical health of this patient is an even greater threat to this patient's well-being with a potentially long-term dangerous and unproven drug than the alopecia areata universalis itself.
Alopecia areata is 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. Furthermore, the more dramatic and extensive this condition is in very young patients, the likelihood of spontaneous hair regrowth is reduced in a similar fashion.
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. 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.
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. There is no significant history of rheumatoid arthritis failing methotrexate to support  Xeljanz for that indication.
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.

The long-term future overall appears to be bright for JAK inhibitors for conditions such as alopecia areata, but in particular for very young patients such as this patient, and with the concerns surrounding safety of Xeljanz crossing over into other JAK inhibitors as well, the short-term future is dramatically incomplete with respect to a form of effective, reliable, but also safe treatment for patients such as this one.