(49) Utilization management and coverage restrictions for gene therapies in rare pediatric diseases: a systematic review of U.S. payer policies from 2019 to 2025

Background: : Gene therapies for rare pediatric diseases offer curative potential but face access barriers due to payer-imposed restrictions and inconsistent coverage criteria. High upfront costs, specialized administration, and variation in benefit design contribute to delays in care. Differences in utilization management practices across payers raise equity concerns for pediatric patients.

Objective: : To evaluate U.S. payer coverage of FDA-approved gene therapies for rare pediatric indications between 2019 and 2025, focusing on eligibility requirements, prior authorization, benefit design, and alignment with regulatory labeling.

Methods: : A systematic review was conducted of payer policy documents published from January 2019 to May 2025. We analyzed 126 coverage documents for seven gene therapies (Zolgensma, Luxturna, Skysona, Roctavian, Elevidys, Hemgenix, Upstaza) across 25 Medicaid programs, 15 commercial insurers, and 8 Medicare Advantage plans. Extracted data included clinical restrictions, site-of-care limits, value-based contract references, and out-of-pocket costs. FDA alignment was assessed based on concordance with approved indications.

Results: : All payers required prior authorization and genetic confirmation. Among commercial insurers, 87% applied criteria more restrictive than FDA labeling, including age limits, specific mutations, and functional status thresholds. In contrast, 48% of Medicaid programs imposed similar restrictions, though 64% relied on individualized reviews. Commercial policies included a median of five utilization management elements per therapy, versus three for Medicaid. Most payers required administration at specialized centers. Commercial cost-sharing ranged from 20% to 40%, while Medicaid typically imposed minimal or no patient cost. Medicare Advantage plans followed Part B cost-sharing rules and generally lacked therapy-specific coverage guidance. From 2019 to 2025, alignment between commercial coverage and FDA labeling increased from 42% to 58%.

Conclusions: : Coverage for pediatric gene therapies remains variable and often restrictive. Commercial payers frequently impose criteria that go beyond FDA-approved indications, while Medicaid offers more flexible but non-standardized approaches. Improved alignment with regulatory labeling and simplified authorization processes may reduce barriers to access and promote more equitable coverage.

Sponsorship:: None