CMS: Cell and gene therapy model to focus first on sickle cell treatments

Expect sickle cell disease to be the primary focus of the new Cell and Gene Therapy Access Model, the Centers for Medicare & Medicaid Services said Tuesday.

About half of individuals with sickle cell disease are enrolled in Medicaid, while the disease disproportionately affects Black Americans. Sickle cell disease impacts more than 100,000 people, according to a press release, and the condition leads to long-term health complications and higher rates of hospitalizations.

The CGT Access Model will test outcomes-based agreements for cell and gene therapies. It will begin in 2025 and could expand to other conditions.

“Many of the more than 100,000 Americans with sickle cell disease face difficulty accessing effective health care and groundbreaking treatments," said Department of Health and Human Services Secretary Xavier Becerra in a statement. "While medical advancements bring us closer to cures, too many individuals with sickle cell disease and their loved ones still face challenges obtaining the care they need. With increased investment, we can improve the quality of life for people affected by this disease and find new, potentially transformative treatments.”

In February, CMS released three new models to help lower drug costs, one of those models being CGT. Cell and gene therapies have been hitting the market with sky-high prices, at times costing $1 million or more, which poses a major hurdle for both private and public payers.

CMS will negotiate an outcomes-based agreement with select manufacturers to improve outcomes for people with Medicaid. States will determine whether to join an agreement based on negotiated terms and rebate offers. The agency will likely require manufacturers to "include a defined scope of fertility preservation services when individuals receive gene therapy for treatment."


Sickle cell gene therapy process may cause cancer-linked mutations in blood stem cells

“Gene therapies for sickle cell disease have the potential to treat this devastating condition and transform people’s lives, offering them a chance to live healthier and potentially avoid associated health issues,” said CMS Administrator Chiquita Brooks-LaSure in a news release. “Increasing access to these promising therapies will not only help keep people healthy, but it can also lead to savings for states and taxpayers as the long-term costs of treating sickle cell disease may be avoided.”