OBJECTIVES: Cell and gene therapies (CGTs) are potentially curative treatments for a growing array of diseases. CGTs pose a particular challenge to health care payers due to their one-time administration, high costs and uncertain long-term benefits. Most patients who are eligible for CGTs are covered by Medicaid or Commercial health plans. In this study we assessed variation in Medicaid and Commercial coverage for 11 CGTs.

METHODS: We used the Tufts Medical Center Specialty Drug Evidence and Coverage (SPEC) Database to identify coverage policies for 11 FDA-approved CGTs issued by 17 large US commercial payers as of April 2021. We also searched individual state websites to identify Medicaid policies. We categorized policies according to type of restriction: subgroup (i.e., age, gene status, diagnostic criteria), step therapy protocol (patients are required to first try an alternative therapy), and prescriber requirements (a particular type of specialist must prescribe the drug) and compared these to the FDA label.

RESULTS: Our sample included 167 Medicaid and 198 Commercial CGT coverage policies. Medicaid policies were more likely to include restrictions beyond the FDA label than commercial policies (68% versus 54%, respectively). Restrictiveness varied across CGTs, with plans imposing few restrictions for access to Imlygic for melanoma, while restrictions were common for Luxturna for genetic retinal dystrophy and Zolgensma for spinal muscular atrophy. Both Medicaid and Commercial plans varied in clinical requirements such as frailty or functional score, life expectancy, genetic factors, disease severity, or age. Step therapy protocols were uncommon.

CONCLUSIONS: Medicaid and Commercial plans frequently use coverage restrictions beyond the FDA label for access to CGTs, however the use of restrictions varies depending on type of plan and CGT. We identified significant variation in coverage restrictions for CGTs both within and across Medicaid and Commercial plans, suggesting inconsistent patient access to these important therapies.