Presentation (CTI)

OBJECTIVES: As more “breakthrough” gene therapies enter the market, previous optimism is met with increasing skepticism as payers contemplate the increasing financial implications of gene therapy proliferation. Payers have mitigated the financial impact of some gene therapies by restricting eligible patient populations (e.g., onerous access criteria). Accordingly, several gene therapy manufacturers offer plans outcomes-based agreements to share coverage risk (e.g., financial, efficacy, durability) in exchange for favorable access. This research analyzes the gene therapy pricing and market access landscape and the success of outcomes-based agreements relative to a previous analysis.
METHODS: Gene therapy management policies with FDA approvals as of January 2025 were pulled from seven national MCOs and thirteen regional plans (n=201.9M covered lives) then compared to corresponding FDA Package Inserts and pivotal clinical trial inclusion/exclusion criteria. Pricing trends, financial reports including ICER papers, on-label competitors, and press releases from gene therapy manufacturers were used to assess pricing and market access trends, including risk mitigation strategies.
RESULTS: Restrictive management is increasing as more gene therapies launch, especially ultra-high-cost products. The newest gene therapies include Beqvez and Kebilidi, among others. Payers’ management approaches include delayed policy decisions, occasional non-coverage, and enforcement of criteria beyond trial in prior authorizations. Payers most frequently require patients to match inclusion/exclusion criteria of those that were studied in trials in their prior authorizations. To mitigate the risk of non-coverage, manufacturers employ site of care contracting, value-based approach contracting, and multi-year payment contracts.
CONCLUSIONS: For gene therapies, the proportion of covered lives facing non-coverage has increased. Ultra-high-cost therapies continue to face additional restrictions compared to lower-priced gene therapies. Therapies on market longer face less restrictive management, likely due to contracting strategies. Products offering outcomes-based agreements see less restrictive management. As payers grow comfortable denying coverage, manufacturers must focus on aligning price to value and innovative contracting to mitigate risks.