Review of Gene Therapy Access Landscape
Author(s)
Fraser M1, Brooks K2, Desai A3, Goldstein L1, Privett B4
1Red Nucleus, Cambridge, MA, USA, 2Red Nucleus, Denver, CO, USA, 3Red Nucleus, San Francisco, CA, USA, 4Red Nucleus, Brookline, MA, USA
Presentation Documents
OBJECTIVES:
Gene therapies are often met with optimism and considered "breakthrough innovations" and the "future of medicine.” But their financial impact to health system may concern payers. Payers may mitigate the financial impact of gene therapies by restricting eligible patient populations (e.g., stringent access criteria). To mitigate aggressive market access restrictions, manufacturers offer plans outcomes-based agreements to alleviate concerns (e.g., financial, efficacy, durability, etc.) in exchange for favorable access. This research analyzes the current gene therapy pricing and market access landscape and the success of those treatments.METHODS:
Gene therapy management policies with FDA approvals as of January 2024 were extracted 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 over time as more gene therapies launch, especially ultra-high-cost products. The newest gene therapies approved include Roctavian (2023 launch, $2.9M WAC) Elevidys (2023 launch, $3.2M WAC), and Hemgenix (2022 launch, $3.5M WAC). Payers’ management approaches include non-coverage and enforcing criteria beyond trial in prior authorizations. They also use solely trial criteria in 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 increased since the first therapy, Luxturna, launched in 2017. Recently launched ultra-high-cost therapies face additional restrictions compared to lower-priced (<$3M) gene therapies. Therapies on the market longer (e.g., Luxturna, Zolgensma) employ more types of contracting strategies and face less restrictive management. As payers grow comfortable denying coverage, manufacturers must focus on aligning price to value and innovative contracting to mitigate risks.Conference/Value in Health Info
2024-05, ISPOR 2024, Atlanta, GA, USA
Value in Health, Volume 27, Issue 6, S1 (June 2024)
Code
HPR142
Topic
Health Policy & Regulatory
Topic Subcategory
Coverage with Evidence Development & Adaptive Pathways, Reimbursement & Access Policy, Risk-sharing Approaches
Disease
Genetic, Regenerative & Curative Therapies, Personalized & Precision Medicine, Rare & Orphan Diseases