US administration advances access to sickle cell disease gene therapies through new funding model

The Biden-Harris Administration's new initiative expands access to FDA-approved sickle cell disease (SCD) gene therapies, LYFGENIA™ and CASGEVY™, through outcomes-based agreements with manufacturers.

The Biden-Harris Administration has introduced a new initiative to expand access to advanced treatments for SCD. The Centers for Medicare & Medicaid Services (CMS) announced agreements with two manufacturers of FDA-approved gene therapies, LYFGENIA™ and CASGEVY™, to participate in the Cell and Gene Therapy Access Model. This model seeks to improve patient health outcomes, broaden access to transformative therapies, and manage healthcare costs.

Led by the Center for Medicare and Medicaid Innovation, the voluntary program uses outcomes-based agreements (OBAs), which link payments to the effectiveness of the therapies in improving patient health. Highlighting the initiative’s potential, CMS Administrator Chiquita Brooks-LaSure stated,

“The Cell and Gene Therapy Access Model will increase access to promising therapies that improve the chances of people living longer, healthier lives. This is a new frontier in providing access for people with sickle cell disease to potentially transformative treatments.”

Addressing the unique challenges of sickle cell disease

SCD is a painful, life-limiting condition that that affects over 100,000 individuals in the United States, disproportionately impacting Black and Hispanic Americans. Of those, 50–60% people with SCD rely on Medicaid for their healthcare needs. The disease often leads to frequent hospitalizations and other serious health complications, costing the healthcare system an estimated $3 billion annually.

Liz Fowler, Deputy Administrator and Director of the CMS Innovation Center, highlighted the significance of the new model in helping the financial challenges for state Medicaid programs stating: “Cell and gene therapies hold significant potential to improve patient outcomes and transform lives, ultimately reducing long-term healthcare spending… This model will afford state Medicaid agencies more budget predictability while helping improve access to these innovative therapies for people with Medicaid with sickle cell disease.”

The recent FDA approval of LYFGENIA (lovotibeglogene autotemcel) and CASGEVY (exagamglogene autotemcel) in December 2023 offers a transformative opportunity for SCD treatment. However, high costs pose significant challenges to state budgets and Medicaid programs. The OBA model aims to balance these financial pressures while ensuring patient access to care.

Experts have identified SCD as a potential test case for OBAs, given the disease’s trackable outcomes. However, challenges surrounding the use of this model remain. As noted in the recently published article, ‘Improving Access to Gene Therapies: A Holistic View of Current Challenges and Future Policy Solutions in the United States’, authors from the National Pharmaceutical Council state:

“SCD could serve as a reasonable test case for OBAs, as it is a disease with outcomes that can potentially be tracked efficiently and objectively. However, selecting appropriate outcomes for SCD poses several challenges, such as the lack of clinically validated patient-reported outcomes, the potential disconnect between clinical trial blood markers and meaningful patient outcomes, and the limitations of claims data in reflecting treatment failure.”

Addressing the financial challenges of gene therapy is also critical. In a second paper published in the Journal of Comparative Effectiveness Research, ‘Managing the Challenges of Paying for Gene Therapy: Strategies for Market Action and Policy Reform in the United States’, the authors note: "As the healthcare payment ecosystem prepares for the growing number of gene therapies entering the market, three key interconnected challenges must be addressed: determining a fair price, managing clinical uncertainty and managing short-term budget impacts."

Implementation and future expansion

The Cell and Gene Therapy Access Model is set to launch in January 2025, with states participating in the Medicaid Drug Rebate Program able to opt in at any time between January 2025 and January 2026. CMS will provide participating states with technical assistance and data infrastructure to implement the model. An optional notice of funding opportunity is available through February 28, 2025.

The initiative reflects the administration’s commitment to addressing high drug costs and improving access to novel therapies. It also aligns with President Biden’s October 2022 Executive Order directing the development of models to increase accessibility and affordability for breakthrough treatments. The program may expand to include other cell and gene therapies in the future.