NEW YORK – The Biden Administration on Tuesday unveiled sickle cell disease as the initial focus of the Cell and Gene Therapy (CGT) Access Model, a program of the US Centers for Medicare & Medicaid Services' Innovation Center.
The decision comes on the heels of the US Food and Drug Administration's approval of the first two gene therapies for sickle cell disease, Vertex Pharmaceuticals and CRISPR Therapeutics' Casgevy (exagamglogene autotemcel), priced at $2.2 million, and Bluebird Bio's Lyfgenia (lovotibeglogene autotemcel), priced at $3.1 million, last month.
The CGT Access Model, which will kick off in January 2025, is designed to increase access to cell and gene therapies and lower healthcare costs through outcomes-based agreements.
Specifically, CMS will negotiate agreements with participating drugmakers that tie pricing to whether the treatment improves certain health outcomes for people with Medicaid coverage. Participating state Medicaid agencies will have the option of entering into an agreement with manufacturers based on those negotiated terms.
"By negotiating with manufacturers on behalf of states, CMS can ease the administrative burden on state Medicaid programs so they can focus on improving access and health outcomes for people with sickle cell disease," Liz Fowler, deputy administrator at CMS and director of the CMS Innovation Center, said in a statement.
An estimated 50 to 60 percent of people living with sickle cell in the US are enrolled in Medicaid, according to CMS, and hospitalizations and other health episodes related to the disease cost the US healthcare system almost $3 billion each year. Although gene therapies are expensive, they may reduce long-term spending by improving patient outcomes, the agency said.
As part of negotiations with manufacturers, CMS expects to address care delivery gaps for patients receiving cell and gene therapies, such as requiring drugmakers to include fertility preservation services. CMS will also offer optional funding to states that engage in certain activities providing comprehensive care for patients, such as behavioral health services.
In the future, the CGT Access Model may expand to other types of cell and gene therapies beyond sickle cell treatments.