33 states to participate in CMS' Cell and Gene Therapy Access Model

The Centers for Medicare and Medicaid Services has named 33 states, as well as Washington, D.C., and Puerto Rico, as participants in its Cell and Gene Therapy Access Model, which is designed to treat people in Medicaid living with sickle cell disease.

Participating states represent approximately 84% of Medicaid beneficiaries with the condition, CMS said.

Spearheaded by the CMSA Innovation Center, the agency said the model is the first time the federal government has negotiated outcomes-based agreements with CGT manufacturers on behalf of state Medicaid agencies. Under the model, participating states receive guaranteed discounts and rebates from participating CGT manufacturers if the therapies fail to deliver their promised therapeutic benefits.

CMS Administrator Dr. Mehmet Oz said the federal government is “giving states the tools to deliver lifesaving therapies to patients in need, while holding manufacturers accountable for outcomes.”

WHAT’S THE IMPACT

Sickle cell disease is often a very painful condition. Many long-term health complications from sickle cell disease – including stroke, acute chest syndrome and chronic end-organ damage – can lead to higher rates of emergency department visits and hospitalizations, and higher long-term health care expenditures, said CMS.

The model includes several key features, including CMS-negotiated outcomes-based contracts with manufacturers, developed with input from state Medicaid agencies, patients and providers.

It also includes optional federal support of up to $9.55 million per state to help with implementation, outreach and data tracking.

Participating states have flexible start dates – between January 2025 and January 2026 – and CMS is leaving open the possibility that the model could extend to other diseases in the future.

The model is voluntary for states and manufacturers. Participation is open to manufacturers that responded to CMS’ Request for Applications and agreed to negotiate terms based on clinical outcomes, the agency said.

The states participating in the model are Arizona, Arkansas, California, Colorado, Connecticut, Delaware, Florida, Illinois, Kansas, Kentucky, Louisiana, Maine, Maryland, Michigan, Mississippi, Missouri, New Jersey, New York, North Carolina, Ohio, Oklahoma, Oregon, Pennsylvania, Rhode Island, South Carolina, Tennessee, Texas, Utah, Vermont, Virginia, Washington, West Virginia and Wisconsin.

THE LARGER TREND

In simplistic terms, gene therapy alters a patient's genome through the removal and replacement of strands of DNA. It's currently being used at Children's National Hospital in Washington, D.C., to treat several diseases, including spinal muscular atrophy, a genetic disorder with symptoms similar to that of muscular dystrophy. 

FDA approvals in December include Casgevy and Lyfgenia, representing the first cell-based gene therapies for the treatment of sickle cell disease in patients ages 12 and older.

By 2030, 54 approved gene and cell therapies are expected in the FDA pipeline.