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

Jeremy Allen; Diane Berry; Francesca Cook; Ashley Hume; Rayne Rouce; Anirudh Srirangam; Jennifer Wellman; Caitlin McCombs

doi:10.1016/j.omtm.2023.05.015

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

Mol Ther Methods Clin Dev. 2023 May 16:29:513-521. doi: 10.1016/j.omtm.2023.05.015. eCollection 2023 Jun 8.

Authors

Jeremy Allen¹, Diane Berry², Francesca Cook³, Ashley Hume⁴, Rayne Rouce⁵, Anirudh Srirangam⁶, Jennifer Wellman⁷, Caitlin McCombs⁸

Affiliations

¹ Spark Therapeutics, Inc., Philadelphia, PA 19104, USA.
² Sarepta Therapeutics, Inc., Cambridge, MA 02142, USA.
³ REGENXBIO Inc., Rockville, MD 20850, USA.
⁴ Emerging Therapy Solutions, Bloomington, MN 55425, USA.
⁵ Center for Cell and Gene Therapy, Texas Children's Cancer Center, Baylor College of Medicine, Houston, TX 77030, USA.
⁶ Bristol Myer Squibb Company, Washington, DC 20004, USA.
⁷ Akouos, Inc., a Wholly Owned Subsidiary of Eli Lilly and Company, Boston, MA 02210, USA.
⁸ American Society of Gene and Cell Therapy, Waukesha, WI 53186, USA.

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.

Publication types

Review