Private Health Insurers Frequently Limit Access to Cell and Gene Therapies, Study Suggests

New research from the Center for the Evaluation of Value and Risk in Health (CEVR) at Tufts Medical Center suggests that U.S. commercial health insurance providers frequently restrict access to cell and gene therapies, often exceeding FDA-approved labeling. The findings were published Sept. 29 in JAMA.

Using data from CEVR’s Specialty Drug Evidence and Coverage (SPEC) Database, researchers reviewed 541 coverage policies from 18 of the largest U.S. commercial insurers, representing about 70% of the commercially insured population. More than half (52%) of policies included restrictions beyond FDA labeling. Additional restrictions focused on specific patient subgroups (78%), while others required step therapy (10%) or combined both approaches (12%).

Furthermore, restrictions were more common for non-cancer indications (63%) compared with cancer indications (45%). Among subgroup restrictions, 59% aligned with clinical trial criteria, 29% were similar but not identical and 12% were inconsistent. The degree of restrictiveness also varied widely among insurers, with the share of restrictive policies ranging from 20% to 78%.

Investigators noted that the cost for cell and gene therapy – sometimes exceeding $2 million per patient – could pose challenges as additional products enter the market. They suggested that policymakers may consider minimum coverage standards or clearer justification for restrictions.

“These findings highlight the tension insurers face when balancing evidence, cost and patient access,” said James Chambers, PhD, MPharm, professor of medicine at CEVR. “While many restrictions mirror trial eligibility criteria, variation across plans can create inequities in access and logistical barriers for patients and providers.”