Appeal type: Formulary Exception
Decision: Overturned

Diagnosis: Bilateral uveitis and chronic macular edema
Treatment: Pharmacy/Prescription Drugs (Acthar IJ Gel)
The insurer denied Acthar IJ Gel. 
The denial is overturned. 

The patient is a male with bilateral uveitis and chronic macular edema for whom coverage of Acthar Gel is the subject of review. The patient has tried and failed steroids including prednisone, methotrexate, Imuran (azathioprine), CellCept (mycophenolate mofetil), and Humira. The provider reports that the patient is currently on the medication, and Acthar has been very effective with uveitis symptoms. The medication was originally denied as the patient did not have a diagnosis of infantile spasms. An exam documents the patient taking Humira, prednisone, and Acthar. Vision was 20/25- the right eye (oculus dexter [OD]) and 20/30- left eye (oculus sinister [OS]).

At issue is whether generic corticosteroids or standard treatments will be or have been ineffective, would not be as effective as the non-formulary drug, Acthar 80UNIT/milliliter (ML) injection (IJ) Gel, or would have adverse effects.

The health plan's determination is overturned in whole.

According to the package insert, Acthar Gel may be used for the following disorders and diseases: rheumatic, collagen, dermatologic, allergic states, ophthalmic, respiratory, and edematous state. Ophthalmic diseases include severe acute and chronic allergic and inflammatory processes involving the eye and its adnexa such as: keratitis, iritis, iridocyclitis, diffuse posterior uveitis and choroiditis, optic neuritis, chorioretinitis, anterior segment inflammation. In this case, Acthar is proposed for the treatment of bilateral panuveitis with chronic macular edema. Several case reports are identified in the peer reviewed medical literature which support the safety and efficacy of Acthar in the treatment of such non-infectious uveitis.

Agarwal, et al recently published a case report describing the potential role of subcutaneously administered adrenocorticotropic hormone (ACTH) gel in the management of a patient with non-infectious panuveitis with significant retinal vasculitis, macular edema, and optic nerve inflammation. Therapy with ACTH gel resulted in rapid and adequate control or retinal vascular leakage, macular edema, and optic nerve inflammation. At the end of 6 weeks, there were no safety concerns, and no systemic or local adverse events were recorded. The authors encouraged future clinical trials employing repository corticotropin injection in the management of uveitis.

Sharon, et al reported the clinical course of three patients with bilateral, non-infectious anterior and intermediate uveitis, treated with ACTH gel for 12 months. They concluded that subcutaneous ACTH gel has shown to be a safe and effective therapy in the management of non-infectious uveitis. Specifically, ACTH gel plays a role in refractory and steroid-dependent cases and in those who do not respond to or are unable to tolerate other immunomodulatory therapies.

Most recently, Nelson, et al performed a retrospective medical records review of United States (US) patients who had a diagnosis of uveitis, received Repository corticotropin injection (RCI) in the past 12 months, and had completed or were receiving RCI treatment at the time of data collection. Their study included 91 patients. Patients used an average of 2.5 medications before RCI. The author concluded that physicians individualized RCI therapy among patients who suffered uveitis for several years and when previous therapies were inadequate. Most patients improved after initiating RCI, most commonly in vision. The findings support use of RCI for uveitis.

As uveitis is usually treated with a stepwise approach using corticosteroids, immunosuppressive therapy, and biologics, and this patient has already tried and failed corticosteroids and these other standard treatments, and as the patient has demonstrated improved control on the requested medication, the medical necessity of Acthar is established.