MMIT Reality Check on Hemophilia A (Factor VIII) (October 2022)
A review of market access for hemophilia A (factor VIII) treatments shows that under the pharmacy benefit, about 37% of the lives under commercial formularies are covered with utilization management restrictions. Around 19% of the lives under health exchange formularies are not covered for at least one of the drugs.
Under the medical benefit, about 59% of the lives under commercial policies are covered with utilization management restrictions. Almost 73% of the lives under Medicare Part B policies have access to at least one of the drugs without utilization management restrictions.
For about 84% 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 48% of payer-controlled pharmacy benefit covered lives require prior authorization, with 45% of those lives covered by policies that are restrictive as compared with a product’s FDA-approved label.
2022 Outlook: Notable FDA Approvals, Payer Strategies Are Specialty Trends
Inflammatory conditions, cancer, hemophilia, multiple sclerosis and Alzheimer’s disease are therapeutic classes that are likely to see multiple significant new therapies approved in the near future, according to Dea Belazi, Pharm.D., M.P.H., president and CEO of AscellaHealth. “Nine hemophilia drugs are projected to receive FDA approval between 2022 and 2023. Five of these therapies are indicated for just hemophilia A, two are indicated for just hemophilia B, and two are indicated for hemophilia types A and B,” he adds.
Genentech Hemophilia A Drug Displays Long-Term Safety, Efficacy
A late-stage study of Roche Group member Genentech USA, Inc.’s Hemlibra (emicizumab-kxwh) reported no new cases of thrombotic microangiopathy or serious thrombotic events associated with the drug, which was first approved by the FDA in 2017 to treat hemophilia A with factor VIII inhibitors. The study also showed that Hemlibra over the long-term follow-up effectively delivers bleeding control.
FDA Gives Additional Approval to Wilate
In October 2019, the FDA expanded the label of Wilate (von Willebrand factor/coagulation factor VIII complex [human]) to include the treatment of adults and adolescents with hemophilia A for routine prophylaxis to reduce the frequency of bleeding episodes and for on-demand treatment and control of bleeding episodes. The agency initially approved the Octapharma USA intravenous injection in 2009.
Market Events Drive Changes
In October 2019, the FDA expanded the label of Octapharma USA’s Wilate (von Willebrand factor/coagulation factor VIII complex [human]) to include the treatment of adults and adolescents with hemophilia A for routine prophylaxis to reduce the frequency of bleeding episodes and for on-demand treatment and control of bleeding episodes. In February 2019, the FDA approved Novo Nordisk’s Esperoct (turoctocog alfa pegol) for use in children and adults with hemophilia A for routine prophylaxis to reduce the frequency of bleeding episodes, on-demand treatment of episodes and perioperative management of bleeding.
Competitive Market Landscape
The market is trending toward longer half-life recombinant products. Anticipated product approvals may develop improved product placement based on potential costs, adherence and efficacy advantages. The market will be managed more tightly as we start seeing an increase in longer acting products and gene therapy products in the pipeline. For the most part, hemophilia products are viewed as undifferentiated brands. The FDA is expected to make decisions on Biologics License Applications (BLAs) for two agents in 2023: Sanofi and Sobi’s efanesoctocog alfa has an action date of Feb. 28, and BioMarin Pharmaceutical Inc.’s Roctavian’s (valoctocogene roxaparvovec) action date is March 31.
Pharmacy, Medical Benefit Implications
While there are very limited instances where products are not covered under either the medical and/or the 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 programs. Coverage is prevalent under Medicare Part B.
Key Players in Market: