MMIT Reality Check on Hemophilia A (Factor VIII) (4Q2023)

Payer Coverage:

A review of market access for hemophilia A (factor VIII) treatments shows that under the pharmacy benefit, about 43% of the lives under commercial formularies are covered with utilization management restrictions. Around 23% of the lives under health exchange formularies are not covered for at least one of the drugs.

Under the medical benefit, about 65% of the lives under commercial policies are covered with utilization management restrictions. Almost 67% of the lives under Medicare Part B policies have access to at least one of the drugs without utilization management restrictions.

For about 80% of the covered lives, payer pharmacy benefit formularies do not require step therapy (ST). Of the lives that require ST, 10% require multiple steps. Around 52% of payer-controlled pharmacy benefit covered lives require prior authorization, with 52% of those lives covered by policies that are restrictive as compared with a product’s FDA-approved label.

Trends:

FDA Approves Roctavian

In June 2023, the FDA approved BioMarin Pharmaceutical Inc.’s Roctavian (valoctocogene roxaparvovec-rvox) for the treatment of adults with severe hemophilia A without antibodies to adeno-associated virus serotype 5 (AAV5) detected by an FDA-approved test. The one-time infusion is the first FDA-approved gene therapy for severe hemophilia A. The therapy’s price is $2.9 million.

PBMs Weigh Coverage of New Muscular Dystrophy, Hemophilia A Gene Therapies

In June 2023, the FDA approved two new gene therapies for Duchenne Muscular Dystrophy (DMD) and hemophilia A, and major PBMs tell AIS Health, a division of MMIT, that they have not yet decided how to cover the new treatments. If current trends are any indication, health plans will impose strict utilization management requirements and attempt to negotiate outcomes-based reimbursement pacts with the treatments’ manufacturers.

FDA Approves Altuviiio

In February 2023, the FDA approved Sanofi and Sobi’s Altuviiio (antihemophilic factor [recombinant], Fc-VWF-XTEN fusion protein-ehtl) for routine prophylaxis and on-demand treatment to control bleeding episodes, as well as perioperative management of bleeding episodes, for people with hemophilia A. Previously referred to as efanesoctocog alfa, the agent is a first-in-class, high-sustained factor VIII replacement therapy. The FDA gave the drug priority review, as well as breakthrough therapy, fast track and orphan drug designations. Dosing for routine prophylaxis is 50 IU/kg once weekly via intravenous infusion, while dosing for on-demand treatment and perioperative management is 50 IU/kg. Sanofi said the agent will be priced at parity to the annual price of prophylaxis treatment with Eloctate (antihemophilic factor [recombinant], Fc fusion protein), also a Sanofi/Sobi collaboration. According to Anton Health, LLC, Eloctate is priced at “$12 per unit or a cost of $12,000 for a patient requiring 1,000 IUs weekly or $625,000 annually. Some patients may require dosing as high as 3,000 IUs weekly resulting in an annual upper end cost of $1.8 [million].”

Key Findings:

Market Events Drive Changes

In June 2023, the FDA approved BioMarin Pharmaceutical Inc.’s Roctavian (valoctocogene roxaparvovec-rvox) for the treatment of adults with severe hemophilia A without antibodies to adeno-associated virus serotype 5 (AAV5) detected by an FDA-approved test. The one-time infusion is the first FDA-approved gene therapy for severe hemophilia A. In February of the same year, the agency approved Sanofi and Sobi’s Altuviiio (antihemophilic factor [recombinant], Fc-VWF-XTEN fusion protein-ehtl) for routine prophylaxis and on-demand treatment to control bleeding episodes, as well as perioperative management of bleeding episodes, for people with hemophilia A.

Competitive Market Landscape

There are several promising drugs in the pipeline that will add a significant benefit to treatment of patients with hemophilia A and B with and without inhibitors. The market will be managed more tightly when there is an increase in longer acting products and gene therapy products in the pipeline. For the most part, hemophilia products are viewed as undifferentiated brands.

Pharmacy, Medical Benefit Implications

While there are very limited instances where products are not covered under either the medical and/or the pharmacy benefit, there is minimal management of specific products. More pharmacy coverage is seen as products are listed as self-administered specialty drugs.

Key Players in Market:

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