Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Alopecia Areata
Treatment: Compounded ointment made with Xeljanz
The insurer denied the compounded ointment made with Xeljanz.
The denial was overturned.

This is a patient with a history of alopecia areata. It was reported that the patient has tried and failed Clobetasol gel and topical Minoxidil. 

The patient's alopecia areata has been resistant to several first and second-line treatments. She now has signs of cutaneous atrophy due to long-term use of corticosteroids, and all reasonable commercially available options have been exhausted. The patient has been experiencing significant adverse psychological consequences as a result.

According to Putterman (2018). "Clinical trials have shown benefit from oral Janus kinase (JAK) inhibitors in adults with AA.2 Potential adverse effects, such as increased risk for infection and myelosuppression, make oral formulations less appealing for pediatric patients. It is presumed that topical JAK inhibitors have minimal adverse effects, the main one being irritation in the area of application. Considering these small molecules are poorly soluble in water, JAK inhibitors might be most effective when delivered in a liposomal base, which often consist of phospholipids mixed with water... The average change in Severity of Alopecia Tool (SALT) score was a reduction of 32.3%. Eight of 11 patients demonstrated an improvement in SALT score (Supplemental Fig 1, A-C; available at http://www.jaad.org). Three had cosmetically acceptable regrowth.6 Hair growth was considered cosmetically acceptable if it was sufficient to cover the scalp or able to conceal residual areas of hair loss.6 All patients tolerated treatment without adverse effects, which were recorded at each visit on the basis of a standard review of systems screening for new fatigue, infection, headache, rash, and gastrointestinal symptoms. One individual reported application-site irritation. Blood levels of tofacitinib were not obtained... These limited results suggest that topical tofacitinib might be a reasonable adjunct or second-line therapy for pediatric patients with AA, AT, and AU for whom systemic therapies are not desired."

Per Liu, L. Y., & King, B. A. (2018), "Alopecia areata (AA) is an autoimmune disease affecting people of all ages. There is currently no cure for AA, and a highly efficacious therapy for severe AA has been elusive. Recently, scientific advances have identified the Janus kinase pathway as a target for treatment. Both Janus kinase inhibitors approved by the US Food and Drug Administration, tofacitinib and ruxolitinib, have shown promise in open-label clinical trials."

Per Kennedy Crispin, M., Ko, J. M., Craiglow, B. G., Li, S., Shankar, G., Urban, J. R.,  King, B. A. (2016), "Of 66 subjects treated, 32% experienced 50% or greater improvement in SALT score. AA and ophiasis subtypes were more responsive than AT and AU subtypes. Shorter duration of disease and histological peribulbar inflammation on pretreatment scalp biopsies were associated with improvement in SALT score. Drug cessation resulted in disease relapse in 8.5 weeks. Adverse events were limited to grade I and II infections. An AA responsiveness to JAK/STAT inhibitors score was developed to segregate responders and nonresponders, and the previously developed AA disease activity index score tracked response to treatment."

Systemic and topical Xeljanz has been found to be safe effective in durable in the treatment of alopecia areata. Since the patient has tried and failed conservative therapy the request for Xeljanz topical should be approved. There is the potential for solid organ or bone marrow adverse effects with systemic therapy which can lead to lifelong adversity.                
                                         
The health care plan did not act reasonably and with sound medical judgment and in the best interest of the patient.      

The patient has tried and failed topical and injectable corticosteroids and minoxidil. Xeljanz has been found to be safe, effective and durable in the treatment of alopecia areata. Therfore, the compounded ointment made with Xeljanz is considered medically necessary for this patient.

Based on the above, the medical necessity for compounded ointment made with Xeljanz is substantiated.  The insurer's denial is overturned.