New sickle cell gene therapies are a breakthrough, but solving how to pay their high prices is a struggle

Biotech
Friday, February 23rd, 2024 4:58 pm EDT

Key Points

  • Approval of two gene therapies offers hope for sickle cell disease patients, primarily affecting Black individuals and people of color.
  • Equitable access to these expensive treatments poses a significant challenge for health officials.
  • Patients, like Michael Goodwin, face unpredictability and hesitancy towards treatments due to intensive preparation and high costs, despite the therapies being hailed as breakthroughs.

The approval of two gene therapies for sickle cell disease offers hope to patients, particularly Black individuals and people of color disproportionately affected by the condition. However, ensuring equitable access to these costly treatments presents a significant challenge for health officials. Despite the breakthrough, patients like Michael Goodwin remain hesitant due to the intensive medical preparation required, including chemotherapy, and concerns about affordability. With therapy costs ranging from $2.2 to $3.1 million, financial burdens loom large for individuals like Goodwin, even with insurance coverage. Dr. Julie Kanter emphasizes that while the therapies are welcomed, only a small percentage of sickle cell patients may opt for them, raising questions about accessibility. To address cost barriers, state and federal officials are negotiating discounts with drugmakers for Medicaid plans, aiming to link payments to patient health outcomes. The Biden administration’s Cell and Gene Therapy Access Model, starting in January 2025, seeks to facilitate access to high-priced treatments. Talks with pharmaceutical companies like Vertex and Bluebird Bio aim to ensure widespread access and mitigate longstanding care inequities. Meanwhile, private employer health plans grapple with covering novel treatments with hefty price tags, prompting exploration of new risk-sharing payment models. Despite efforts to address payment concerns, patients like Goodwin remain cautious, underscoring the need for guarantees regarding treatment outcomes. Vertex and Bluebird Bio are also focused on educating doctors and patient communities about the benefits of their therapies, with the first commercial patients expected to begin treatment soon.

For the full original article on CNBC, please click here: https://www.cnbc.com/2024/02/23/sickle-cell-disease-gene-therapies-casgevy-lyfgenia-insurance-cost-issues.html