The most expensive drug in the world: Ensuring access and managing cost 

January 12, 2023

The second half of 2022 was marked by significant activity in the gene therapy market, with several landmark FDA approvals, including Hemgenix, a $3.5M gene therapy indicated for treatment of Hemophilia B. With this hefty price tag, Hemgenix wins the title of most expensive drug in the world, knocking down the previous title holder, the $2.1M gene therapy called Zolgensma, indicated for spinal muscular atrophy.

What is Hemophilia?

Hemophilia is a rare, usually inherited, bleeding disorder characterized by insufficient blood clotting which may lead to easy bruising and potentially life-threatening bleeding. If bleeding is not controlled, patients may also bleed into joint spaces and may develop degenerative joint disease and osteoarthritis. Hemophilia is divided into two main types: A and B, depending on the specific blood clot factor that is deficient. Hemophilia B is considered a rarer and more severe form of this disease as compared to Hemophilia A.

Traditional hemophilia treatment, administered on-demand and/or on a preventive basis, replaces the missing blood clotting factors. While clinically effective, traditional treatment may also be costly, sometimes exceeding $500,000 per year for members with more severe forms of the disease.

Gene therapy for Hemophilia

Gene therapies are novel drug treatments that modify or replace missing or faulty genes, thereby potentially curing or significantly improving a chronic genetic disease. These cutting-edge treatments promise to fundamentally change the natural course of the illness, offering hope to many individuals.

Approved by the FDA in November 2022, Hemgenix is the first hemophilia gene therapy to hit the market. It is indicated for the treatment of adults with Hemophilia B, which impacts approximately 1 in 30,000 live male births. Hemgenix is intended to be a one-time potentially curative treatment; it is administered as an intravenous infusion in a specialized healthcare setting. Among other clinical criteria, potentially eligible patients must undergo screening for presence of certain antibodies called factor IX inhibitors, which may render the therapy less effective. Patients who test positive for inhibitors are not eligible for Hemgenix treatment. Long term efficacy data is not available yet for Hemgenix and it is possible that individuals may still need to use some clotting factor product after Hemgenix.

Additional gene therapies for both Hemophilia A and B are currently in development, with clinical trials showing promising results. One of the most notable products nearing potential FDA approval is Roctavian indicated for treatment of severe Hemophilia A. This gene therapy may be approved and launch in early 2023 with an anticipated price tag of $2-$3M per one-time potentially curative dose. Some healthcare providers believe that Roctavian may not be a fully curative treatment for all patients requiring some patients who receive Roctavian to continue utilizing infused factor therapies.

Considerations and strategies to explore

Hemophilia patients already benefit from various effective treatment options and life expectancy with traditional treatment is relatively long. Thus, it is hard to predict whether there will be strong initial uptake for these new therapies.

Gene therapies are administered by healthcare professionals in specially designated treatment facilities; they require high-touch clinical support for optimal health outcomes and are typically billed through the medical benefit. In addition to the high cost of the drug itself, substantial administration and procedural fees may also come into play. Given that long-term data showing outcomes is still limited, it’s important to recognize the potential for an incomplete clinical response or even treatment failure.

As Hemgenix and other high-cost gene therapies enter the market, employers should create a long-term comprehensive approach to managing these therapies from a clinical and cost perspective by exploring a broad spectrum of strategies. A key first step in tailoring strategies specific to your plan involves assessing the likelihood of these claims occurring in your plan’s population; ideally, such assessments should be conducted on a regular basis as the member population changes. Once you get a better understanding of your unique population and the potential risk for these claims, inventory and evaluate available vendor strategies for gaps and opportunities. This step may include reviewing your medical carrier’s utilization management programs, network strategy, and care management programs, checking for availability of outcomes-based reimbursement and other payment models, and exploring alternative approaches to funding these claims. Lastly, as the market continues to evolve, regularly engage with your medical and pharmacy vendor on availability of new strategies.

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