The U.S. Department of Health and Human Services (HHS) has issued a setback for Bluebird bio’s gene therapy program. In a recent advisory, the HHS inspector general delivered a “negative opinion” regarding the company’s proposal to fund fertility preservation services for patients covered by federal insurance who are undergoing treatment with Zynteglo, Bluebird’s gene therapy for beta thalassemia.
The inspector general’s opinion, released on Monday, suggested that Bluebird’s plan to provide up to $22,500 for fertility preservation might breach anti-kickback regulations. While the opinion does not conclusively determine that Bluebird’s program is in violation, it means that the company will not receive a guarantee of immunity from legal repercussions.
This development represents a significant hurdle for Bluebird’s efforts to expand the use of Zynteglo, which, despite its transformative potential, requires a chemotherapy regimen that poses a high risk of infertility. Bluebird’s initiative to support fertility preservation was intended to mitigate this risk and encourage patients to pursue the treatment.
The advisory affects patients with federal insurance programs such as Medicaid but does not impact those with commercial insurance. Bluebird expressed concern over the ruling, arguing that it could deepen the disparity between patients with federal and commercial insurance. “No patient should have to choose between a potentially life-changing or lifesaving therapy and the chance to have a child,” the company stated.
In addition to Zynteglo, Bluebird sought to extend a similar fertility support program to patients receiving Skysona, another gene therapy for cerebral adrenoleukodystrophy. However, the inspector general noted a lack of data on the costs and accessibility of these services, which prevented a determination of the program’s compliance with anti-kickback rules.
The opinion follows a recent legal challenge by Vertex Pharmaceuticals, which also faces hurdles regarding fertility support for its gene therapy Casgevy. Casgevy, used for sickle cell disease and beta thalassemia, involves similar preconditioning chemotherapy.
A potential pathway for additional data collection is a pilot program initiated by the Centers for Medicare and Medicaid Services. This program will explore various payment models for gene therapies, including those from Bluebird and Vertex, and will require limited fertility support.
Both Bluebird and Vertex have criticized the requirement for limited fertility support, suggesting it conflicts with the HHS Center for Medicare and Medicaid Innovation’s acknowledgment of fertility preservation’s importance in gene therapy access.
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