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. 2025 May;43(5):583-594.
doi: 10.1007/s40273-025-01474-3. Epub 2025 Feb 21.

Innovative Payment Models for Sickle-Cell Disease Gene Therapies in Medicaid: Leveraging Real-World Data and Insights from CMMI's Gene Therapy Access Model

Antal Zemplenyi  1   2 , Jim Leonard  3 , Garth C Wright  4 , Michael J DiStefano  4 , Kavita Nair  4 , Kelly E Anderson  4 , R Brett McQueen  4
Affiliations

Innovative Payment Models for Sickle-Cell Disease Gene Therapies in Medicaid: Leveraging Real-World Data and Insights from CMMI's Gene Therapy Access Model

Antal Zemplenyi et al. Pharmacoeconomics. 2025 May.

Abstract

Objective: This study aims to evaluate the financial implications of implementing various payment models, including outcome-based agreements (OBAs), volume-based rebates, and guaranteed rebates, for the newly approved gene therapies, exagamglogene autotemcel (exa-cel) and lovotibeglogene autotemcel (lovo-cel), in the treatment of sickle-cell disease (SCD) from the perspective of Colorado Medicaid. The analysis specifically examines the cost of standard of care (SoC) for severe SCD, the impact of different eligibility criteria based on vaso-occlusive events (VOEs), and the potential financial impacts associated with rebate structures.

Methods: Data from the Colorado Department of Health Care Policy & Financing (HCPF) database was used to estimate the annual costs for Medicaid-enrolled patients with severe SCD from 2018 to 2023. Patients were selected based on various eligibility criteria, including the number of VOEs, acute chest syndrome events, and stroke diagnoses. Three-state Markov models (SCD, stable, and dead) were constructed to compare the costs of SoC and gene therapies. The durability of gene therapy effectiveness and the financial impact of OBAs, volume-based rebates, and guaranteed rebates were evaluated over a 6-year contract period, with scenarios reflecting different VOE criteria and treatment durability.

Results: The average annual SoC cost for severe SCD patients (N = 138) was US$45,941 (SD US$59,653), with higher costs associated with more frequent VOEs. Gene therapies exa-cel and lovo-cel, with one-off list prices of US$2.2 million and US$3.1 million, respectively, exhibited high upfront costs, resulting in a negative cumulative balance averaging - US$2.11 million for exa-cel and - US$3.00 million for lovo-cel per patient over 6 years compared with SoC. Outcome-based rebates could potentially save Medicaid approximately US$260K (uncertainty interval 88K-772K) per patient on average for exa-cel and US$367K (uncertainty interval 122K-1111K) for lovo-cel after they pay the full up-front cost. Volume-based and guaranteed rebates also offered potential savings but varied in impact based on contract duration and effectiveness of gene therapy.

Conclusions: The study highlights critical considerations for Medicaid in negotiating OBAs for SCD gene therapies. Achieving budget neutrality over 6 years is unlikely due to low SoC costs. However, payment models can enhance value-based spending by linking high therapy costs and potential rebates to the health gains these treatments may offer. OBAs offer offsets contingent on therapy effectiveness durability and contract terms (such as length and price), while varying eligibility criteria impact budgets and outcomes. Medicaid real-world data is crucial for navigating complexities in defining eligible populations and structuring OBAs.

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Conflict of interest statement

Declarations. Conflict of interest: Dr Leonard is affiliated with the Colorado Department of Health Care Policy & Financing. The authors have disclosed no conflicts of interest concerning the research, authorship, or publication of this article. None of the authors are affiliated with or hold financial interests in the manufacturers of the gene therapies investigated in this study. The views expressed in this article represent the opinions of the authors and do not necessarily reflect those of their respective employers. Funding: The University of Colorado is funded by the Department of Health Care Policy & Financing for the assessment of value-based contracts. Ethics approval: The study was declared exempt by the Colorado Multiple Institutional Review Board. Consent for publication: Not applicable. Consent to participate: Not applicable. Data availability: All data analyzed during this work are from the Colorado Department of Health Care Policy & Financing. The data are not publicly available. The model used during this work was developed by the University of Colorado under a contract with the Colorado Department of Health Care Policy & Financing. Code availability: Not applicable. Author contributions: Antal Zemplenyi, Brett McQueen, Jim Leonard, Michael DiStefano, Kelly Anderson, and Kavita Nair contributed to the study conception and design. Material preparation, data collection and analysis were performed by Garth Wright and Antal Zemplenyi. The first draft of the manuscript was written by Antal Zemplenyi and Brett McQueen, and all authors commented on previous versions of the manuscript. All authors read and approved the final version of the paper.

Figures

Fig. 1
Fig. 1
Visualization of the method of identifying discrete VOEs. OP outpatient, ED Emergency department, VOE vaso-occlusive events
Fig. 2
Fig. 2
Comparison of per-patient costs between exa-cel gene therapy and SoC for SCD over 6 years. GT gene therapy, SCD sickle-cell disease, SoC standard of care
See this image and copyright information in PMC

References

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