Medicaid coverage practices for approved gene and cell therapies: Existing barriers and proposed policy solutions

Abstract

The current Medicaid system is ill equipped to handle the anticipated approvals of new gene and cell therapy products. These advanced therapies tend to be single-dose, potentially durable options for a variety of indications spanning oncology, rare disease, and more. The up-front cost of these therapies contrasts with chronic care treatment, which may incur cost over the life of a patient. The cost of these innovative treatments, along with the anticipated larger patient pools, can limit patient access as Medicaid programs operate on limited or fixed budgets. Given the value of these therapies for diseases that may have large Medicaid populations, the system will need to grapple with the existing barriers to access to ensure equitable patient care. This review focuses on one such barrier, discrepancies between product indications and state Medicaid and Medicaid Managed Care Organization coverage policies, and it proposes federal policy solutions to this barrier to better accommodate the exponential growth of the gene and cell therapy pipeline.

Keywords: CMS; Medicaid; cell therapy; coverage to label; development pipeline; gene therapy; patient experience; payment policy; time to treatment; value-based payment.

Graphical abstract

Figure 1

Summary of “to labeled indication” coverage determinations for 16 states and three MCOs.