Boston, MA, September 30, 2025 — A new study from the Center for the Evaluation of Value and Risk in Health (CEVR) at Tufts Medical Center provides new empirical data on how US commercial health insurers cover cell and gene therapies; finding that they frequently impose restrictions on patient access, in some cases beyond what is specified in the therapies’ FDA-approved labeling.
Cell and gene therapies, single-administration treatments designed to provide durable benefits for patients with serious genetic or cellular diseases, can cost more than $2 million per patient. Payers have responded to their high prices and uncertain long-term outcomes with a variety of coverage approaches, some of which align with the FDA label while others use different mechanisms to narrow coverage.
Using CEVR’s Specialty Drug Evidence and Coverage (SPEC) Database, researchers reviewed 541 coverage policies from 18 of the largest U.S. commercial insurers, representing approximately 70% of the commercially insured market.
Key Findings
- 52% of policies included restrictions beyond FDA labeling.
- Restrictions targeted patient subgroups (78%), required step therapy (10%), or both (12%).
- Restrictions were more common for non-cancer indications (63%) than cancer indications (45%).
- Alignment with trial criteria varied:
- 59% of subgroup restrictions were consistent with trial criteria,
- 29% were similar but not identical, and
- 12% were inconsistent.
- Restrictiveness varied widely across insurers, with the share of policies containing restrictions ranging from 20% to 78%.
Implications
“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.”
The study notes that coverage for cell and gene therapies is likely to remain complex as more products enter the market. Policymakers may need to consider minimum coverage standards or clearer justification for restrictions. Manufacturers can also play a role by generating post-approval evidence and pursuing outcomes-based agreements that address payer uncertainty.
About CEVR
CEVR is a national leader on research on the value of health and medical interventions, with the goal of supporting better societal decision-making on health care spending. CEVR is a part of Tufts Medical Center’s Institute for Clinical Research and Health Policy Studies (ICRHPS). We undertake scientific research projects, advance methods development for the field, train the next generation of practitioners and users, and work with policymakers worldwide to develop reasoned policy solutions.
Contact Information
Matt Seidner, Senior Program Manager
matthew.seidner@tuftsmedicalcenter.org