Medicare moves to improve access to new sickle-cell gene therapies
2024-04-12
上市批准基因疗法ASH会议
The US Centers for Medicare and Medicaid Services (CMS) is proposing to raise reimbursement rates for two recently authorised gene therapies for sickle-cell disease (SCD), including the first CRISPR-based medicine, in a bid to improve access to the potentially curative, though costly treatments.
In its proposed rule for fiscal year 2025, CMS said it will reimburse hospitals 75% of the cost of administering Vertex Pharmaceuticals and CRISPR Therapeutics' Casgevy (exagamglogene autotemcel), as well as bluebird bio's Lyfgenia (lovotibeglogene autotemcel). This exceeds the typical 65% new technology add-on payment (NTAP) hospitals receive for cutting-edge treatments.
Both Casgevy, which uses CRISPR/Cas9 gene-editing technology, and Lyfgenia were approved by the FDA in December for SCD patients 12 years and older. CMS said the enhanced payments are intended to "better promote access" to these cell-based gene therapies, where the list prices are $2.2 million for Casgevy and $3.1 million for Lyfgenia.
According to the proposed rule, the increased reimbursement would last for the two-to-three year "newness" period of the medicines, and would cease once the therapies no longer qualify as new technologies.
The approvals of Casgevy and Lyfgenia were hailed as a major advance, but their hefty price tags raised concerns about patient affordability and access.
"The negotiations happen above my head in these hospital settings, but cost is a problem, obviously," remarked Alexis Leonard, a haematologist and assistant faculty member at St. Jude Children's Research Hospital, in a recent interview with FirstWord. "At the same time, I was at a lot of [American Society of Hematology] sessions for haemophilia and other disorders with genetic therapies that are even more expensive and less effective or at least durable, so it is complicated," she said.
For more, see Physician Views Results: Haematologists' insights on gene therapies for SCD balance promise with healthy dose of pragmatism.
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