MMIT Reality Check on Hemophilia B (Factor IX) (2Q2023)

Payer Coverage:

A review of market access for hemophilia B (factor IX) treatments shows that under the pharmacy benefit, about 28% of the lives under commercial formularies are covered with utilization management restrictions. Around 21% of the lives under health exchange formularies are not covered for at least one of the drugs.

Under the medical benefit, about 56% of the lives under commercial policies are covered with utilization management restrictions. Almost 65% of the lives under Medicare policies have access to at least one of the drugs without utilization management restrictions.

For about 98% of the covered lives, payer pharmacy benefit formularies do not require step therapy (ST). Of the lives that require ST, about 48% of the lives require multiple steps. Around 44% of payer-controlled pharmacy benefit covered lives require prior authorization, with 5% of those lives covered by policies that are restrictive as compared with a product’s FDA-approved label.

Trends:

FDA Approves Hemgenix

In November 2022, the FDA approved uniQure N.V.’s Hemgenix (etranacogene dezaparvovec-drlb) for the treatment of people at least 18 years old with hemophilia B who currently use factor IX prophylaxis therapy or have current or historical life-threatening hemorrhage or have repeated serious spontaneous bleeding episodes. CSL will market the gene therapy. The agency gave the drug priority review and orphan and breakthrough therapy designations. The one-time treatment via intravenous infusion will cost $3.5 million.

FDA Approves Hemophilia B Gene Therapy, but Its Cost May Pose Access Problems for Payers

The FDA recently approved the fifth and sixth gene therapies, with four of those decisions coming in the second half of 2022. While payer respondents to a Zitter Insights survey have expressed interest in hemophilia B treatment Hemgenix (etranacogene dezaparvovec-drlb), its price may prove to be an obstacle to coverage.

On Nov. 22, the FDA approved uniQure N.V.’s Hemgenix for the treatment of people at least 18 years old with hemophilia B who currently use factor IX prophylaxis therapy or have current or historical life-threatening hemorrhage or have repeated serious spontaneous bleeding episodes. CSL Behring LLC, a CSL business, will market the gene therapy. The agency gave the first-in-class adeno-associated virus A5-based gene therapy — which previously was known as EtranaDez — priority review and orphan and breakthrough therapy designations.

America’s New Most Expensive Drug Reignites Debate Over How to Price Gene Therapies

The FDA on Nov. 22 approved Hemgenix (etranacogene dezaparvovec-drlb), the first gene therapy for adults with hemophilia B, a genetic bleeding disorder. CSL Behring, the medication’s commercial rights holder, set a list price of $3.5 million, making it the most expensive drug in the U.S.

That price exceeds the $2.96 million health benefit price threshold (HBPB) that the nonprofit Institute for Clinical and Economic Review (ICER) determined recently in an evidence report, meaning it is not fairly priced according to the nonprofit’s guidelines.

Key Findings:

Market Events Drive Changes

In November 2022, the FDA approved uniQure N.V.’s Hemgenix (etranacogene dezaparvovec-drlb) for the treatment of people at least 18 years old with hemophilia B who currently use factor IX prophylaxis therapy or have current or historical life-threatening hemorrhage or have repeated serious spontaneous bleeding episodes. In May 2017, the agency 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.

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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