Appeal type: Experimental/Investigational
Decision: Upheld

Diagnosis: Alopecia. 
Treatment: Pharmacy/Prescription Drugs. 
The health plan denied the requested prescription drug (Xeljanz). 
The health plan's determination is upheld. 

The patient is a male with alopecia areata (AA), associated with round patches of alopecia on the scalp and beard. The patient has tried and failed topical and intralesional steroids with little benefit. Coverage of Xeljanz is under review for medical necessity. 

The health plan's denial of Xeljanz coverage is upheld, in whole. 

No. Unfortunately there are no standard treatments for this patient's skin condition, and this condition is neither life-threatening nor disabling despite the significant social/psychological impacts upon those affected with this condition. Xeljanz is not, therefore, more beneficial than any other treatment for this patient's condition. 
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, 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 [1-4,7] 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, 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. 

The same cannot be said for Xeljanz in the treatment of alopecia areata, 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 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. 

Tofacitinib should be considered for the treatment of severe AA, alopecia totalis, and alopecia universalis; Tofacitinib dose response will be better defined by randomized controlled trials.[5] 

Tofacitinib is a promising therapy for AA in adolescents. The use of Tofacitinib and other Janus kinase inhibitors for the treatment of AA in this age group should be further evaluated in prospective clinical trials. [6] 

Therefore, as per the evidence-based literature, Xeljanz is acceptable only within the investigational setting and not medically necessary for less-monitored outpatient clinical use.