Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Hemophilia
Treatment: Hemlibra
The insurer denied Hemlibra.
The determination is overturned.

This is a male patient with a diagnosis of hemophilia A. He is classified as having moderate to severe factor VIII deficiency, with a factor VIII level around 2 percent (%). He has had many spontaneous bleeds in the past, approximately 3 to 4 per year, despite the use of factor VIII replacement. He has been treated with Hemlibra for the past year, with great success, and has had minimal breakthrough bleeding episodes. His past medical history is also significant for a recent diagnosis of thyroid cancer, requiring surgical treatment. He has been using factor VIII replacement at least weekly prior to starting on Hemlibra, primarily prophylactically before physical activity, but also for treatment of injuries. Specifically, he has been taking Advate 3000 units daily as needed for bleeds. He was started on Hemlibra. The continuation of Hemlibra therapy is currently under review for medical necessity.
The requested health service/treatment of Hemlibra is medically necessary for this patient.
This patient has a diagnosis of moderate to severe hemophilia A. His provider has requested Hemlibra therapy to be continued as treatment of his condition.
 
As per recent hemophilia treatment guidelines, prophylaxis is considered the standard of care for people with severe hemophilia, and for some people with moderate hemophilia. These include those that are considered to be at high risk of spontaneous life-threatening bleeding. Episodic factor replacement should not be considered a long-term option for the management of hemophilia, as it does not alter its natural history of spontaneous bleeding and related complications. Musculoskeletal complications in particular can lead to significant comorbidities, and long-term functional deficits.
Specifically, the World Federation of Hemophilia recommends that Hemlibra be used for regular prophylaxis for all patients with hemophilia A with no inhibitors. This product has been associated with very low rates of bleeding, lower than that which this patient previously reported while on prophylaxis with other factor products. The guideline again iterates that for patients with moderate hemophilia A, especially those who have experienced significant and/or life-threatening bleeds, product such as Hemlibra should be used in order to prevent a recurrent significant bleed.
As per the National hemophilia foundation standard of care guidelines, documents 267 and 268, the use of Hemlibra for prophylaxis should be considered by physicians caring for persons with hemophilia A of any age or severity, with or without inhibitors, based on the individual situation, and need for prophylactic therapy.
In regards to this individual patient, this patient has moderate hemophilia A with severe clinical phenotype, and has experienced breakthrough bleeds on factor VIII replacement therapy in the past. He has had multiple bleeds, and is at risk for significant orthopedic dysfunction in the future. Hemlibra prophylaxis appears to be the most clinically appropriate step in his management going forward.
Therefore, based on the clinical information provided for review, the current standard of care in the field, and the current medical literature, the use of Hemlibra as requested is medically necessary at this time.