Appeal type: Medical necessity
Decision: Upheld

Diagnosis: Immunologic Disorders - Alopecia.
Treatment: Pharmacy/Prescription Drugs - Xeljanz.
The health plan denied the requested prescription drug, Xeljanz, as not medically necessary. 
The health plan's determination is upheld.

The patient is a male with severe alopecia areata (55 percent (%) hair loss of the scalp). The patient was previously receiving Xeljanz (tofacitinib) at a dosage of 5 milligrams (mg). The patient also has seborrheic dermatitis, for which topical clobetasol has been used.
At issue is the medical necessity of Xeljanz, dosed 10 mg twice daily.
The health plan's determination of medical necessity is upheld in whole.
The requested health service/treatment of Xeljanz 10 mg tablet is not medically necessary.
Xeljanz is neither Food and Drug Administration (FDA) -approved nor sufficiently evidence-supported to determine the safe, effective, and proper dosing (with long-term favorable outcomes) to support its use for this patient's clinical condition.
While it is understandable to consider the Janus Kinase (JAK) inhibitor 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 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 [7] only covers Xeljanz for these four conditions.
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.
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.