Study Suggests Innovative Value-Based Models for Expanding Cell and Gene Therapy Access
Cell and gene therapies (CGTs) offer groundbreaking approaches for treating rare and complex conditions by providing “living drugs” that repair or replace damaged tissue. Although these therapies hold great potential for treating conditions such as blood disorders, cancers, neurological diseases, and immune conditions, the high costs of CGTs and limitations within the US health care system create barriers to equitable access and affordability. A study published in Journal of Medical Economics reviewed the current landscape of CGTs, as well as challenges and the outlook for the future of these therapies in the US from a financial perspective.
Payers face risks regarding CGTs due to uncertainties around treatment efficacy and long-term benefits, while manufacturers seek sustainable coverage solutions. While only a few CGTs currently have US Food and Drug Administration (FDA) approval, thousands of clinical trials for CGTs are underway, indicating that the pipeline for these therapies will continue to grow in the coming years. To prepare for this, there are key issues that researchers suggest should be considered from current FDA-approved therapies. For example, despite low disease prevalence for hemophilia B and sickle cell disease, large upfront payments to the current FDA-approved CGTs pose substantial financial burdens. The researchers shared concerned about fragmentation in the US health care system, where a lack of continuity in insurance plans for patients undermines installment-based value models that could help mitigate costs of CGTs.
Value-based care methods, particularly outcomes-based contracts (OBCs), are emerging as ways to address these concerns. The Centers for Medicare and Medicaid Services (CMS) have introduced the Cell and Gene Therapy Access model, a voluntary program aimed at expanding CGT access for Medicaid enrollees. Although the Cell and Gene Therapy Access model has helped expand access for Medicaid enrollees, the researchers suggest that a publicly funded special plan leveraging value-based payments and centralized risk management could bridge the gaps in affordability for CGTs. They estimate that a special plan could address coverage gaps in private and public insurance by offering additional benefits they cannot provide, use real-world evidence to adjust payments over time, and lower administrative costs through centralized oversight.
“In conclusion, while CGTs offer promise for those difficult-to-treat and rare conditions, the fragmented insurance market and uncertain long-term benefits may discourage adoption of the technology,” the researchers stated. “Innovative approaches in value-based payments are needed to bridge gaps in insurance coverage and link the outcomes with payment to improve market efficiency and health for millions of Americans.”
Reference
Zhang JX, Shugarman LR. Value-based payment and financing for cell and gene therapies: challenges and potential solutions. J Med Econ. 2024;27(1):678-681. doi:10.1080/13696998.2024.2346406.
