Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Gynecological - Endometriosis. 
Treatment: Pharmacy/Prescription Drugs - Orilissa. 

The health plan denied the prescription Orilissa as not medically necessary. 
The reviewer has overturned in whole the health plan's determination. 

This is a female with long standing severe endometriosis diagnosed at a young age, at the time of her first surgery. She has had a total of three surgeries and has failed hormonal treatment with oral contraceptive pills (OCP) and Mirena intrauterine device (IUD). Since she began Depo Lupron, she has been successfully treated for many years. The Lupron prior authorization was a denial, however, the appeal was unsuccessful. The patient was prescribed Orilissa (Elagolix). 

While Lupron prevents the production of endogenous hormones centrally, Orilissa blocks the receptors of endogenous hormones peripherally, essentially with the same net effect suppressing endometriosis. Orilissa was denied because the patient has not failed the preferred formulary alternatives of Danazol and Synarel. 

Yes, the requested Orilissa 200 milligrams (mg) tablet is medically necessary for this patient. 

Given the extent and severity of this young woman's disease, every effort to treat her with conservative, fertility sparing, non-surgical therapy should be made available to her. She has either failed hormonal therapy or had therapy known to be successful, Lupron, denied. 

A trial of Danazol is not reasonable. As a synthetic steroid, Danazol has many adverse effects. The most commonly reported include weight gain, gastrointestinal symptoms including bloating, nausea, vomiting, gastroenteritis, elevated liver function tests, joint pain, muscle spasm, lethargy, headache, and depression. Gynecologic side effects such as intermenstrual bleeding, breast atrophy, flushes, and androgenic side effects such as hirsutism, decreased breast size, acne, hair loss, oily skin, oily hair, menstrual irregularities and hoarseness, side effects due to weak mineralocorticoid activity such as swelling, and edema have also been reported. Since this drug is a synthetic steroid, elevated blood pressure can occur and remains an area that merits further investigation, along with its effects on adrenal activity. [1] Side effects such as acne, hirsutism, and weight gain make it a poor option in the face of a newer, better tolerated, more efficacious medication available today. 

A trial of Synarel is not unreasonable. It is a nasal spray gonadotropin-releasing hormone agonist similar to Lupron, it has the potential to be an option, however, it is not commonly used. Both of these medications are not current standard of care medications. 

Pain associated with endometriosis is a burden for women, permeating all aspects of their lives, from their ability to perform daily activities to their quality of life. Although there are many options for endometriosis-associated pain management, they are often limited by insufficient efficacy, inconvenient routes of administration, and/or intolerable side effects. Elagolix, a nonpeptide, small-molecule gonadotropin-releasing hormone (GnRH) receptor antagonist, is the first new oral therapy to be approved for the treatment of endometriosis-associated pain in the United States in more than a decade. Modulation of estradiol with elagolix is dose dependent and ranges from partial to full suppression. Clinical evidence has shown that elagolix at both approved doses (150 mg once daily and 200 mg twice daily) is effective for reducing symptoms of pelvic pain (dysmenorrhea, non-menstrual pelvic pain, and dyspareunia), improving quality of life, and decreasing use of rescue analgesics. The availability of two dosing options allows for individualization of treatment based on baseline clinical factors and response to therapy. Elagolix is well tolerated, with less pronounced hypoestrogenic effects compared with GnRH agonists. [3]