2026 REVIEW OF GENE THERAPY ACCESS LANDSCAPE
Author(s)
Brian Privett1, Kevin Brooks, M.B.A.2, Madeline Meersman, B.S.3, Kaylee Lee, M.P.H.3;
1Red Nucleus, Senior Analyst, Brookline, MA, USA, 2Red Nucleus, Denver, CO, USA, 3Red Nucleus, Cambridge, MA, USA
1Red Nucleus, Senior Analyst, Brookline, MA, USA, 2Red Nucleus, Denver, CO, USA, 3Red Nucleus, Cambridge, MA, USA
OBJECTIVES: Following continued market entry of more ‘breakthrough’ gene therapies, skepticism of associated financial implications has persisted as payers gain more experience. Payers may mitigate the financial impact of gene therapies by restricting eligible patient populations (e.g., via clinical trials criteria). As a result, outcomes-based agreements may be offered by manufacturers to payers in order to share coverage risk in exchange for favorable access. This research reviews changes to the gene therapy pricing and market access landscape and the success of outcomes-based agreements relative to previous analyses.
METHODS: Gene therapy management policies with FDA approvals as of January 2026 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. Press releases from gene therapy manufacturers, pricing databases, financial reports and ICER papers were used to assess pricing and market access trends, including use of risk mitigation strategies.
RESULTS: Restrictive management continues to grow as more gene therapies launch. The newest gene therapies include Itvisma and Papzimeos, among others. Payers’ management approaches include delayed policy decisions, occasional non-coverage, and enforcement of criteria beyond trial in prior authorizations. Management has evolved in a nuanced manner, with specific products seeing more or less restrictive management. Payers most frequently require patients to match inclusion/exclusion criteria of those that were studied in trials in their prior authorizations. To mitigate noncoverage risk, manufacturers employ site of care contracting, value-based approach contracting, and multi-year payment contracts.
CONCLUSIONS: Ultra-high-cost therapies continue to face additional restrictions compared to lower-priced gene therapies. Therapies on market longer generally face less restrictive management, likely due to contracting strategies. Products offering outcomes-based agreements see less restrictive management. As payers continue to grow comfortable denying coverage, manufacturers must maintain focus on aligning price to value and innovative contracts to appropriately mitigate risks.
METHODS: Gene therapy management policies with FDA approvals as of January 2026 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. Press releases from gene therapy manufacturers, pricing databases, financial reports and ICER papers were used to assess pricing and market access trends, including use of risk mitigation strategies.
RESULTS: Restrictive management continues to grow as more gene therapies launch. The newest gene therapies include Itvisma and Papzimeos, among others. Payers’ management approaches include delayed policy decisions, occasional non-coverage, and enforcement of criteria beyond trial in prior authorizations. Management has evolved in a nuanced manner, with specific products seeing more or less restrictive management. Payers most frequently require patients to match inclusion/exclusion criteria of those that were studied in trials in their prior authorizations. To mitigate noncoverage risk, manufacturers employ site of care contracting, value-based approach contracting, and multi-year payment contracts.
CONCLUSIONS: Ultra-high-cost therapies continue to face additional restrictions compared to lower-priced gene therapies. Therapies on market longer generally face less restrictive management, likely due to contracting strategies. Products offering outcomes-based agreements see less restrictive management. As payers continue to grow comfortable denying coverage, manufacturers must maintain focus on aligning price to value and innovative contracts to appropriately mitigate risks.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
HPR69
Topic
Health Policy & Regulatory
Topic Subcategory
Insurance Systems & National Health Care, Reimbursement & Access Policy, Risk-sharing Approaches
Disease
STA: Genetic, Regenerative & Curative Therapies