Sickle Cell Disease Announced as Initial Focus of Federal CGT Access Model   

The disease afflicts about 100,000 Americans and drastically lowers life expectancy.  

Federal officials announced last week that sickle cell disease (SCD) will be the initial focus of the Cell and Gene Therapy (CGT) Access Model introduced last year, heralding improved access to such therapies and lower healthcare costs for the estimated 100,000 Americans afflicted with the painful and chronic condition.

SCD, which is often hereditary, disproportionately impacts Black Americans and has limited treatment options, with life expectancy among adults with the disease being about 20 years below the baseline average. Additionally, many long-term health complications from SCD, such as stroke, acute chest syndrome, and chronic end-organ damage, can lead to higher rates of emergency department visits and hospitalizations.

“Medical advancements are bringing us closer to a cure for sickle cell disease (SCD). Yet, many of the more than 100,000 Americans with SCD today face difficulty accessing effective healthcare and groundbreaking treatments,” U.S. Department of Health and Human Services (HHS) Secretary Xavier Becerra said in a statement. “Our CGT Access Model gives us a chance to streamline a cumbersome process and put transformative cell and gene therapies within reach for Americans with sickle cell disease.”

The CGT Access Model is part of the Biden Administration’s broader effort to reduce prescription drug costs, developed in response to an executive order Biden issued in October 2022 directing HHS to consider developing models that increase access to novel therapies and lower drug costs. The model, led by the Centers for Medicare & Medicaid Services (CMS) Innovation Center, will test outcome-based agreements (OBAs) for what officials labeled “groundbreaking” CGTs. The model will begin in 2025 and may be expanded to other types of CGTs in the future.

“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,” CMS Administrator Chiquita Brooks-LaSure said. “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.”

A majority – 50 to 60 percent – of Americans living with SCD are enrolled in Medicaid, and officials estimated that hospitalizations and other health episodes related to SCD cost almost $3 billion annually. Gene therapies for treatment of SCD, as well as other complex conditions, hold significant promise, but often come with a high price tag, presenting challenges to state budgets.

Officials said CMS plans to partner with participating states and manufacturers to build a framework expanding 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, according to a press release issued by CMS last week. Negotiations will also reportedly include additional pricing rebates and a standardized access policy, and participating states will 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 said it will also negotiate financial and clinical outcome measures with drug manufacturers and then reconcile data, monitor results, and evaluate outcomes. States can begin participation at a time of their choosing between January 2025 and January 2026.

“The goal of the Cell and Gene Therapy Access Model is to increase access to innovative cell and gene therapies for people with Medicaid by making it easier for states to pay for these therapies,” said Liz Fowler, CMS Deputy Administrator and Director of the CMS Innovation Center. “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.”

Officials also said they anticipate 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 multi-disciplinary, comprehensive care for people with Medicaid with SCD receiving gene therapy,” a press release from CMS read. “These activities may include expanding or increasing reimbursement rates for optional Medicaid benefits and services, such as behavioral health or care management services.”

To view a fact sheet on the CGT Access Model, go to https://www.cms.gov/files/document/cgt-model-ovw-fact-sheet.pdf.