MMIT Reality Check on Hemophilia B (Factor IX)

Payer Coverage:

A review of market access for hemophilia B (factor IX) treatments shows that under the pharmacy benefit, about 32% of the lives under commercial formularies are covered with utilization management restrictions. Around 18% of the lives under health exchange formularies are not covered for at least one of the drugs.

Under the medical benefit, about 57% of the lives under commercial policies are covered with utilization management restrictions. Almost 75% of the lives under Medicare policies have access to at least one of the drugs without utilization management restrictions.

For about 99% of the covered lives, payer pharmacy benefit formularies do not require step therapy (ST). Of the lives that require ST, about 45% of the lives require multiple steps. Around 48% of payer-controlled pharmacy benefit covered lives require prior authorization, with 3% of those lives covered by policies that are restrictive as compared with a product’s FDA-approved label.

Trends:

2022 Outlook: Notable FDA Approvals, Payer Strategies Are Specialty Trends

Etranacogene dezaparvovec (AMT-061) [from uniQure], an investigational single dose adeno-associated virus five (AAV5)-based gene therapy for the treatment of patients with severe to moderately severe hemophilia B, should be on a payer’s radar and could have a significant financial impact, according to industry experts.

UniQure and CSL Behring Announce Primary Endpoint Achieved in HOPE-B Pivotal Trial of Etranacogene Dezaparvovec Gene Therapy in Patients with Hemophilia B

In December 2021, uniQure and CSL Behring LLC said their investigational adeno-associated virus five-based gene therapy for the treatment of patients with severe to moderately severe hemophilia B, etranacogene dezaparvovec, achieved the pre-specified primary endpoint of non-inferiority in annualized bleeding rate 18 months following administration compared to baseline factor IX prophylactic therapy in the Phase III HOPE-B gene therapy trial. CSL Behring said it planned to submit regulatory applications for marketing approval of the drug to the FDA and European Medicines Agency in the first half of 2022.

FDA Approves Rebinyn

In May 2017, the FDA approved Novo Nordisk A/S’s Rebinyn (coagulation factor IX [recombinant], glycoPEGylated) for use in people at least 12 years old with hemophilia B to provide on-demand treatment and control of bleeding episodes and perioperative management of bleeding.

Key Findings:

Market Events Drive Changes

In May 2017, the FDA approved Novo Nordisk A/S’s Rebinyn (coagulation factor IX [recombinant], glycoPEGylated) for use in people at least 12 years old with hemophilia B to provide on-demand treatment and control of bleeding episodes and perioperative management of bleeding. In March 2016, the agency approved CSL Behring LLC’s Idelvion (coagulation factor IX [recombinant], albumin fusion protein) for on-demand control and prevention of bleeding episodes, management of postoperative bleeding and as a prophylactic treatment for children and adults with hemophilia B.

Competitive Market Landscape

Anticipated product approvals may develop improved product placement based on potential cost, adherence and efficacy advantages. The hemophilia market will be managed more tightly as there is an increase in long-acting products.

Pharmacy, Medical Benefit Implications

While there are very limited instances where products aren’t covered under either the medical and/or pharmacy benefits, there is minimal management of specific products. More pharmacy coverage is seen as products are listed as self-administered specialty drugs. For managed Medicaid, few MCOs will carve out hemophilia products to the state Medicaid fee-for-service program. Coverage is prevalent under Medicare Part B. The majority of the time where prior authorization is defined, diagnosis will be required.

Key Players in Market:

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