A Targeted Literature Review of Value-Based Agreements (VBAs) for Cell and Gene Therapies in the United States
Author(s)
Ryan A. Babakhani, MS, PharmD1, Darrin Wade Benjumea, BS, MPH2, Alice E. Jackson, MRes2, John J. Ko, MS, PharmD1.
1Beam Therapeutics, Cambridge, MA, USA, 2Genesis Research Group, Hoboken, NJ, USA.
1Beam Therapeutics, Cambridge, MA, USA, 2Genesis Research Group, Hoboken, NJ, USA.
Presentation Documents
OBJECTIVES: High costs of cell and gene therapies (CGTs) in the United States (US) have prompted payers to adopt new payment models. Value-based agreements (VBAs) are contracts between pharmaceutical manufacturers and payers where payment/reimbursement is tied to pre-specified and measurable outcomes. Although VBAs are becoming common, terms often remain highly confidential, and no standard approach has been established. The objective of this study is to review publicly available information on the utilization terms of VBAs for CGTs in the US.
METHODS: A review of VBAs for CGTs was conducted based on the CBER list of FDA approved therapies from September 2024. Procedure-related therapies (e.g., cord blood) were excluded. Findings from PubMed, manufacturer/payer press releases, and policy statements were extracted to identify any VBAs. VBA terms, such as payer, length of agreement, and outcomes assessed, were reported.
RESULTS: From the list of FDA approved CGTs, 24 approved CGTs were identified with any mention of VBAs, 10 of which (42%) with limited details of VBA terms publicly available for extraction. The therapies with VBAs in place included: Beqvez, Casgevy, Hemgenix, Kymriah, Luxturna, Lyfgenia, Roctavian, Vyjuvek, Zolgensma, and Zynteglo. Specific outcomes detailed in the VBAs ranged from measures of clinical efficacy (e.g., symptom improvement, patient response) to healthcare resource utilization (e.g., transfusion dependence, hospitalizations).
CONCLUSIONS: In this review, multiple VBAs for CGTs were identified across multiple disease areas. Most payers did not publicly disclose which outcomes measures the VBAs were assessing. Of those that did, outcomes assessed could be sourced from routine patient visits and/or adjudicated claims, placing no additional burden on providers to collect data for the sole purpose of the VBA. VBAs continue to evolve with emerging approaches (e.g., annuities), and initiatives such as the Center of Medicare and Medicaid Innovation’s CGT Access Model will shape a multistakeholder approach to VBAs.
METHODS: A review of VBAs for CGTs was conducted based on the CBER list of FDA approved therapies from September 2024. Procedure-related therapies (e.g., cord blood) were excluded. Findings from PubMed, manufacturer/payer press releases, and policy statements were extracted to identify any VBAs. VBA terms, such as payer, length of agreement, and outcomes assessed, were reported.
RESULTS: From the list of FDA approved CGTs, 24 approved CGTs were identified with any mention of VBAs, 10 of which (42%) with limited details of VBA terms publicly available for extraction. The therapies with VBAs in place included: Beqvez, Casgevy, Hemgenix, Kymriah, Luxturna, Lyfgenia, Roctavian, Vyjuvek, Zolgensma, and Zynteglo. Specific outcomes detailed in the VBAs ranged from measures of clinical efficacy (e.g., symptom improvement, patient response) to healthcare resource utilization (e.g., transfusion dependence, hospitalizations).
CONCLUSIONS: In this review, multiple VBAs for CGTs were identified across multiple disease areas. Most payers did not publicly disclose which outcomes measures the VBAs were assessing. Of those that did, outcomes assessed could be sourced from routine patient visits and/or adjudicated claims, placing no additional burden on providers to collect data for the sole purpose of the VBA. VBAs continue to evolve with emerging approaches (e.g., annuities), and initiatives such as the Center of Medicare and Medicaid Innovation’s CGT Access Model will shape a multistakeholder approach to VBAs.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
HPR93
Topic
Health Policy & Regulatory
Topic Subcategory
Coverage with Evidence Development & Adaptive Pathways, Insurance Systems & National Health Care, Reimbursement & Access Policy
Disease
STA: Genetic, Regenerative & Curative Therapies