Appeal type: Experimental/Investigational
Decision: Overturned

Nature of Statutory Criteria/ Case Summary:  The enrollee requested the medication, tofacitinib, (Xeljanz) for alopecia areata. Her dermatologist noted she developed diffuse and rapid shedding of scalp hair late last year. Her hair loss progressed despite steroid injections, prednisone, clobetasol propionate, ketoconazole shampoo, Rogaine, iron, and biotin supplements. Her dermatologist noted significant shedding and loss bitemporally; consistent with severe and progressive alopecia areata. She was prescribed tofacitinib and has noticed some hair growth to her scalp and stability of her hair loss. Her dermatologist indicates the patient will need to remain on the medication for at least six to nine months to realize its full effect; a positive result will necessitate long-term use of the medication. The record indicates the patient has suffered from depression and anxiety as a result of her hair loss. Xeljanz is likely to be more beneficial for this patient as there are no FDA-approved therapies for alopecia areata. Immunosuppressant medication, such as methotrexate, is inappropriate for long-term use in females; especially those of reproductive age, because of potential organ and bone marrow toxicity, which could have negative life-long effects. The use of immunosuppressive medications, such as methotrexate has not been found to be consistently effective. However, Xeljanz has been found to be safe and effective in the treatment of alopecia areata. Alopecia areata (AA) is a highly prevalent autoimmune disease in the US. However, treatment for AA remains a significant unmet need in dermatology. Janus kinase inhibitors are emerging as potential therapies for many autoimmune conditions; including AA. Current treatments for severe cases include broad immune-suppressants, which are associated with significant adverse effects, precluding long-term use. Rapid hair loss follows treatment termination. Because the disease is extensive in severe cases, topical contact sensitizers and intralesional treatments are of limited use. Tissue analyses of both animal models and human lesions following broad-acting and cytokine-specific therapeutics, such as Janus kinase inhibitors and ustekinumab, provide an opportunity for important insights into the pathogenesis of AA. Numerous novel therapeutics are undergoing clinical trials for AA, emphasizing the potential transformation of the clinical practice of AA, which is currently lacking. Since Xeljanz has shown benefit and data is lacking due to small sample sizes, Xeljanz is likely to be more beneficial for this patient.Final Result: Two of the three reviewers determined that the requested services are likely to be more beneficial than any available standard therapy for treatment of the enrollees medical condition. Credentials/Qualifications: Three reviewers are board certified in dermatology and are actively practicing.  All three reviewers are experts in the treatment of the enrollees medical condition and knowledgeable about the proposed treatment through recent or current actual clinical experience treating those with the same or a similar medical condition.