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How Health Plans and Employers Can Prepare for New and Upcoming Gene Therapies

Posted on October 16, 2024

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Two gene therapies, Casgevy and Lyfgenia, were approved late last year by the FDA for treating Sickle Cell Disease (SCD). SCD is a debilitating blood disorder that most commonly impacts individuals of African ancestry. These therapies were and continue to be groundbreaking for multiple reasons, including that they were the first gene therapies to potentially cure a hereditary condition.

Understanding Sickle Cell Disease

SCD produces abnormal red blood cells, becoming sickled or crescent-shaped, leading to vascular occlusion and unpredictable episodes of severe pain. These crises can begin as early as 6-9 months of age and persist throughout a patient’s life. Treatment of SCD crisis could often necessitate hospitalization to manage, especially if there is elevated risk of life-threatening complications such as stroke, severe chest pain, or other organ damage.

The newly approved gene therapies offer the first treatment to address the root cause of SCD by inserting functional genes to produce healthy red blood cells. However, these treatments come with a staggering price tag of over $2 to $3 million per patient. It is crucial to understand that these therapies may not be essential for every patient with SCD to achieve a better quality of life.

The Power of Integrated Medical and Pharmacy Data

With an estimated 100,000 Americans affected by SCD, ensuring appropriate utilization of these costly therapies is critical for healthcare payers. By analyzing a patient’s comprehensive healthcare journey—including over-the-counter and prescription drug use, ER visits, medical facility treatments, and hospitalizations—payers can identify those at the highest risk of severe, costly complications.

Many well-managed SCD patients can lead relatively normal lives with existing treatments, making the multi-million dollar gene therapy potentially unnecessary. Conversely, patients experiencing frequent hospitalizations and disruptions to work or school despite being on treatments could be ideal candidates for these high-cost gene therapies. Preventing a few crises per year can make the therapy cost-effective while dramatically improving a patient’s quality of life. However, this level of insight for healthcare payers is only possible by following a patient’s journey using integrated pharmacy and medical data.

While the arrival of gene therapies for SCD is a promising medical advancement, it requires a nuanced, data-driven approach and robust care management insights to ensure appropriate utilization. By leveraging integrated data to help inform care, we can help SCD patients truly thrive.

The Path Forward

Following FDA approval, only one patient in the United States has been treated with one of these therapies to treat SCD. The process can take up to a year for a patient to go through to receive gene therapy, which includes chemotherapy to destroy faulty stem cells. The patient’s commitment to the journey is an important consideration for gene therapies.  It is estimated that up to 60 patients have begun the process to receive the therapy and further uptake is expected next year.  This is the fundamental challenge for healthcare payers – how to accurately predict the potential exposure within a given population of gene therapies such as Lyfgenia and Casgevy, including projecting the rate of uptake that may occur.

Leveraging Artemetrx GeneCQ™ for Gene Therapy Management

As healthcare payers navigate the complexities of gene therapy management, Artemetrx GeneCQ provides a comprehensive solution for forecasting and managing high-cost gene therapies like Casgevy and Lyfgenia. Artemetrx GeneCQ enables payers to identify potential candidates for gene therapy and project financial impact more precisely by utilizing sophisticated algorithms to analyze clinical, demographic, diagnosis, and medication use data. This data-driven approach ensures both optimal patient outcomes and responsible resource allocation.

Ready to Transform Your Gene Therapy Management?

Download our Artemetrx GeneCQ overview to learn how our platform can help your organization prepare for and manage the impact of revolutionary gene therapies.

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About the Authors

Angela Luong

Angela Luong, PharmD, RPh

Angela has over 12 years of experience in managed care following her doctorate of pharmacy training from the University of Michigan College of Pharmacy.
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Rebekah Gregg, MBA

When Rebekah Gregg began working at a hospice provider after college, she found more than a job—she found a calling. Ever since then, Rebekah’s…
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