Evaluating the design and implementation challenges of the Centers for Medicare & Medicaid Services' Cell and Gene Therapy Access Model.

Abstract

Cell and gene therapies (CGTs) offer potentially curative treatments for conditions like sickle cell disease but pose major affordability and access challenges for Medicaid mainly due to multimillion-dollar upfront prices. In response, the Centers for Medicare & Medicaid Services (CMS) introduced the CGT Access Model, a purchasing approach that leverages the Medicaid Drug Rebate Program to implement multistate, outcome-based agreements for CGTs. Through the Model, CMS negotiates core terms with manufacturers, states adopt these terms through rebate agreements, and CMS coordinates data collection and evaluation. The initiative aims to expand access, improve clinical outcomes, and generate long-term cost savings by aligning payment with demonstrated therapeutic benefit. However, critical uncertainties remain, including outcome-based agreement design, access policy standards, moral hazard risks, fertility-related barriers, out-of-state care challenges, and challenges with data collection. Although imperfect, the CGT Access Model marks an important step toward integrating sustainable financing of high-cost, transformative therapies into public insurance programs.