Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Sickle cell disease (SCD)
Treatment: Oxbryta 500 mg (milligrams) or (oral) tabs (tablets)
The insurer denied Oxbryta 500 mg or tabs. 
The denial is overturned. 

The patient is a female with sickle cell disease (SCD) sickle cell anemia (HbSS) with a history of acute chest syndrome, chronic pain with frequent Vaso occlusive crisis (VOC). Additional past medical history includes asthma, migraine, congestive heart failure (CHF), subperiosteal maxillary/sinus abscess requiring surgery, abdominal hernia repair, and recent coronavirus (COVID) infection treated with monoclonal antibodies.

The patient has dilated cardiomyopathy with hypokinesis with ejection fraction of 45% (percent) on an echocardiogram, and congestive heart failure (HFrEF) and being followed by a cardiologist and medication regimen has been optimized with Carvedilol, Losartan. She also has sickle cell nephropathy with proteinuria treated with Losartan and Carvedilol. Most recent hemoglobin was 7.0, with hematocrit 21.7, reticulocyte count of 9.9, total bilirubin 4.6, and creatinine 0.77. Oxbryta 1000 milligrams (mg) has been recommended by her treating hematologist to improve hemoglobin with goal to improve patient's cardiac and renal compensation.

At issue is the medical necessity of Oxbryta 500 milligrams (mg) oral (or) tablets (tabs).

The health plan's determination is overturned in whole.

Yes, the requested health service/treatment of Oxbryta 500 mg or tabs is medically necessary for this patient.

The health plan denied Oxbryta and suggested the alternative medication hydroxyurea for six consecutive months. Hydroxyurea has the greatest long-term evidence for benefit in reducing acute painful episodes as well as reducing other Vaso-occlusive complications of SCD such as acute chest syndrome and in some cases stroke, decreasing the requirement for blood transfusions, and improving survival. In clinical trials leading to approval of Oxybryta, 35% patients were not receiving Hydroxyurea. The primary goal in this case is to improve hemoglobin to prevent further decompensation of cardiac and renal function. Oxbryta is a hemoglobin S polymerization inhibitor indicated for the treatment of sickle cell disease in adults and pediatric patients 4 years of age and older. The efficacy and safety of Oxybryta in SCD was evaluated in Heart Outcomes Prevention Evaluation (HOPE), a Phase 3 randomized, double-blind, placebo-controlled, multicenter trial. Efficacy was based on hemoglobin (Hgb) response rate defined as a Hgb increase of greater than (>) 1 gram (g)/deciliter (dL) from baseline to week 24 in patients treated with Oxbryta 1,500 mg versus placebo. The response rate for Oxybryta 1,500 mg was 51.1%, compared to 6.5% in the placebo group. This indication is approved under an accelerated approval based on increase in hemoglobin (Hgb). Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).

In this particular case of SCD, the treating hematology provider is requesting Oxbryta to improve hemoglobin to prevent further decompensation of cardiac and renal function. Oxbryta is indicated in this case with a goal to improve the patient's hemoglobin and improve the outcome of SCD, preventing acute congestive heart failure and renal failure.
Therefore, the requested health service/treatment of Oxbryta 500 mg or tabs is medically necessary for this patient.