New CMS model will focus on sickle cell treatments
The model is designed to improve outcomes, increase access to therapies and lower healthcare costs for vulnerable populations.
Photo: Marko Geber/Getty Images
Sickle cell disease (SCD) will be the initial focus of the Centers for Medicare and Medicaid Services Cell and Gene Therapy (CGT) Access Model, which was initially announced in February 2023.
The model is designed to improve health outcomes, increase access to cell and gene therapies, and lower healthcare costs for vulnerable populations.
CMS described sickle cell disease as extraordinarily painful and an affliction that disproportionately affects Black Americans and historically has had limited treatment options. In the U.S., more than 100,000 people live with SCD. Sickle cell disease is a group of inherited red blood cell disorders.
Individuals with the disease have a shorter life expectancy by more than 20 years, compared to someone living without SCD. And many long-term health complications from SCD – including stroke, acute chest syndrome and chronic end-organ damage – can lead to higher rates of emergency department visits and hospitalizations, CMS said. Patients with SCD experience challenges with access to quality and affordability of care.
The model is designed to help improve health outcomes for patients and families with SCD while also ensuring taxpayer dollars are being used more effectively, CMS said.
WHAT'S THE IMPACT?
The CGT Access Model is part of the administration's broader effort to further drive down prescription drug costs. It was developed in response to an executive order President Biden issued in October 2022 directing the Department of Health and Human Services to consider developing models that increase access to novel therapies and lower the high cost of drugs.
The model, led by the CMS' Innovation Center, will test outcomes-based agreements (OBAs) for groundbreaking cell and gene therapies. Successful OBAs, CMS said, will increase affordable access to potentially lifesaving and life-changing treatment. The model will begin in 2025 and may be expanded to other types of CGTs in the future.
About 50 to 60% of people living with SCD are enrolled in Medicaid. Hospitalizations and other health episodes related to SCD cost the health system almost $3 billion per year, according to CMS data.
Over the next year, CMS will partner with participating states and manufacturers to build a framework that expands access to gene therapies for the treatment of SCD. Under the model, CMS will negotiate an OBA with participating manufacturers, which will tie pricing for SCD treatments to whether the therapy improves health outcomes for people with Medicaid. Negotiations will also include additional pricing rebates and a standardized access policy.
Participating states will then decide whether to enter into an agreement with manufacturers based on the negotiated terms and offer the agreed-upon standard access policy in exchange for rebates as negotiated by CMS. As part of the CGT Access Model, CMS will negotiate financial and clinical outcome measures with drug manufacturers and then reconcile data, monitor results and evaluate outcomes.
The CGT Access Model will begin in January 2025, and states may choose to begin participation at a time of their choosing between January 2025 and January 2026.
THE LARGER TREND
CMS anticipates addressing additional care delivery gaps and other hurdles for people receiving cell and gene therapy during the OBA negotiation process, including requiring manufacturers to include a defined scope of fertility preservation services when individuals receive gene therapy for treatment of SCD.
CMS will also offer optional funding to states that engage in activities that increase equitable access to cell and gene therapies and promote multidisciplinary, comprehensive care for people with Medicaid with SCD receiving gene therapy. These activities may include expanding or increasing reimbursement rates for optional Medicaid benefits and services, such as behavioral health or care-management services.
Jeff Lagasse is editor of Healthcare Finance News.
Email: jlagasse@himss.org
Healthcare Finance News is a HIMSS Media publication.
