Sickle Cell Disease Gene Therapies to Be Part of Initial Focus for Biden-Harris Administration’s CGT Access Model

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The new Cell and Gene Therapy Access Model will be led by the Centers for Medicare & Medicaid Services’ Innovation Center.

Liz Fowler, PhD, JD, the CMS deputy administrator and director of the CMS Innovation Center

Liz Fowler, PhD, JD
Credit: CMS

The Biden-Harris Administration’s new Cell and Gene Therapy (CGT) Access Model, which is led by the Centers for Medicare & Medicaid Services (CMS)'s Innovation Center, will initially focus its efforts on improving access to CGT treatment options for patients with sickle cell disease (SCD).1

To prepare for the advent of the model, which is slated to become active in January 2025, CMS will negotiate with CGT manufacturers over the coming year in order to obtain outcomes-based agreements that will determine pricing for SCD CGTs based on their effect on the health outcomes of patients with Medicaid. Furthermore, US states that partake in the model may obtain CMS-negotiated rebates by offering an agreed-upon standard access policy in accordance the terms negotiated between CMS and the manufacturers. An additional role of CMS in the model will be to negotiate financial and clinical outcome measures with the aforementioned companies and to then assess the actual outcomes. States may begin participating in the model at any time from January 2025 through January 2026.

“The goal of the CGT 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,” Liz Fowler, PhD, JD, the CMS deputy administrator and director of the CMS Innovation Center, said in a statement.1 “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.”

Just a few months ago, on December 8, 2023, the FDA approved both Vertex Pharmaceuticals' and CRISPR Therapeutics’ autologous gene-edited cell therapy exagamglogene autotemcel (exa-cel), marketed under the name Casgevy, and bluebird bio’s autologous gene-edited cell therapy lovotibeglogene autotemcel (lovo-cel), marketed as Lyfgenia, for SCD indications.2,3 Together, the 2 therapies constitute the first gene therapies to receive FDA approval for SCD.

“Gene therapies for SCD 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,” Chiquita Brooks-LaSure, MPP, the CMS administrator, added to the statement.1 “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 SCD may be avoided.”

In addition to the aforementioned elements of the model, CMS noted that it is also working to require CGT manufacturers to offer fertility preservation services within a defined scope for patients receiving SCD gene therapies. Furthermore, CMS will seek to entice states to increase equitable access to CGTs and to promote comprehensive, multidisciplinary care for patients on Medicaid who are undergoing gene therapy for SCD by offering optional funding, such as broadened or heightened rates of reimbursement for optional Medicaid benefits and services.

The CGT Access Model is 1 of 3 models that were selected for testing by the CMS early last year.4 The models were announced after President Biden gave Executive Order 14087 in October 2022, “Lowering Prescription Drug Costs for Americans,” directing the Department of Health and Human Services to work to lower costs. In addition to the CGT Access Model, the models include the Accelerating Clinical Evidence Model, which will seek to develop payment methods for drugs approved under the accelerated approval pathway, and a model that will cap “high-value drugs” for chronic conditions like high blood pressure and diabetes.

"The 3 proposed payment models have the potential, if implemented properly, to make gene and cell therapies more accessible to all who stand to benefit from them," Jeffrey S. Chamberlain, PhD, Professor and McCaw Chair, Muscular Dystrophy, and director, Sen. Paul D. Wellstone Muscular Dystrophy Specialized Research Center, University of Washington School of Medicine, and president-elect, American Society of Gene and Cell Therapy (ASGCT), told CGTLive™ in February 2023. "Access is a key component of ASGCT’s mission as advocates for the clinical application of genetic and cellular therapies. Ultimately, our hope is that these models improve both quality of care and access to transformative therapies."

REFERENCES
1. Biden-Harris administration announces action to increase access to sickle cell disease treatments. News release. Centers for Medicare & Medicaid Services. January 30, 2024. Accessed February 6, 2024. https://www.cms.gov/newsroom/press-releases/biden-harris-administration-announces-action-increase-access-sickle-cell-disease-treatments
2. FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease. News release. FDA. December 7, 2023. Accessed February 6, 2024. https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-disease
3. FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease. News release. FDA. December 8, 2023. Accessed February 6, 2024. https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-disease
4. HHS Secretary Responds to the President’s Executive Order on Drug Prices. News release. Centers for Medicaid & Medicare Services. February 14, 2023. Accessed February 6, 2024. https://www.cms.gov/newsroom/press-releases/hhs-secretary-responds-presidents-executive-order-drug-prices
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