Value‑Based Contracting for Cell and Gene Therapies: A Scoping Review of Payer-Manufacturer Agreements and Treatment Center Considerations in the United States

Abstract

Cell and gene therapies (CGTs) offer the potential for durable or curative outcomes but come with high upfront costs and uncertainty about long-term safety and effectiveness. To address these challenges, manufacturers and payers have expressed sustained interest in value-based contracts (VBCs), which tie reimbursement to real-world outcomes. While publicly disclosed VBCs have been most visible around earlier CGT launches, details on their structure, assessed outcomes, and operational execution remain limited. For treatment centers, understanding this landscape is important because they are well positioned to support data collection, outcome measurement, and validated reporting in the future. These areas represent some of the most commonly cited challenges by manufacturers and payers, suggesting a potential opportunity for treatment centers as uptake of VBCs is anticipated to increase. This study was conducted to review publicly reported VBCs between payers and CGT manufacturers in the United States and characterize outcomes assessed, implementation features, reporting gaps, and implications for treatment center involvement. A scoping review of peer-reviewed publications and press releases (January 2017 to January 23, 2026) describing VBCs involving CGTs was conducted across 7 databases: PubMed, Embase, International Pharmaceutical Abstracts, ABI/INFORM, Factiva, Newspaper Source Plus, and Regional Business News. Included articles described real-world VBCs between manufacturers and payers in the United States and are written in English. Exclusion criteria included VBCs between stakeholders other than manufacturers and payers and VBCs implemented in countries other than the United States. Extracted elements included therapy type, payer/manufacturer involvement, outcome measures, contract structure, and any disclosed adjudication. Of 1324 screened records, 16 studies/press releases met the inclusion criteria, describing 23 VBCs across such conditions as spinal muscular atrophy, sickle cell disease, beta-thalassemia, inherited retinal disorders, hemophilia, acute lymphoblastic leukemia, and rare metabolic and dermatologic diseases. Most VBCs involved Medicaid and commercial payers (e.g. Cigna, Harvard Pilgrim Health Care). Reported outcomes focused on patient-level milestones tied to clinical benefit; for example, early remission status, improvement in functional visual testing, sustained transfusion independence, or the need to resume prophylaxis over prespecified time windows (ranging from 1 month to 5 years). Considerable variability was observed across contracts in terms of outcome definitions, assessment time horizons, and contractual structures. Additionally, no contracts disclosed how these outcomes were assessed, adjudication processes, third-party roles, or contract performance results, leaving practical implementation details largely unknown. Current VBCs for CGTs show substantial variability and minimal reporting, offering limited insight into outcome definitions, assessment timing, or how models are implemented in practice. As broader interest in VBCs increases, treatment centers may have an opportunity to help address implementation and outcome measurement challenges frequently cited by payers and manufacturers. Their experience with clinical assessment, longitudinal follow-up, and existing CGT program operations could help inform feasible, clinically meaningful approaches to scaling the adoption of VBCs.

Keywords: Cell and gene therapy; Outcomes-based agreements; Payer-manufacturer agreements; Treatment centers; Value-based contracting.