Increasing Access to Revolutionary Sickle Cell Therapies

In the past few years, we’ve witnessed incredible advancements in treatments for pediatric diseases. Thanks to groundbreaking gene and cell therapies (CGT), conditions once considered untreatable are now potentially curable. For instance, two revolutionary treatments offer hope for people with sickle cell disease (SCD), a genetic disorder caused by a mutation in hemoglobin.

The Food and Drug Administration approved two CGTs for SCD in late 2023. Casgevy, developed by Vertex Pharmaceuticals, and Lyfgenia, made by bluebird bio, are approved for use in people over 12.

Even so, very few people with SCD have received these therapies, in large part due to their complexity and cost. The therapies are one-time treatments, but they are resource intensive. They require extended hospital stays and highly specialized care from a team of providers. CGTs are also extremely expensive. The list price for Casgevy is $2.2 million; Lyfgenia is $3.1 million. These and other factors present access challenges for children who desperately need treatment.

That’s why we’re pleased Vertex Pharmaceuticals and Bluebird Bio have agreed to participate in the Centers for Medicare & Medicaid Services' (CMS) Gene and Cell Therapy Access Model.

CMS created the model in January 2024 to improve health outcomes, increase access to cell and gene therapies, and potentially address historic disparities, poor health outcomes, and low life expectancy associated with sickle cell disease.

For the estimated 100,000 Americans living with sickle cell disease, the announcement brings renewed hope and is a testament to the power of collaboration across health care, children’s hospitals, and policymakers.

“We hope this first step paves the way for more participation,” said CHA President and CEO Matthew Cook. “As states participate in the model, Medicaid beneficiaries — which include nearly half of the nation's children — may have greater access to these lifechanging treatments.”

How the access model works

The CGT Access Model brings CMS, states, and drugmakers together to improve access to FDA-approved cell and gene therapies — primarily through outcomes-based payment agreements (OBAs).

Under the voluntary model:

• CMS negotiates a contract with pharmaceutical manufacturers on behalf of states. The terms include a standard access policy and prices (including a variety of rebates) tied to outcomes.
• State Medicaid agencies decide whether to sign the negotiated contract.

The result:

• Manufacturers provide rebates and price concessions to states based on outcomes, making the therapy more affordable.
• States implement access policies, allowing more people access to the therapies.

Given Medicaid’s complex processes and regulations, OBAs are not commonly used. The CGT Access Model addresses these issues by having CMS help negotiate terms, manage data, and simplify the process.

The model will initially include therapies for sickle cell disease with plans to expand to other CGTs. CMS noted that between 50% and 60% of people living with SCD are enrolled in Medicaid, live in underserved communities with limited access to specialized care, and experience significant health disparities.

"Children’s hospitals welcome the expansion of treatment access for individuals with sickle cell disease,” Cook said. “CHA remains committed to advocating for and supporting the model while encouraging all state Medicaid programs to participate, advancing health equity in addressing this life-altering disease.”

States can apply to participate in the CGT Access Model until Feb. 28.