Creating win-win-win situations with managed entry agreements?: Prioritizing gene and cell therapies within the window of opportunity

Marcelien H E Callenbach; Wim G Goettsch; Aukje K Mantel-Teeuwisse; Mark Trusheim

doi:10.1016/j.drudis.2024.104048

Creating win-win-win situations with managed entry agreements? Prioritizing gene and cell therapies within the window of opportunity

Marcelien H E Callenbach, Wim G Goettsch, Aukje K Mantel-Teeuwisse, Mark Trusheim^*

^*Corresponding author for this work

Tufts Medical Center

Research output: Contribution to journal › Short survey › peer-review

Abstract

Outcome-based reimbursement models are gaining attention for managing the clinical uncertainties and financial impact of gene and cell therapies. Little guidance exists on how such models can create win-win-win situations, benefiting health-care payers, health-technology developers and patients. Our innovative approach prospectively prioritizes therapies for which a 'window of opportunity' might occur through the analysis of health-technology assessments and product characteristics. Within this window, one size does not fit all, and depending on the extent of clinical uncertainty and potential added benefit levels, different win-win-win situations exist in the United States, the United Kingdom and the Netherlands. Dutch Horizon scanning data prioritized etranacogene dezaparvovec (Hemgenix) and mozafancogene autotemcel for their potential to benefit from outcome-based reimbursement models. These insights extend beyond gene and cell therapies, and could help to provide sustainable health care and patient access to innovative therapies.

Original language	English
Article number	104048
Journal	Drug Discovery Today
Volume	29
Issue number	7
Early online date	1 Jun 2024
DOIs	https://doi.org/10.1016/j.drudis.2024.104048
Publication status	Published - Jul 2024

Bibliographical note

Publisher Copyright:
© 2024

Funding

The authors thank Francine Brinkhuis for validating a subset of the analysis and Christine Leopolds for critically reviewing the paper. The Dutch National Health Care Institute funded this project as part of the Research Network for Health Technology Assessment. The results were not contingent on the sponsor's approval. The funder as an institute had no role in the design and conduct of the study; collection, management, analysis, and interpretation of the data; preparation, review, or approval of the manuscript; and decision to submit the manuscript for publication. The views expressed in this article are the personal views of the authors and may not be understood or quoted as being made on behalf of or reflecting the position of the agencies or organizations with which the authors are affiliated. The authors report no other conflicts of interest relevant to this study. Concept and design: M.H.E.C. W.G.G. A.K.M-T. and M.T. Acquisition of data: M.H.E.C. Analysis and interpretation of data: M.H.E.C. W.G.G. A.K.M-T. and M.T. Drafting of the manuscript: M.H.E.C. Critical revision of the paper for important intellectual content: M.H.E.C. W.G.G. A.K.M-T. and M.T. Obtaining funding: A.K.M-T. W.G.G. and M.T. Supervision: A.K.M-T. W.G.G. and M.T.

Funders	Funder number
Dutch National Health Care Institute

Keywords

ATMPs
Delayed payments
Gene and cell therapies
Horizon scanning
Managed entry agreements
Outcome-based reimbursement

Access to Document

10.1016/j.drudis.2024.104048Licence: CC BY

1-s2.0-S1359644624001739-mainFinal published version, 2.5 MBLicence: CC BY

Cite this

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title = "Creating win-win-win situations with managed entry agreements?: Prioritizing gene and cell therapies within the window of opportunity",

abstract = "Outcome-based reimbursement models are gaining attention for managing the clinical uncertainties and financial impact of gene and cell therapies. Little guidance exists on how such models can create win-win-win situations, benefiting health-care payers, health-technology developers and patients. Our innovative approach prospectively prioritizes therapies for which a 'window of opportunity' might occur through the analysis of health-technology assessments and product characteristics. Within this window, one size does not fit all, and depending on the extent of clinical uncertainty and potential added benefit levels, different win-win-win situations exist in the United States, the United Kingdom and the Netherlands. Dutch Horizon scanning data prioritized etranacogene dezaparvovec (Hemgenix) and mozafancogene autotemcel for their potential to benefit from outcome-based reimbursement models. These insights extend beyond gene and cell therapies, and could help to provide sustainable health care and patient access to innovative therapies.",

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